Category Archives: Gene Medicine

In 2013, a mysterious new flu virus hit the world. It was the H7N9 strain of bird flu, and by the time it infected 120 and killed 23, researchers still didnt know how it was transmitted or how to stop it. As the virus arrived in the dense Chinese province of Hunan, health officials began to fear the worst.

In the U.S., they called Dan Gibson. Gibson was Vice President of DNA Technology atSynthetic Genomics, the research institute founded in 2005 by genomics pioneers Craig Venter, Ph.D., and Nobel Laureate Hamilton Smith, M.D., shortly after the completion of the Human Genome Project. Among its research milestones, the institute created the first synthetic cell, designed in a computer and DNA-printed completely from scratch.

Gibson was given a download link to the DNA sequence of the bird flu virus and told to do one thing: design a vaccine.

Gibson is the namesake of the Gibson assembly method, the worlds leading branded method of joining DNA fragments together that he pioneered just four years before. The method lets you join DNA fragments together in a single step, and it proved to have a huge multiplying effect on the entire field ofsynthetic biology. So who better to ask for a DNA solution to the bird flu?

Dan Gibson is a pioneer in synthetic biology who can design vaccines against coronavirus and other pathogens in about an hour. Now, labs can put his methods to work on their own benchtops. Codex

In truth, Gibson had been preparing for this moment for years. His team was already collaborating with Novartis on a grant from the Biomedical Advanced Research and Development Authority (BARDA) on pandemic preparedness. The goal of that project was to create a vaccine against the H1N1 virus. BARDA designed some ambitious, forward-thinking exercises where project members were given an H1N1-type pandemic scenario, a DNA sequence, and a one-week deadline to manufacture a vaccine.

Even in 2013, Gibsons team had shown they could design a vaccine candidate within about 24 hours once given the virus sequence. But they knew that many of the things they were doing in the laboratory could be automated, which would enable others to pursue alternative vaccine candidates in parallel. They had been building a prototype machine that integrated all of their learnings and processes into a single box when H7N9 hit.

This is not a fire drill anymore, Gibson recalls thinking to himself. This is the real deal.

Gibsons team and its prototype system successfully developed an H7N9 vaccine, which Novartis made and the US government stockpiled. Fortunately, the virus did not become a pandemic. But the goal of putting their labs genius into a single box became the origin of a new company and a new product.

The genetic evolution of H7N9 bird virus in China, 2013. Like the novel coronavirus, the H7N9 bird virus probably originated in so-called wet markets. U.S. Centers for Disease Control

Codex DNA(formerly SGI DNA) was launched to make this technology available to researchers everywhere. Codex says that its flagship machine the BioXp 3200 system is the worlds first commercially available, fully automated gene synthesis platform. It synthesizes ready-to-use custom DNA overnight.

The same machine can be used to make DNA to create diagnostic tools, design and develop vaccines, or synthesize the cells to make those vaccines.

The instrument can make up to 32 genes at a time, and it can incorporate downstream processes like assembling the genes into the customers favorite host organism. It can also be used with newer cell-free methods of amplifying DNA to create micrograms of material.

Part of the special sauce in the BioXp system is its error correction process. The Codex team puts specific enzymes in the reagent mix that recognize and remove any unwanted mutations from the population. This allows Codex to synthesize extremely high-fidelity genes, which is critical to applications involving human therapies.

Its not only a research tool, but also a biomanufacturing tool across a wide range of applications. The system streamlines clinical research and allows for distributed manufacturing of personalized therapies, Gibson told me. Its the kind of tool thats needed in hospitals everywhere to not only make brand-new drugs locally, but also to realize the dream of individualized treatments for cancer, rare diseases, and other maladies that humans suffer.

In September, Codex closed a $25 Million Series A financing round to support the commercial launch of the BioXp system, and the company is now focused on making the system accessible to anyone in the research community who has a need for on-demand DNA synthesis across a variety of fields, including vaccine development, antibody drug discovery, personalized medicine, and beyond.

In particular, Gibson and his team are on a mission to get this platform to any researcher working on coronavirus to help in the effort to develop and make therapies and vaccines. There are now hundreds of BioXp systems around the world, with customers now doing in their lab with the BioXp system what Gibson alone was doing back in 2013.

