There has been so much progress in treating cancer in just the past 10 years that it could make your head spin. Researchers have identified new ways to fight cancer -- and new ways to diagnose cancer more effectively, too.

Close to $150 billion will likely be spent on cancer drugs this year. This creates lots of opportunities for investors. But which cancer treatment stocks are the top picks right now? Here's why I think thatbluebird bio (NASDAQ:BLUE), Bristol-Myers Squibb (NYSE:BMY), and Intuitive Surgical (NASDAQ:ISRG) especially stand out.

Image source: Getty Images.

I recently include Bluebird on my list of the three best biotech stocks to buy for the next decade. This list was admittedly a stretch since it's impossible to accurately predict how any stocks will perform in the future. That's especially true for biotech stocks.

However, there are several reasons Bluebird could fly significantly higher. For one thing, the company recently lauched its first product, Zynteglo, in Germany. It will take a while for revenue to pick up for the gene therapy, which targets transfusion-dependent beta-thalassemia. But Bluebird expects to introduce Zynteglo in other key European markets later this year. It also plans for file for U.S. approval in the first half of 2020.

Zynteglo isn't a cancer treatment, but Bluebird has a couple of cell therapies that are. The company's partner, Bristol-Myers Squibb, should file for FDA approval of ide-cel in treating multiple myeloma, a type of blood cancer, this year. Bluebird and BMS are also collaborating on another promising multiple myeloma cell therapy, bb21217. I think that both drugs should become blockbusters if approved.

In addition, Bluebird has another rare disease drug in late-stage testing.The biotech should file for U.S. and European approvals for Lenti-D in treating cerebraladrenoleukodystrophy by late 2020.

Bluebird's partner, Bristol-Myers Squibb, ranks as one of the biggest cancer-focused drugmakers on the planet. Market researcher EvaluatePharma projects that the company's cancer immunotherapy will be the No. 4 top-selling drug in the world within the next few years. BMS has a rising star with its multiple myeloma drug Empliciti. And that's just the tip of the iceberg for BMS.

Thanks to its acquisition of Celgene in late 2019, BMS now has several other blockbuster cancer drugs in its lineup. Revlimid is the biggest moneymaker that BMS picked up in the deal. The company also now claims multiple myeloma drug Pomalyst and solid tumor drug Abraxane.

In addition, BMS's pipeline is bursting at the seams with potential blockbusters. The company expects to win FDA approval for liso-cel in treating relapsed or refractory large B-cell lymphoma by Aug. 17, 2020. We've already mentioned Bluebird's ide-cel, which BMS licensed.

Don't forget BMS's drugs outside the cancer arena. Blood thinner Eliquis should become the No. 3 best-selling drug in the world by 2024. Autoimmune disease drug Orencia continues to perform very well. BMS also hopes to win FDA approval for Celgene's ozanimod in treating multiple sclerosis next month.

You might be a little surprised to see Intuitive Surgical included along with two drugmakers as a top cancer treatment stock to buy right now. But Intuitive's da Vinci robotic surgical system is used extensively in treating prostate cancer, with 138,000 urological procedures performed with the system in the U.S. alone last year.

To be transparent, though, Intuitive's prominence in treating prostate cancer isn't the main reason why I included this stock. What I most like about Intuitive Surgical is its overall business model. The company basically uses a modern version of the old razor-and-blades approach. It makes money by selling robotic surgical systems but makes a whole lot more by selling replacement instruments, accessories, and providing services.

Intuitive Surgical's recurring revenue in 2019 stood at 72% of total revenue. That figure should continue to grow as the install base for da Vinci grows and as customers use the system for more procedures. Intuitive is also leasing more robotic surgical systems than ever before, which will boost recurring revenue even more.

Other companies hope to challenge Intuitive Surgical's dominance in the robotic surgical systems market. My view, though, is that this competition will be a good thing for Intuitive by expanding the market. I also think Intuitive Surgical's huge head start in developing an ecosystem supporting its technology gives it a solid moat.

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3 Cancer Treatment Stocks to Buy Right Now - The Motley Fool

I'll admit that I have no idea which stock will deliver the biggest returns over the next 10 years. If I had to guess, it would probably be a stock that hardly anyone has heard of right now. After all, the unknown tiny stocks have the most room to run.

But identifying the most important stock to own over the next decade is a different story. By my definition, the most important stock will be one that's most likely to have a profound impact on the greatest number of people.

There are several worthy candidates to get the nod as the most important stock for the 2020s. If I had to choose just one stock right now, though, it would be... Alphabet (NASDAQ: GOOG) (NASDAQ: GOOGL).

Image source: Getty Images.

We can look around today to see which trends are likely to matter the most over the next decade. Perhaps the most critical trend is the growing senior population across the world.

The U.S. Census Bureau projects that 2030 will be a turning point for the country with every member of the baby boomer generation over age 65. Nearly 25% of residents of the European Union will be at least 65 years old 10 years from now. Over 25% of Chinese citizens will be at least 60 years old by 2030. The percentages in other heavily populated Asian countries are even higher.

There lots of implications from these demographic trends. Hundreds of millions of people will have more time on their hands, for one thing. More importantly, though, the demand for healthcare products and services will almost certainly increase significantly.

Another key trend that should shape the next decade is the expanded role of artificial intelligence (AI). There has been a dramatic surge in the use of AI in recent years. Just ask your smartphone. But I expect the 2020s will usher in an era where AI is even more pervasive, from smarter apps to self-driving cars. This shift to increased use of AI will likely add fuel to the fire of another unstoppable technology trend -- the migration of apps and data to the cloud.

Speaking of trends that are already under way and should pick up momentum, how often do you use cash these days? If you're like most people, the answer is "at little as possible." The war on cash (i.e., the transition to digital payment methods) should intensify over the next decade.

Of course, there are other important trends that will impact many people in the 2020s. However, few will rival the significance that aging demographics, the increased prevalence of AI, and the war on cash will have.

Quite a few companies will play a role in at least one of these critical trends shaping the next decade. But a handful will be involved in all of them. I'd argue that Alphabet is the one company that covers the major trends from A to Z and that will make the biggest difference overall.

Let's start with aging demographics. Alphabet's apps, particularly Google Search and YouTube, are likely to be used much more frequently as older individuals have more leisure time. The more significant impact for the company, though, could be in healthcare.

Alphabet's Verily Life Sciences subsidiary is working with DexComon the development of its cutting-edge G7 continuous glucose monitoring (CGM) system. Verily is collaborating with several major drugmakers, including Novartisand Pfizer, on Project Baseline -- an effort to map a baseline of human health. This project could change how new drugs are developed and improve the quality of care provided to patients.

Another Alphabet subsidiary, Calico, has an even more ambitious goal: extending the human lifespan. There's no guarantee that Calico will unveil earthshaking developments over the next 10 years, but it might.

But I'd say that it is a certainty that Alphabet will be at the forefront of AI development in this decade. It already ranks as a leader in AI and has the resources to stay on top. Alphabet's Waymo unit expects to expand its fleet of self-driving cabs beyond the initial testbed in Phoenix, Arizona. It's partnered with Jaguar to develop the first fully self-driving car. And Waymo's self-driving big-rig trucks could move out of the testing phase in the not-too-distant future.

Image source: Getty Images.

Alphabet is also addressing two major trends at the same time by applying AI to healthcare. The company's DeepMind AI lab achieved a big breakthrough last year by using AI to predict a potentially fatal kidney disease 48 hours before it became critical. Alphabet's AI can even detect breast cancer more accurately than most human radiologists.

On top of all of this, Alphabet is also a major player in the war on cash with its Google Pay digital payment platform. Google Pay is available on more than 2.5 billion active Android devices worldwide. Alphabet even plans to launch online checking accounts for Google Pay users in 2020.

I don't expect Alphabet will be the best-performing stock of the decade. It's too big already. The good news, though, is that Alphabet's role in the key trends for the next 10 years should also lead to big stock gains.

Despite its $1 trillion market cap, Alphabet is still a growth stock. Its core businesses should power plenty of growth on their own. But I think that the company's healthcare, AI, and digital payment efforts will pay off in a major way, too. My view is that Alphabet is the most important stock to own over the next decade but also a winning stock to own over the next decade.

10 stocks we like better than AlphabetWhen investing geniuses David and Tom Gardner have a stock tip, it can pay to listen. After all, the newsletter they have run for over a decade, Motley Fool Stock Advisor, has tripled the market.*

David and Tom just revealed what they believe are the 10 best stocks for investors to buy right now... and Alphabet wasn't one of them! That's right -- they think these 10 stocks are even better buys.

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Suzanne Frey, an executive at Alphabet, is a member of The Motley Fool's board of directors. Keith Speights owns shares of Alphabet (A shares) and Pfizer. The Motley Fool owns shares of and recommends Alphabet (A shares) and Alphabet (C shares). The Motley Fool recommends DexCom. The Motley Fool has a disclosure policy.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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The Most Important Stock to Own Over the Next Decade - Nasdaq

For Immediate Release: January 28, 2020

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patients cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, said FDA Commissioner Stephen M. Hahn, M.D. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products.

The six final guidances issued today provide the agencys recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies. The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDAs standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often cant be fully answered in pre-market trials of reasonable size and duration. For some gene therapy products, therefore, although they have met the FDAs standards for approval, we may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization. Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDAs proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity. The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the draft guidance and our regulations, the agencys determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).

With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations. The draft guidance has potential positive implications both for product developers and patients by providing insight into the agencys most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition. For patients, this policy could help lead to the development and approval of multiple treatments, creating a more competitive market with choices. We encourage stakeholders to provide their comments.

In sum, these policy documents are representative of efforts to help advance product development in the field of gene therapy. We will continue to work with product innovators, sponsors, researchers, patients, and other stakeholders to help make the development and review of these products more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We also encourage developers of new gene therapy products to make full use of our expedited programs available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions. These programs include breakthrough therapy designation, regenerative medicine advanced therapy designation, and fast track designation, as well as priority review and accelerated approval. Developers should pursue these programs whenever possible to help bring the benefits of important advances to patients as soon as possible. We believe our work will help advance innovations in a way that assures their safety and effectiveness, provides new therapeutic choices to patients and providers and continues to build confidence in this novel and emerging area of medicine.

The FDA is an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov

Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine.

The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies.

Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.

Company executives weren't, however, asked to answer how Pfizer views the emerging gene therapy competition. BioMarin Pharmaceutical looks set to get to the market earlier in hemophilia A than Pfizer, while Uniqure in hemophilia B and Sarepta Therapeutics in Duchenne muscular dystrophy appear ahead.

Pfizer's hemophilia A project, the Sangamo Therapeutics-originated SB-525, is up against BioMarin's valrox, which has been submitted to the Food and Drug Administration for an approval decision later this year.

In hemophilia B, fidanacogene elaparvovec, licensed from Roche subsidiary Spark Therapeutics, is in a neck-and-neck race with UniQure's etranacogene dezaparvovec in Phase 3 testing. Duchenne research, meanwhile, is led by Sarepta, which is launching a Phase 3 trial of its drug this year, putting Pfizer's at a disadvantage.

Other than announcing its intent to launch Phase 3 trials in hemophilia A and Duchenne, Pfizer didn't provide much more detail about these clinical programs. Mikael Dolsten, Pfizer's chief scientific officer, said more could be revealed about the DMD program at an upcoming research & development day.

Progress on that project had been delayed after one patient was hospitalized with kidney complications, but Dolsten said trial investigators had dosed additional patients. The Phase 2 will wrap up this spring, and the new data and longer follow-up will help guide a Phase 3 trial design, the company said.

Dolsten also described the hemophilia A project as having a 'best-in-class profile," even though BioMarin's valrox has impressed hematologists with its ability to increase expression of a key blood-clotting protein.

In addition, he said the company hopes it can bring one new gene therapy into its pipeline per year.

Building its drug development portfolio is one reason why the company has chosen not to buy back shares, said CEO Albert Bourla.

He pointed to the company's need in the past to buy back shares to support their valuation because of revenue declines, but now he said the company is in a different strategic position.

"The company is going to have a best-in-class revenue growth story," he said. "We can use the capital to invest in good Phase 2, Phase 3 assets to grow our pipeline."

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Pfizer lays out gene therapy aspirations - BioPharma Dive

Docket Number: FDA-2019-D-5392 Issued by:

Guidance Issuing Office

Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research

This guidance provides FDAs current thinking on determining sameness of human gene therapy products under FDAs orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity. This guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan-drug designation and orphan-drug exclusivity, in the development of gene therapies for rare diseases. This guidance focuses specifically on factors that FDA generally intends to consider when determining sameness for gene therapy products and does not address sameness determinations for other types of products.