In addition, Codex can provide researchers mostanything they might need to fight the SARS-CoV-2 virus, including positiveSynthetic RNA controlsfor tests, thefull synthetic genomefor vaccine and treatment development, and custom-order antigen panels and antibody librariesall of it made on the BioXp system.

The challenge with coronavirus, says Gibson, who is now the CTO of Codex, is that theres no defined process yet for how you go from your vaccine candidate to a final vaccine. Codexs customers and partners arent immune to that challenge, but the BioXp system is drastically speeding up the process of developing vaccine candidates.

Gibson says of one of his customers: He had ordered reagents from us and six days later he was injecting a vaccine candidate into mice.

All of this is light-years ahead of the current seasonal flu vaccine process, where you typically wait to receive a mucus sample in the mail, isolate the virus, inject it into chicken eggs, and then let them incubate. Thewhole vaccine selection and production processcan take six months, by which time the virus has mutated and your vaccine may not work that well.

Whats so amazing about synthetic biology is that if you have the DNA sequence, you can just download it and start synthesizing it right away, he says.

In a world where infections can travel as fast and far as a jet plane, well need biological defenses that can travel at the speed of the internet.

Follow me on twitter at@johncumbersand@synbiobeta. Subscribe to my weekly newsletters insynthetic biologyThank you toStephanie Michelsenfor additional research and reporting in this article. Im the founder ofSynBioBeta, and some of the companies that I write aboutincluding Codex DNAare sponsors of theSynBioBeta conferenceandweekly digestheres the full list of SynBioBeta sponsors.

Originally published on Forbes: https://www.forbes.com/sites/johncumbers/2020/05/14/a-coronavirus-vaccine-in-six-days/

Originally posted here:
A Coronavirus Vaccine Candidate In Six Days? This Company Is Fast-Tracking Vaccine Development With The First Fully Automated Gene Synthesis Platform...

INDIANAPOLIS (AP) A dime-size nanochip developed by a world-renowned researcher who recently relocated to Indianapolis could help transform the practice of medicine. It could also turn Indianapolis into a manufacturing and research hub for radically new disease and trauma treatment techniques.

It all began in August 2018, when Chandan Sen, one of the worlds leading experts in the nascent field of regenerative medicine, moved his lab from Ohio State University to the Indiana University School of Medicine. He brought along a team of about 30 researchers and $10 million in research grants, and now serves, among a myriad of other positions, as director of the newly formed Indiana Center for Regenerative Medicine and Engineering, to which IU pledged $20 million over its first five years.

IU recruited Sen away from Ohio State in part because of its desire not just to promote academic research in his field but also to help develop practical, commercial products and uses for his breakthroughs.

A scientist prefers to be in the lab and keep on making more discoveries, said Sen, 53.

But I thought that, unless we participate in the workforce development process and the commercialization process, I dont think that the businesspeople would be ready to do it all by themselves. Because its such a nascent field.

Its definitely new and its potential sounds like the stuff of science fiction.

Regenerative medicine, as its name hints, seeks to develop methods for replacing or reinvigorating damaged human organs, cells and tissues.

For instance, instead of giving a diabetic a lifetimes worth of insulin injections, some of his skin cells could be altered to produce insulin, curing him. Such techniques might also be used for everything from creating lab-grown replacement organs to, someday, regenerating severed limbs.

Regenerative medicine offers a form of medicine that is neither a pill nor a device, Sen said.

It is a completely new platform, where you dont necessarily depend on any given drug, but are instead modifying bodily functions.

Sen and his teams signal contribution to the field is a technique theyve dubbed tissue nanotransfection, or TNT. Put simply, it uses a nanotechnology-based chip infused with a special biological cargo that, when applied to the skin and given a brief electrical charge, can convert run-of-the-mill skin cells into other cell types. Potentially, the technique could be used for everything from regrowing blood vessels in burn-damaged tissue to creating insulin-secreting cells that could cure diabetics.

Obviously, such applications are still down the road a ways. But the technology is far enough along that some products are already making it to market and investors, entrepreneurs and established companies are sniffing around for opportunities. According to the Alliance for Regenerative Medicine, more than 1,000 clinical trials worldwide are using regenerative medicine technologies.

Thousands of patients are already benefiting from early commercial products, and we expect that number will grow exponentially over the next few years, said Janet Lambert, the alliances CEO.