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets ManagementFood and Drug Administration5630 Fishers Lane, Rm 1061Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-5392 .

01/30/2020

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Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov

With more than 900 investigational new drug (IND) applications for ongoing clinical studies related to gene therapies, and with the number of advanced therapy medicinal products at clinical stage worldwide exceeding 1,000, the US Food and Drug Administration (FDA) this week released a number of policies.

The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, commented Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, adding The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.

Regarding the draft guidanceInterpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, the agency explained that it focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

The final guidance titled Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) aims to inform sponsors on how to provide sufficient CMC information, in order to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product and to be able to claim market authorization from the regulatory body.

Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.

Titled Long-Term Follow-Up After Administration of Human Gene Therapy Products, the third document includes recommendations by the FDA regarding the design of long-term follow-up studies for the collection of data on delayed adverse events.

Specifically, the FDA suggests that, as a result of long-term exposure to an investigational gene therapy, patients may be at increased risk of undesirable and unpredictable outcomes, and therefore they may be monitored for an extended period of time past the active follow-up period. The document outlines several factors based on which a risk assessment should be performed to determine the necessity of long-term monitoring for each product.

Another guidance of the FDA is focused on Human Gene Therapy for Hemophilia, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays.

Focusing on Human Gene Therapy for Retinal Disorders, the fourth FDA guidance includes recommendations related to product development, preclinical testing, and clinical trial design for such gene therapy products.

Finally, the guidance on Human Gene Therapy for Rare Diseases, with suggestions on the clinical design for such products, is needed, according to the FDA, due to the limited study population size and potential feasibility and safety issues. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product.

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FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com

DUBLIN, Jan. 30, 2020 /PRNewswire/ -- The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is projected to reach USD 13,005.6 million by 2024 from an estimated USD 3,814.9 million in 2019, at a CAGR of 27.8% during the forecast period.

This report provides a detailed picture of the global gene therapy market. It aims at estimating the size and future growth potential of the market across different segments (by vector, indication, delivery method, and region). The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper the market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast period

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. In 2018, the non-viral vectors segment accounted for the largest share of this market. However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast period

Geographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Key Topics Covered

1 Introduction

2 Research Methodology 2.1 Research Data2.2 Secondary Data2.2.1 Secondary Source2.3 Primary Data2.4 Market Size Estimation2.4.1 Bottom-Up Approach2.4.2 Bottom-Up Approach for Non-Viral Vectors and Viral Vectors Market2.4.3 Growth Forecast2.5 Market Breakdown and Data Triangulation2.6 Assumptions for the Study

3 Executive Summary

4 Premium Insights 4.1 Gene Therapy Market Overview4.2 North America: Market, By Vector (2018)4.3 Geographical Snapshot of the Market

5 Market Overview 5.1 Introduction5.2 Market Dynamics5.2.1 Drivers5.2.1.1 High Incidence of Cancer and Other Target Diseases5.2.1.2 Product Approvals5.2.1.3 Funding for Gene Therapy Research5.2.2 Opportunities5.2.2.1 Strong Product Pipeline5.2.3 Challenges5.2.3.1 High Cost of Treatments

6 Gene Therapy Market, By Vector 6.1 Introduction6.2 Non-Viral Vectors6.2.1 Oligonucleotides6.2.1.1 North America Accounted for the Largest Share of the Oligonucleotides Segment6.2.2 Other Non-Viral Vectors6.3 Viral Vectors6.3.1 Retroviral Vectors6.3.1.1 Gamma-Retroviral Vectors6.3.1.1.1 The Availability of A Wide Range of Gamma-Retroviral Vectors Supports the Growth of This Market6.3.1.2 Lentiviral Vectors6.3.1.2.1 North America Accounted for the Largest Share of the Lentiviral Vectors Segment6.3.2 Adeno-Associated Virus Vectors6.3.2.1 Possible Applications in In Vivo Applications Have Driven Interest in Adeno-Associated Virus Vectors6.3.3 Other Viral Vectors

7 Gene Therapy Market, By Indication 7.1 Introduction7.2 Neurological Diseases7.2.1 Neurological Diseases Account for the Largest Share of the Market7.3 Cancer7.3.1 Cancer is Expected to Show the Highest Growth in This Market7.4 Hepatological Diseases7.4.1 Increasing Prevalence of Hepatitis B Infections Will Support Market Growth7.5 Duchenne Muscular Dystrophy7.5.1 North America Accounted for the Largest Share of the Dmd Gene Therapy Segment7.6 Other Indications

8 Gene Therapy Market, By Delivery Method 8.1 Introduction8.2 In Vivo Gene Therapy8.3 Ex Vivo Gene Therapy

9 Gene Therapy Market, By Region 9.1 Introduction9.2 North America9.2.1 US9.2.1.1 The US Dominates the Global Market9.2.2 Canada9.2.2.1 Growing Burden of Cancer Will Support Market Growth in Canada9.3 Europe9.3.1 Germany9.3.1.1 Germany Accounted for the Largest Share of the Market in Europe9.3.2 France9.3.2.1 Increasing Cancer Incidence Supports Market Growth9.3.3 UK9.3.3.1 Rising Incidence of Melanoma Will Drive Demand in the UK9.3.4 Italy9.3.4.1 High Incidence of Targeted Diseases and Increasing Per Capita Healthcare Spending Will Drive Market Growth in Italy9.3.5 Spain9.3.5.1 Non-Viral Vectors Dominate the Spanish Market, By Vector9.3.6 Rest of Europe9.4 Asia Pacific9.4.1 Japan9.4.1.1 Japan Dominates the APAC Market for Gene Therapy9.4.2 China9.4.2.1 Rising Prevalence of Cancer and Established Base for Gene Therapy are Supportive Factors in China9.4.3 Rest of APAC9.5 Rest of the World

10 Competitive Landscape 10.1 Overview10.2 Market Share Analysis, 201810.3 Key Strategies10.4 Competitive Leadership Mapping (2018)10.4.1 Visionary Leaders10.4.2 Innovators10.4.3 Dynamic Differentiators10.4.4 Emerging Companies