Lambert predicts that the number of approved gene therapies will double in the next one to two years. Last year, the U.S. Food and Drug Administration predicted it would be approving 10 to 20 cell and gene therapies each year by 2025.

These new techniques could do more than just revolutionize medicine. They could also upend the medical industry as we know it. And the IU School of Medicine and Indianapolis could lead the way.

There are really only two or three places in the country that did the kind of comprehensive work that Dr. Sens group was doing, said Anantha Shekhar, executive associate dean for research at IU School of Medicine. And they were doing it from the lab all the way to the clinic, where they were already applying those technologies in patients.

So it was very attractive to think of starting with a bang bringing a comprehensive group here and creating a new center.

Instead of merely treating chronic conditions, regenerative medicine could end them, once and for all.

For instance, consider a car with an oil leak. The traditional medical approach might be to live with the chronic condition by pouring in a fresh quart of oil every few days. The regenerative medicine approach would fix the leak. Its good for the car, good for the cars owner but not necessarily good for the guy who was selling all those quarts of oil.

Which is why these new techniques, if they catch on, could cause turmoil in the medical industry.

Because regenerative medicine has the potential to durably treat the underlying cause of disease, rather than merely ameliorating the symptoms, this technology has the potential of being extremely disruptive to the current practice of medicine, Lambert said.

This has the potential to be hugely disruptive, Sen added, because so much of medicine today relies on huge industrial infrastructures to manage, not cure, chronic diseases and disabilities.

If such disruption comes to pass, the leaders of 16 Tech, a 50-acre innovation district northwest of downtown that aspires to house dozens of medical-related startups and established firms, would love to be its epicenter.

The Center for Regenerative Medicine will be one of the tenants of 16 Techs first building, a $30 million, 120,000-square-foot research and office building scheduled to open in June.

Regenerative medicine is probably one of the next major waves of medical innovation in the world, 16 Tech CEO Bob Coy said. To have him here doing this work gives Indianapolis and Indiana an opportunity to develop an industrial cluster in regenerative medicine.

Coy believes the most momentous early step on that road was the recent establishment by Sen of masters and doctoral programs in regenerative medicine at the IU School of Medicine. Its the first degree of its type in the country, earning IU and Indianapolis the enviable status of first mover.

I think, for example, of (Pittsburghs) Carnegie Mellon University, which, back in the late 1960s, created the first college of computer science in the country, Coy said. And now you know Carnegie Mellons reputation in computer science.

What isnt in place yet is a state or city program to promote development of a regenerative medicine hub.

We need to start doing that, Coy said. That means putting a lot of the infrastructure in place to support startups that are based on this technology, as well as recruiting companies that want to collaborate with Dr. Sen.

In spite of the lack of a coherent recruitment program, Coys phone has started to ring, thanks largely to Sens presence.

There have been a few meetings Ive had with people who already have relationships with him, who, when they come to town, have reached out to meet and talk about what were doing at 16 Tech, he said.

One of the first 16 Tech startups with designs on the regenerative medicine niche is Sexton Biotechnologies.

The company was groomed by Cook Regentec, a division of Bloomington-based Cook Group charged with incubating and accelerating technologies for regenerative medicine and the related field of cell gene therapy.

Any products that show promise are either folded into the company, turned into their own divisions or, as in Sextons case, spun off as an independent entity with Cook retaining a financial stake.

Its a measure of the newness of this field that Sextons 17 employees arent working on new medicines, but rather marketing basic tools needed to conduct research. The companys offerings include a vial for storing cell and gene products in liquid nitrogen, and a cell culture growth medium.

Theres a ready market for such tailor-made gear, because, for years, researchers in the regenerative medicine field had to make do with jury-rigged equipment.

What most of those companies did was repurpose things like tools from the blood banking industry, or tools from bio pharma, said Sean Werner, Sextons president.

So thats why a lot of newer companies are starting to build tools explicitly for the industry, as opposed to everybody just having to cobble together stuff that was already out there.

Werner said investors recognize the momentous opportunity in regenerative medicine and are flocking to the field.

Its not something you have to explain, he said. Companies and VC groups are trying to get a piece of it.

What has investors and medical researchers charged up is the almost unlimited range of potential applications, from healing burns to, perhaps someday, regenerating limbs.

I think it would be a huge revolution if were able to, for example, regenerate insulin-secreting cells in children who have become juvenile diabetics or have for whatever reason lost their pancreas, Shekhar said. Those are the kinds of things that will start to change the way we see certain diseases.

Lambert predicted that, as the science advances, so will the business case.

While early programs focused primarily on rare genetic diseases and blood cancers, were already seeing the field expand into more common age-related neurological disorders, such as Parkinsons and Alzheimers, she said.

I expect this trend to continue in the coming years, greatly increasing the number of patients poised to benefit from these therapies.

Werner said regenerative medicine also is seeking advancements in manufacturing technologies that will lower the cost of product development.

It all adds up to a huge opportunity the state is well-positioned to seize, Werner believes.

Indiana is a perfect place for this kind of thing to really ramp up, he said. Theres no reason we cant lead the field.

__

Source: Indianapolis Business Journal

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IU team pursues breathtaking regenerative medicine advances - The Advocate

BIRMINGHAM, Ala. - Is personalized medicine cost-effective? University of Alabama at Birmingham researcher Nita Limdi, Pharm.D., Ph.D., and colleagues across the United States have answered that question for one medical treatment.

Patients experiencing a heart attack -- known as a myocardial infarction or an acute coronary syndrome -- have sharply diminished blood flow in coronary arteries, with a high risk of heart failure or death. Coronary angioplasty, a procedure to open narrowed or blocked arteries in the heart, and percutaneous coronary intervention, known as PCI or stenting, can restore blood flow to minimize damage to the heart. These procedures reduce the risk of subsequent major adverse cardiovascular events, or MACE, which include heart attacks, strokes or death.

But then, a treatment decision has to be made.

After stenting, all patients are treated with two antiplatelet agents for up to one year. Which combination of antiplatelets is best? The answer comes through pharmacogenomics, says Limdi, a professor in the UAB Department of Neurology and associate director of UAB's Hugh Kaul Precision Medicine Institute.

Pharmacogenomics combines pharmacology, the study of drug action, with genetics, the study of gene function, to choose the best medication according to each patient's personal genetic makeup. This is also called precision medicine -- tailored medical treatment for each individual patient.

The most commonly used antiplatelet combination after PCI is aspirin and clopidogrel, which is trademarked as Plavix. Clopidogrel is converted to its active form by an enzyme called CYP2C19. However, patients respond to clopidogrel differently based on their genetic makeup.

More than 30 percent of people have loss-of-function variants in the CYP2C19 gene that decrease the effectiveness of clopidogrel. The FDA warns that these patients may not get the full benefit of clopidogrel, which would increase the risk of MACE. So the FDA advises doctors to consider a different treatment such as prasugrel or ticagrelor, trademarked as Effient and Brillinta, to replace clopidogrel.

While most patients undergoing PCI receive clopidogrel without receiving any CYP2C19 loss-of-function testing, academic institutions like UAB that offer precision medicine use pharmacogenomics to guide the selection of medication dosing.

In 2018, Limdi and other investigators across nine United States universities -- all members of the Implementing Genomics in Practice consortium, or IGNITE -- showed that patients with loss-of-function variants who were treated with clopidogrel had elevated risks. There was a twofold increase in MACE risk for PCI patients, and a threefold increase in MACE risk among patients with acute coronary syndrome who received PCI, as compared to patients prescribed with prasugrel or ticagrelor instead of clopidogrel. Prasugrel and ticagrelor are not influenced by the loss-of-function variant and can substitute for clopidogrel, but they are much more costly and bring a higher risk of bleeding.

The IGNITE group then leveraged this real-world data to conduct an economic analysis to determine the best drug treatment for these heart disease patients.

A study led by Limdi and colleagues, published in the Pharmacogenomics Journal, examines the cost-effectiveness of genotype-guided antiplatelet therapy for acute coronary syndrome patients with PCI. This cost-effectiveness study is the first to use real-world clinical data; many cost-effectiveness studies use clinical trial data, which tends to exclude the sicker patients normally seen in clinical practice.

The study compared three main strategies: 1) treating all patients with clopidogrel, 2) treating all patients with ticagrelor, or 3) genotyping all patients and using ticagrelor in those with loss-of-function variants.