11 Company Profiles 11.1 Biogen11.2 Gilead Sciences, Inc.11.3 Amgen, Inc.11.4 Novartis AG11.5 Orchard Therapeutics PLC11.6 Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)11.7 Molmed S.p.A.11.8 Anges, Inc.11.9 Bluebird Bio, Inc.11.10 Human Stem Cells Institute (HSCI)11.11 SIBIONO Genetech Co. Ltd.11.12 Shanghai Sunway Biotech Co. Ltd.11.13 Uniqure N.V.11.14 Gensight Biologics S.A.11.15 Celgene Corporation (A Bristol-Myers Squibb Company)11.16 Cellectis11.17 Sangamo Therapeutics11.18 Mustang Bio11.19 AGTC (Applied Genetic Technologies Corporation)11.20 Poseida Therapeutics, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/h6pehn

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View original content:http://www.prnewswire.com/news-releases/global-gene-therapy-industry-outlook-2020-2024---strong-product-pipeline-gives-rise-to-lucrative-growth-opportunities-300996293.html

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Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community

Dr. Jennifer Kwon, a pediatric neurologist at UW Health, tests Piper Droessler's strength. Born to Caiti and Ben Droessler (background), the Platteville baby was the first child in Wisconsin identified through newborn screening to have spinal muscular atrophy, a potentially fatal muscle-wasting disease. She was also the state's first such baby to receive a gene therapy thought to be a cure.

Like their first two children, Caiti and Ben Droesslers daughter Piper, born in late November, looked healthy, with plump cheeks and tufts of reddish hair.

But a week later, the Droesslers learned Piper, unlike her siblings, had spinal muscular atrophy, a rare muscle-wasting disease. In its most severe form, SMA takes away the ability to walk, talk, swallow and breathe, often becoming fatal by age 2.

Wisconsin began testing newborns for the genetic disorder in October. Piper was the first to test positive, which led to a second milestone: In December, she became the states first child identified through screening to get a new gene therapy for SMA, before developing any sign of weakness.

By counteracting the condition before it can take hold, the treatment is thought to be a cure and should allow her to live a normal life.

The fact that we were able to catch it before theres any loss of function is just amazing, said Caiti Droessler, 32, of Platteville.

Caiti and Ben Droessler, of Platteville, had never heard of spinal muscular atrophy, a potentially fatal muscle-wasting disease, until they learned a week after the Nov. 25 birth of their daughter, Piper, that she tested positive for it. She received a gene therapy that is expected to prevent the condition.

Pipers strength and tone and recoil are all what I would expect, Dr. Jennifer Kwon, a UW Health pediatric neurologist overseeing Pipers care, said in January while examining her during a checkup. The expectation is that she will continue to function normally.

The gene therapy, an intravenous infusion called Zolgensma, was approved by the Food and Drug Administration in May. At $2.1 million, it is believed to be the drug markets most expensive one-time treatment.

Concerns about the cost grew in August, when the FDA said manufacturer Novartis manipulated data involving tests on mice and waited until after approval to report the problem. The FDA said it was investigating, but the findings do not change the agencys positive assessment of the drugs human trials.

The first SMA drug, Spinraza, approved in 2016, costs $750,000 the first year and $375,000 each subsequent year. It is taken for life.

Zolgensmas $2.1 million price is no more ludicrous than paying more than $1,000 a month to rent a portable ventilator, especially when that one treatment will likely eliminate the need for the ventilator and many other costs associated with SMA care, Nathan Yates, a financial consultant who has SMA, wrote for the biomedical news website STAT.

At UW Health, about 40 SMA patients have been treated with Spinraza and 11 have received Zolgensma, including three during clinical trials and three in a special access program prior to approval, Kwon said.

UW Hospital staff draw some of Piper's blood. She was later weaned off steroids after receiving a new gene therapy to prevent SMA.

UW requires insurance approval before giving the drugs. Some insurers initially refused but eventually agreed to pay, Kwon said.

About 1 in 11,000 babies is born with SMA, or fewer than 400 a year nationwide. If both parents are carriers, as the Droesslers learned they are, theres a 25% chance each child will have it.

The condition is caused by a mutation in a gene that normally makes a protein needed by nerve cells that control muscles. Without the protein, the nerve cells die and people lose the ability to move.

Newborn screening

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Spinraza helps a related gene make more of the protein. Zolgensma replaces the mutated gene with a normal one, restoring regular protein production.

Any weakness developed before Zolgensma is given isnt reversed, but the drug is expected to prevent additional problems, Kwon said. If given shortly after birth, before any symptoms develop, the treatment is considered a cure, she said.

However, there is no proof the gene therapy will be long-lasting; the oldest children to receive the drug are 5 years old and doing well. We have the theoretical expectation that they will have normal motor function for their lifetime, Kwon said.

The availability of the treatments has led 17 states to add SMA to their newborn screening programs, according to Cure SMA. Another 15 states have approved doing so.

Wisconsin added SMA to its program Oct. 15. Through blood collected from heel pricks before babies leave the hospital, the state now tests for 47 conditions, many of which can be offset with special diets or medications.

Without family history, newborn screening is the only way we can find (SMA) before symptoms appear, said Dr. Mei Baker, co-director of the newborn screening lab at UW-Madisons Wisconsin State Laboratory of Hygiene.

Four to six new cases of SMA are expected in Wisconsin each year, Baker said.

So far, Piper Droessler remains the only baby to test positive through screening.

Caiti and Ben Droesslers 6-year-old daughter and 3-year-old son dont have SMA, and the couple had never heard of the condition. Caiti works at Southwest Health in Platteville, in human resources. Ben runs a masonry restoration business with his dad and brother.

A week after Piper was born Nov. 25, they got a call that she had SMA.

It was very scary, one of those phone calls you never want to get, Caiti Droessler said.

The next day, they met with Kwon in Madison and decided to try Zolgensma as quickly as possible. Their health insurance, Quartz, approved, and Piper received the hour-long infusion at UnityPoint Health-Meriter on Dec. 18.

The couple is thankful for their fortunate circumstances. If Piper had come six weeks earlier or in a state without SMA screening they would not have known about the disorder in advance.

By the time we found out, she would have already presented with weakness, and at that point, the damage is done, Caiti Droessler said. I feel very blessed that we were given this opportunity to keep her well.

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Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com

Abstract / Synopsis:

Research is finding key patient benefits to gene therapy as a promising treatment strategy for Leber's hereditary optic neuropathy (LHON).

This article was reviewed by Patrick Yu-Wai-Man, FRCOphth, FRCPath, BMedSci, MBBS, PhD

Data from two clinical studies of Lebers hereditary optic neuropathy (LHON) showed substantial visual improvements in patients with both disease durations of less than six months and between six months and one year. The improvements resulted from a unilateral injection of a gene therapy vector (GS010) and remarkably, the viral vector seemed to be carried over to the untreated eye.

The mechanism of action for these unexpected results need to be clarified with further experimental work.

Related: Research targets precision dosing for gene, cell therapy

LHON is the most common cause of mitochondrial blindness with a minimal prevalence of one in 30,000 individuals in the population. It causes blindness mostly in young adult men with a peak age of onset in the third decade of life. It is invariably a bilateral disorder in which the fellow eye becomes affected within three to six months after disease onset in the first eye.

Both eyes are affected simultaneously in about 25% of patients, according to Patrick Yu-Wai-Man, FRCOphth, FRCPath, BMedSci, MBBS, PhD, an academic neuro-ophthalmologist with faculty positions at the University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, in the UK.

Three primary mutations within the mitochondrial genome cause about 90% of cases worldwide, namely, m.3460G>A, m.11778G>A and m.14484T>C, with m.11778G>A being the most common mutation by far, accounting for over 70% of those affected with LHON. Unfortunately, most affected patients remain legally blind with vision worse than 1.3 logarithm of the minimum angle of resolution (logMAR) or 3/60 in Snellen equivalent.

Given the poor prognosis, there is an urgent clinical need to identify effective treatments for this blinding optic nerve disease.

Related: LHON gene therapy: Deciphering phase III data

TreatmentGene therapy is obviously a very attractive treatment option, because the underlying pathophysiology is due to insufficient amount of the wild-type protein, Dr. Yu-Wai-Man said. Therefore, if the defective gene is replaced, we should be able to rescue the retinal ganglion cells, preserving function and improving the visual prognosis.

He described the principles of allotopic gene expression that involves inserting the mitochondrial gene of interest, in this case MTND4, into the nuclear genome with a modified viral vector. The wild-type protein produced has a specific mitochondrial targeting sequence that directs it to be imported into the mitochondrial compartment.

The use of an intravitreal injection is a big advantage for this treatment approach as it is a relatively straightforward procedure that provides direct access to the inner retina. Previous preclinical work indicates that allotopic expression is able to rescue the retinal ganglion cells from the deleterious effects of the m.11778G>A mutation.

Related: Gene therapy offering hope for retinal, corneal patients

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Studies target unilateral gene therapy injection - Ophthalmology Times

Passage Bio Jim Wilsons self-described legacy company has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

He joins as the biotech preps IND filings for three lead programs in rare, monogenic diseases of the central nervous system in 2020 the lysosomal storage disorders GM1 gangliasidosis and Krabbe disease, as well as frontotemporal dementia.

Bruce is ideally suited to lead Passage Bio as chief executive officer given his strong neuroscience background coupled with his robust healthcare and biotechnology industry experience, board chairman Tachi Yamada said in a statement.

Passage launched last February with $115 million from a marquee group of Series A investors including Frazier (where Yamada is a partner), OrbiMed, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures. With an office just a 10-minute walk away from Wilsons lab at the University of Pennsylvania, the company was designed to apply the gene therapy pioneers 35-year experience into cross-correctional therapies for CNS.

According to what he calls the Jim Wilson 90/10 rule, Squinto previously told Endpoints News, AAV vectors can cover and transduce 90% of motor neuron cells but only 10% to 15% of other brain cells making it difficult to go after indications where broad transduction is needed. But it can still prove useful in disorders that result from mutations in enzymes that can be taken up by neighboring cells once secreted normally.

A close pact with Penns Gene Therapy Program and Orphan Disease Center gave Passage rights to five programs right out of the gate, with options to license seven more.

Its a very aggressive clinical development strategy across a multitude of programs, Squinto said as he closed a $110 million Series B in September. Were not gonna rely on any one program to drive the value of Passage, were gonna rely on what is a very very full pipeline of opportunities.

Goldsmith will now lead a team of about 25 to build on preclinical and IND-enabling data from Wilsons lab a company growing exercise he honed as COO of Lycera. There, he was also credited for a number of business development initiatives.

The transition into the clinic would also mean moving production from early facilities at Penn to Paragons GMP sites, and eventually to a customized suite slated for completion in the third quarter of this year.

Squinto, a rare disease expert who devoted much of his career to Alexion, will continue to help oversee all of that as a board director.

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Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News

SAN DIEGO, Jan. 28, 2020 (GLOBE NEWSWIRE) -- Otonomy, Inc.(OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical results from the companys gene therapy collaboration with Applied Genetic Technologies Corporation (AGTC) focused initially on treating GJB2 deficiency for congenital hearing loss, and preclinical results demonstrating the therapeutic potential of a class of compounds being evaluated for otoprotection against cisplatin-induced hearing loss (CIHL). These results were presented during the ongoing Association for Research in Otolaryngology (ARO) 43rd Annual MidWinter Meeting being held in San Jose, California.

Together with our strategic partner, AGTC, we are encouraged by these initial preclinical results that demonstrate our ability to express a gene of interest in the target cells relevant to the treatment of congenital hearing loss due to GJB2 deficiency," said David A. Weber, Ph.D., president and chief executive officer of Otonomy. Also, the preclinical results presented for our OTO-510 program highlight the therapeutic potential of a novel class of cisplatin-binding molecules for protection against CIHL and the higher potency of these agents versus other molecules currently in clinical development.

Preclinical Results for GJB2 Gene Therapy Collaboration

In October 2019, Otonomy and AGTC announced a strategic collaboration to co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by a mutation in the gap junction protein beta 2 gene (GJB2) -- the most common cause of congenital hearing loss. The joint presentation by Otonomy and AGTC at ARO provided initial demonstration that a gene of interest can be expressed in support cells of the cochlea, which are the relevant target cells for treating GJB2 deficiency, using novel and proprietary AAV capsids. Furthermore, these studies identified several capsids with favorable tropism and gene expression level in support cells compared to previously reported capsids used in the field. Importantly, none of the novel AAV capsids evaluated for further development exhibited signs of cellular toxicity.