"We showed that tailoring antiplatelet selection based on genotype is a cost-effective strategy," Limdi said. "Support is now growing to change the clinical guidelines, which currently do not recommend genotyping in all cases. Evidence like this is needed to advance the field of precision medicine."

Costs, QALYs and ICERs

In the analysis, Limdi and colleagues considered differences in event rates for heart attacks and stent thrombosis in patients receiving clopidogrel versus ticagrelor versus genotype-guided therapy, during the one-year period following stenting. They also included medical costs from those events that are borne by the payer, such as admissions, procedures, medications, clinical visits and genetic testing. The analysis considered variations in event rates and medication costs over time to ensure that the results held under different scenarios.

The study uses an economic measure -- the QALY, which stands for the quality-adjusted life year.

"First, we looked at which strategy provided the highest QALY," Limdi said. "The QALY is the gold standard for measuring benefit of an intervention -- in our case, genotype-guided treatment compared to treatment without genotyping. Universal ticagrelor and genotype-guided antiplatelet therapy had higher QALYs than universal clopidogrel -- so those are the best for the patient."

But health care resources are not infinite. So, Limdi and colleagues then evaluated whether those interventions that have higher QALYs were also reasonable from a cost perspective. This analysis considered the willingness to pay. What would a payor or a patient pay for the highest QALY?

"In our case, the payor would recognize that ticagrelor is more expensive than clopidogrel -- $360 per month vs. $10 per month -- and there is a $100 cost for each genetic test," Limdi said. "So, from the payor perspective, the more effective strategy (one with a higher QALY) -- if more expensive (higher cost) -- would have to lower the risks of bad outcomes like heart attacks and strokes for the gains in QALY that are at, or below, the willingness-to-pay threshold."

A calculation called incremental cost-effectiveness ratios, or ICERs, assesses the incremental cost of the benefit (improvement in QALY). In the United States, a treatment is considered cost-effective if its associated ICER is at or below the willingness-to-pay threshold of $100,000 per QALY.

"In our assessment, the two strategies with the highest QALY had very different ICERs," Limdi said. "The genotype-guided strategy was cost-effective at $42,365 per QALY. Universal ticagrelor was not; it had an ICER of $227,044 per QALY."

The researchers also looked at some secondary strategies for a real-world reason. A number of clinicians now prescribe ticagrelor or prasugrel for the first 30 days after PCI, which is considered a period of greater risk, and then switch their patients to the less expensive drug clopidogrel.

The secondary analysis allowed Limdi and colleagues to explore the cost-effectiveness of giving all patients ticagrelor for 30 days, and then switching them to clopidogrel, without genetic testing, versus switching the patients based on genotype. Both strategies were better -- in terms of QALYs -- than a universal switch to clopidogrel at 30 days. However, neither of the two appeared to be cost-effective. Because these secondary strategies used estimated parameters, "the findings should only be considered as hypothesis-generating," Limdi said.

Read more:
Precision medicine guides choice of better drug therapy in severe heart disease - Science Codex

Sarepta Therapeutics (NASDAQ:SRPT)'s stock had its "buy" rating reaffirmed by investment analysts at Robert W. Baird in a report released on Friday, TipRanks reports. They currently have a $192.00 target price on the biotechnology company's stock. Robert W. Baird's target price suggests a potential upside of 35.93% from the company's previous close.

Other analysts also recently issued research reports about the stock. BidaskClub raised shares of Sarepta Therapeutics from a "hold" rating to a "buy" rating in a research report on Thursday, May 7th. Nomura Securities reaffirmed a "buy" rating and set a $230.00 price target on shares of Sarepta Therapeutics in a report on Tuesday, February 25th. Cantor Fitzgerald reiterated an "overweight" rating and issued a $217.00 price objective (up previously from $211.00) on shares of Sarepta Therapeutics in a report on Thursday, February 27th. Oppenheimer reissued a "hold" rating on shares of Sarepta Therapeutics in a research report on Thursday, May 7th. Finally, SVB Leerink reaffirmed a "buy" rating and set a $216.00 target price on shares of Sarepta Therapeutics in a research report on Thursday, January 23rd. One investment analyst has rated the stock with a sell rating, one has given a hold rating and twenty-four have given a buy rating to the stock. The company has an average rating of "Buy" and a consensus target price of $193.05.