Preclinical Results for OTO-510 Otoprotection Program

Cisplatin is a potent chemotherapeutic agent that is widely used to treat a variety of cancers in adults and children. Unfortunately, the administration of cisplatin is commonly associated with severe adverse effects including CIHL that is progressive, bilateral and irreversible. At ARO, Otonomy presented preclinical results demonstrating varying degrees of otoprotection against CIHL for several different classes of therapeutic agents. In particular, a novel proprietary class of agents that potently bind to cisplatin demonstrated greater otoprotection than anti-oxidant and anti-apoptotic molecules, and increased potency relative to other cisplatin-binding molecules currently in clinical development. These results highlight the therapeutic potential of Otonomys novel otoprotectant agents as the basis for the OTO-510 program for CIHL.

About Otonomy

Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs including Mnires disease, hearing loss, and tinnitus. For additional information please visit http://www.otonomy.com.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or the future financial or operating performance of Otonomy. Forward-looking statements in this press release include, but are not limited to expectations regarding the potential benefits, development activity and advancement of preclinical programs; the potential benefits of and activity under the collaboration agreement between AGTC and Otonomy, including but not limited to development activity; and statements by Otonomys president and CEO. Otonomys expectations regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: Otonomys limited operating history and its expectation that it will incur significant losses for the foreseeable future; Otonomys ability to accurately forecast financial results; Otonomys ability to obtain additional financing; Otonomys dependence on the regulatory success and advancement of its product candidates; the uncertainties inherent in the clinical drug development process, including, without limitation, Otonomys ability to adequately demonstrate the safety and efficacy of its product candidates, the nonclinical and clinical results for its product candidates, which may not support further development, and challenges related to patient enrollment in clinical trials; Otonomys ability to obtain regulatory approval for its product candidates; the risks of the occurrence of any event, change or other circumstance that could give rise to the termination of the collaboration agreement between AGTC and Otonomy; the risks of the occurrence of any event, change or other circumstance that could impact Otonomys ability to repay or comply with the terms of the loan provided by Oxford Finance LLC; side effects or adverse events associated with Otonomys product candidates; Otonomys ability to successfully commercialize its product candidates, if approved; competition in the biopharmaceutical industry; Otonomys dependence on third parties to conduct nonclinical studies and clinical trials; Otonomys dependence on third parties for the manufacture of its product candidates; Otonomys dependence on a small number of suppliers for raw materials; Otonomys ability to protect its intellectual property related to its product candidates in the United States and throughout the world; expectations regarding potential therapy benefits, market size, opportunity and growth; Otonomys ability to manage operating expenses; implementation of Otonomys business model and strategic plans for its business, products and technology; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in Otonomys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 5, 2019, and Otonomys future reports to be filed with the SEC. The forward-looking statements in this press release are based on information available to Otonomy as of the date hereof. Otonomy disclaims any obligation to update any forward-looking statements, except as required by law.

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Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance

With so much money and so many promises in biotech, somethings got to go bust. And in 2019, a lot did.

Although last year saw the second-most new drug approvals since 1997, it also saw more biotechs file for bankruptcy than in any year since 2011: 14, if you include disgraced giant Purdue Pharma. And thats not including all the reverse mergers, which led to the disappearance of a host of failed biotechs: Proteon, NewLink, Conatus and Vical, among many others.

Bankruptcies in biotech are generally rare. Rarer still in the absence of a recession and with the industry still awash in cash. Like every year, a couple of biotechs simply ran out of money as their main asset fizzled. But other failures can be read as a stress-test for the industrys blind spots and vulnerabilities in a year where federal investigators pursued opioid manufacturers and biotech execs joined public health experts in raising alarms about the state of antibiotics research. Those failures include:

The biotechs had collectively raised at least $2 billion since 2010, according to data from Deal Formas Chris Dokomajilar. Well go through each.

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The FDA unveils a new regulatory framework to speed along gene therapies, rewarding the leading players - Endpoints News

After years of ethical debates and breakthroughs in the lab, CRISPR has finally made its way to clinical trials. Researchers are now looking at whether the DNA-editing tool, as well as more conventional gene therapies, can effectively treat a wide array of heritable disorders and even cancers.

Theres been a convergence of the science getting better, the manufacturing getting much better, and money being available for these kinds of studies, says Cynthia Dunbar, a senior investigator at the National Heart, Lung, and Blood Institute. Its truly come of age.

CRISPR formally known as CRISPR-Cas9 has been touted as an improvement over conventional gene therapy because of its potential precision. CRISPR (clustered regularly interspaced short palindromic repeats) is a genetic code that, contained in a strand of RNA and paired with the enzyme Cas9, acts like molecular scissors that can target and snip out specific genes. Add a template for a healthy gene, and CRISPRs cut can allow the cell to replace a defective gene with a healthy one.

In April, scientists at the University of Pennsylvania announced they had begun using CRISPR for cancer treatments. The first two patients one with multiple myeloma, the other with sarcoma had cells from their immune systems removed. Researchers used CRISPR to genetically edit the cells in the lab, and then returned them back into their bodies.

On the other side of the country, Mark Walters, a blood and bone marrow transplant specialist at the University of California, San Francisco, Benioff Childrens Hospital in Oakland, is gearing up for trials that will use CRISPR to repair the defective gene that causes sickle cell disease. With CRISPR, once youve made that type of correction, [that cell] is 100 percent healthy, says Walters.

Another team is tackling the same disease using a type of hemoglobin, a protein in red blood cells, thats normally made only in fetuses and newborn babies. Researchers found that some adults continue to produce these proteins throughout their lives, and when those adults also have sickle cell disease, their symptoms are mild. So the international team used CRISPR to disable the gene that interferes with production of this hemoglobin, resuming its production and protecting the adult patients against sickle cell disease.

Several other CRISPR studies are in the works to treat a range of inherited disorders, including hemophilia and SCID-X1 (also known as X-linked severe combined immunodeficiency, the so-called bubble boy disease in which babies are born without a functioning immune system).

At St. Jude Childrens Research Hospital, a gene therapy trial cured Gael Jesus Pino Alva (pictured with his mother, Giannina) of SCID-X1, the bubble boy disease. (Credit: St. Jude Children's Research Hospital/Peter Barta)

The past year also saw success in a handful of experiments on conventional gene therapy. Instead of using CRISPR to repair disease-causing genes, these treatments use hollowed-out viruses to ferry healthy versions of genes into cells. Millions of these altered cells are released into the bloodstream or bone marrow in hopes that enough will land in the right places. But because scientists cant predict where the circulating genes may end up, this shotgun approach has had unintended, sometimes fatal, consequences including, in an earlier study, inadvertently activating leukemia-causing genes in patients treated for SCID-X1.

But in 2019, researchers learned that using a different type of virus one related to HIV to transport the genes may prevent these side effects. In an April study, researchers at St. Jude Childrens Research Hospital in Memphis, Tennessee, and UCSF Benioff Childrens Hospital in Oakland collected bone marrow from eight newborns with SCID-X1. They loaded corrective genes into the disabled HIV-related virus, which carried them into the patients bone marrow stem cells. The infants also received low doses of busulfan, a chemotherapy that gave the doctored stem cells room to grow. So far, we havent seen anything worrisome, says Ewelina Mamcarz, a pediatric oncologist at St. Jude who led the research team. The study recently added its 12th patient.

Gene therapy does have its momentum [back], says Mamcarz, reflecting on the fields setback after the earlier studys leukemia side effects. Theres so much that still needs to be done, and so many questions, she says. [But] this is how medicine evolves. We always want to be better than we were a week ago.

In the future, the hope is that gene therapy technologies will move beyond mending simple genetic mistakes and be used to combat big killers like diabetes or heart disease. [Those diseases are] more challenging, but a lot of them would benefit from knocking out a bad gene, says Dunbar.

For now, though, researchers are optimistic about the progress thats already been made. All of this has been very encouraging, says Dunbar. [And] for sickle cell in the U.S. and hemophilia in the developed world, these diseases may soon be solved.

[This story originally appeared in print as "Gene Therapy Gets Clinical."]

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Gene Therapies Make it to Clinical Trials - Discover Magazine

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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2019: The year gene therapy came of age - INQUIRER.net

Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrigs disease, is a neurodegenerative disease affecting nerve cells in the brain and spinal cord. Researchers at the University of California San Diego School of Medicine published research describing a new way to deliver a gene-silencing vector to mice with ALS. The therapy resulted in long-term suppression of the disease if the treatment was given before the disease started. It also blocked disease progression in the mice if symptoms already appeared.

The study was published in the journal Nature Medicine.

At present, this therapeutic approach provides the most potent therapy ever demonstrated in mouse models of mutated SOD1 gene-linked ALS, said senior author Martin Marsala, professor in the Department of Anesthesiology at UC San Diego School of Medicine. In addition, effective spinal cord delivery of AAV9 vector in adult animals suggests that the use of this new delivery method will likely be effective in treatment of other hereditary forms of ALS or other spinal neurodegenerative disorders that require spinal parenchymal delivery of therapeutic gene(s) or mutated-gene silencing machinery, such as in C9orf72 gene mutation-linked ALS or in some forms of lysosomal storage disease.

ALS appears in two forms, sporadic and familial. The most common form is sporadic, responsible for 90 to 95% of all cases. Familial ALS makes up 5 to 10% of all cases in the U.S., and as the name suggests, is inherited. Studies have shown that a least 200 mutations of the SOD1 gene are linked to ALS.

In healthy individuals, the SOD1 gene provides instructions for an enzyme called superoxide dismutase. This enzyme is used to break down superoxide radicals, which are toxic oxygen molecules that are a byproduct of normal cellular processes. It is believed that the mutations in the gene cause ineffective removal of superoxide radicals or potentially cause other toxicities resulting in motor neuron cell death.

The new research involves injecting shRNA, an artificial RNA molecule that can turn off, or silence, a targeted gene. This delivers shRNA to cells by way of a harmless adeno-associated virus (AAV). In the research, they injected the viruses carrying shRNA into two locations in the spinal cord of adult mice expressing an ALS-causing mutation of the SOD1 gene. They were performed just before disease onset or after the laboratory animals started showing symptoms.

The researchers have tested the approach in adult pigs, whose have spinal cord dimensions closer to those in humans. They found that by using an injector developed for adult humans, the procedure could be performed without surgical complications and in a reliable fashion.

The next step will be more safety studies with a large animal model.

While no detectable side effects related to treatment were seen in mice more than one year after treatment, the definition of safety in large animal specimens more similar to humans is a critical step in advancing this treatment approach toward clinical testing, Marsala said.

About 5,000 people are diagnosed with ALS in the U.S. each year, with about 30,000 people living with the disease. There are symptomatic treatments, but no cure. Most patients die from the disease two to five years after diagnosis.

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Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace

Home News Drugs in the Pipeline

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec.

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A, said Hank Fuchs, MD, President, Global Research and Development at BioMarin. Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.

For more information visit biomarin.com.

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BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference

Maximum Life Foundation ("MaxLife"), is rapidly transforming the way we treat aging diseases. MaxLife plans to use a promising gene therapy offered by Integrated Health Systems to give free access to ten (10) early to mid-stage Alzheimer's Disease (AD) patients. David Kekich, MaxLife's CEO, stated "MaxLife will grant 100% of the therapy costs to help bring pioneering gene therapy to cure this disease and make Alzheimer's Disease a thing of the past."

NEWPORT BEACH, Calif. (PRWEB) December 30, 2019

Cure Now Instead of Palliative Care

According to the Alzheimer's Association:

Alzheimer's costs Americans $277 billion a year and rising. Sharp increases in Alzheimer's disease cases, deaths and costs are stressing the U.S. healthcare system and caregivers. About 5.7 million Americans have Alzheimer's disease. To date, no one has survived it.

Improvements of AD symptoms and the recovery of normal brain functions have been demonstrated in-vivo in mouse experiments, and in-vitro in human cell experiments through the rejuvenation of microglia (the brain's first line of defense against infection) and neurons as well as stimulating mitochondrial function using the telomerase reverse transcriptase (TERT) protein.

One human patient received a lower dose therapy in August 2018 with no adverse side effects. To date, the patient's disease has not progressed. MaxLife hopes to see symptom reversals in the next patients.

"If we can prove a benefit to patients that have no other option now, we can potentially treat Alzheimer's Disease in people in early to mid-stage Alzheimer's, finally creating effective medicine at the cellular level," states Kekich. "If successful, this treatment could potentially be used on other diseases such as Parkinson's and ALS."

The unique difference is developing treatments against the cellular degeneration caused by aging as the root cause of most major diseases. Studies have proven aging is the leading risk factor for many life-threatening diseases, including Alzheimer's.

With a world class Scientific Advisory Board, MaxLife is ready to push forward into practical solutions. A gene therapy facilitator, Integrated Health Systems plans to treat other adult aging-related diseases with no previous cure such as Sarcopenia, Atherosclerosis, Chronic Kidney Disease (CKD) and even aging itself with gene therapies.

"This technology could halt many of the big age associated killers in industrialized countries'" states Kekich. "Compassionate care helps patients with no other option to get access to experimental therapies that may benefit both themselves and society as a whole."

MaxLife also seeks grants and donations for human gene therapy studies for atherosclerosis, sarcopenia and chronic kidney disease as well as for human aging. The protocols have already been developed. Please Click Here and scroll to the bottom of the page to see how to donate.

To apply for a free therapy or for more information, see http://www.maxlife.org/alzheimers-disease/ and https://maxlife.org/how-to-register-and-qualify-for-the-alzheimers-human-study/.

For Further Information, Contact: David Kekich, CEO Maximum Life Foundation.

Maximum Life Foundation is a 501(c)(3) Not-For-Profit corporation founded in 1999.

Tax I.D. #31-1656405. David A. Kekich Tel. #949-706-2468. Info@MaxLife.org

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First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy. "I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency." Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells. The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors." The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured. "This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting." In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden. For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs. The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself. "It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy. Scientists practising the technique insert a normal gene into cells containing a defective gene. It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient. Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union. They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness. Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution. "Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster." Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period." "We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies. They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor. However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine. Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans. The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process. "That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations. "It's very easy to do if you don't care about the consequences," Musunuru added. Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability. There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US. The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online. "Not everyone is a biologist or scientist," she said. And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops? Charpentier thinks that technology generally tends to be used for the better. "I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18

For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, there's no getting around the fact that it's been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360% over the last 10 years to the S&P 500's 190%. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, it's worth highlighting how far we've come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivizes the search for new discoveries. Here are some of the decades biggest medical breakthroughs:

Cell therapies: First approved in the U.S. two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumors, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumors. Scientists wouldn't be looking so far into the future without this decades extraordinary progress.

Gene therapies: Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get U.S. approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalizing potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution: The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy: Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the body's defense mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the U.S. alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumors is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C: From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programs and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen at mnisen@bloomberg.net

@2019Bloomberg

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Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard

Like any reporting beat, biopharma has its slow news days. But its rare to find a whole year when few events of note occur, and 2019 was no exception. Indeed, the last year of the 21st centurys second decade saw its fair share of major mergers and acquisitions, controversies and historical milestones across the medical biotechnology and pharmaceutical industries.

The year kicked off with a mega-merger, when on Jan. 3, New York-based Bristol-Myers Squibb said it would acquire Summit, New Jersey-based Celgene for $74 billion. A wave of mergers and acquisitions across the biopharma industry soon followed. This was a decade after another wave of biopharma consolidation, when 2009 kicked off with Pfizers acquisition of Wyeth, followed by Merck & Co. buying Schering-Plough and Roche buying Genentech. The next large-scale deal happened in June, when Chicago-based AbbVie said it would spend $63 billion to acquire Allergan.

Other than the two aforementioned large deals, however, most M&A activity this year has taken the form of large biotechnology and pharmaceutical companies buying much smaller, but still large-cap players. Earlier this month, Roche concluded its $4.3 billion acquisition of gene therapy maker Spark Therapeutics, originally announced in February. That deal was dogged by a 10-month investigation by U.S. and U.K. regulators into the question of whether the Roche might have a disincentive to develop Sparks Phase III gene therapy candidate for hemophilia A, given that the Swiss drugmaker already makes a drug for the disease. Indianapolis-based Eli Lilly & Co.s $8 billion acquisition of Loxo Oncology days after the BMS-Celgene deal did not face such hurdles, though Loxo handed off its one approved product, the cancer drug Vitrakvi (larotrectinib), to development partner Bayer. Numerous other deals have followed, including Novartis deal to acquire The Medicines Co. for $9.7 billion, Astellas announcement that it would buy Audentes Therapeutics for $3 billion, Mercks move to buy ArQule for $2.7 billion, among others.

While dwarfed by its counterpart in 2009, the current M&A wave has not been without controversy. In September, a group of eight Democratic senators and one independent several of whom are or had been running for president wrote to the Federal Trade Commission urging greater scrutiny over such deals amid concerns about competition and high drug prices. While its uncertain if the FTC was responding to their concerns, the agency said Dec. 17 that it would seek to block genomic sequencing company Illumina from buying a smaller firm, Pacific Biosciences of California, in a previously announced $1.2 billion deal.

Another controversy that arose in 2019 was on the regulatory front. In August, the Food and Drug Administration said it was looking into a disclosure from Novartis subsidiary AveXis that mouse data from a disused assay used in its application for the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) in spinal muscular atrophy which the FDA approved in May had been manipulated. The scandal, which predated Novartis acquisition of AveXis in 2018, led to two of the gene therapy developers executives being fired and created a publicity crisis for the drugmaker.

Yet, while the Zolgensma scandal did not affect the FDAs overall position on the product itself, another FDA decision still raised eyebrows. The approval of Karyopharm Therapeutics Xpovio (selinexor) in highly refractory multiple myeloma patients attracted significant criticism from physicians, who pointed both to what some called a low response rate in the Phase IIa study on which the approval was based and the FDA using Phase III data that were not disclosed to the public. The approval also happened despite the FDAs Oncologic Drugs Advisory Committee voting not to recommend it.

2019 also saw some important milestones on the regulatory front. Nine months after Scott Gottliebs resignation as commissioner of the FDA, the Senate confirmed Dr. Stephen Hahn, a radiation oncologist and chief medical executive at The University of Texas MD Anderson Cancer Center, to take his place. And the agency approved the first ever drug to tackle the root cause of sickle cell disease and its first vaccine against the Ebola virus.

In addition to specific events, many trends that have garnered increasing public and political attention moved to front and center in 2019 as well. The national conversation about drug pricing showed no signs of quieting down, as the Trump administration rolled out various programs designed to tackle high drug prices, and the House fired a shot across the bow with a bill this month that would require the Centers for Medicare and Medicaid Services to negotiate prices for some drugs.

Many of these events will continue to play out in 2020, as a new FDA commissioner takes the helm of the agency and large companies see their acquisitions bear fruit, for good or ill. And the conversation about drug pricing will only get louder amid the 2020 presidential election, which in turn could lead to greater scrutiny over biopharma industry consolidation.

Photo: klenger, Getty Images

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What were the biggest biopharma stories of 2019? - MedCity News