Shares of NASDAQ SRPT opened at $141.25 on Friday. The stock has a fifty day moving average price of $112.04 and a 200 day moving average price of $112.85. The company has a market cap of $9.91 billion, a P/E ratio of -16.00 and a beta of 1.87. Sarepta Therapeutics has a 12-month low of $72.05 and a 12-month high of $158.80. The company has a current ratio of 8.31, a quick ratio of 7.75 and a debt-to-equity ratio of 0.66.

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Sarepta Therapeutics (NASDAQ:SRPT) last issued its earnings results on Wednesday, May 6th. The biotechnology company reported ($0.23) earnings per share for the quarter, topping the Thomson Reuters' consensus estimate of ($2.17) by $1.94. The company had revenue of $113.67 million during the quarter, compared to the consensus estimate of $118.18 million. Sarepta Therapeutics had a negative return on equity of 64.67% and a negative net margin of 160.96%. The company's revenue was up 30.6% compared to the same quarter last year. During the same quarter in the prior year, the business earned ($1.07) earnings per share. On average, sell-side analysts forecast that Sarepta Therapeutics will post -8.44 EPS for the current fiscal year.

In other news, Director Richard Barry sold 30,000 shares of the company's stock in a transaction that occurred on Friday, May 15th. The stock was sold at an average price of $141.26, for a total transaction of $4,237,800.00. Following the completion of the transaction, the director now directly owns 3,163,813 shares of the company's stock, valued at $446,920,224.38. The transaction was disclosed in a filing with the Securities & Exchange Commission, which can be accessed through the SEC website. Also, Director Hans Lennart Rudolf Wigzell sold 5,000 shares of the stock in a transaction that occurred on Wednesday, March 4th. The stock was sold at an average price of $116.89, for a total value of $584,450.00. Following the sale, the director now owns 18,792 shares in the company, valued at $2,196,596.88. The disclosure for this sale can be found here. 6.60% of the stock is currently owned by corporate insiders.

A number of institutional investors and hedge funds have recently modified their holdings of SRPT. Amundi Pioneer Asset Management Inc. increased its position in Sarepta Therapeutics by 32.8% in the first quarter. Amundi Pioneer Asset Management Inc. now owns 154,611 shares of the biotechnology company's stock worth $18,428,000 after buying an additional 38,194 shares during the period. DNB Asset Management AS acquired a new position in shares of Sarepta Therapeutics during the fourth quarter valued at $1,386,000. Zeke Capital Advisors LLC grew its position in shares of Sarepta Therapeutics by 10.1% during the fourth quarter. Zeke Capital Advisors LLC now owns 5,343 shares of the biotechnology company's stock worth $689,000 after acquiring an additional 491 shares during the last quarter. Assenagon Asset Management S.A. acquired a new stake in shares of Sarepta Therapeutics in the 4th quarter worth about $860,000. Finally, Janney Montgomery Scott LLC lifted its position in Sarepta Therapeutics by 7.7% in the 4th quarter. Janney Montgomery Scott LLC now owns 5,312 shares of the biotechnology company's stock valued at $685,000 after purchasing an additional 382 shares during the last quarter. Hedge funds and other institutional investors own 93.76% of the company's stock.

Sarepta Therapeutics Company Profile

Sarepta Therapeutics, Inc focuses on the discovery and development of RNA-based therapeutics, gene therapy, and other genetic medicine approaches for the treatment of rare diseases. The company offers EXONDYS 51, a disease-modifying therapy for duchenne muscular dystrophy (DMD). Its products pipeline include Golodirsen, a product candidate that binds to exon 53 of dystrophin pre-mRNA, which results in exclusion or skipping of exon during mRNA processing in patients with genetic mutations; and Casimersen, a product candidate that uses phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene.

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10 Buy and Hold Stocks to Add to Your Portfolio

Set it and forget it are words many investors dont want to hear. Even the most venerable brokerage houses are encouraging their clients to actively trade so they can beat the market. Buy and hold is a relic, they say. It doesnt reflect the reality of today.

In other words, this time its different.

As the ongoing volatility in the market shows you, its not different. Its not even close to being different. The simple fact is that many active traders lose money by being too aggressive and too active for their own good.

And while its true that the market wont always be this choppy, and certain stocks may be a great buy in months to come, right now investors are looking for safe harbors. One of the safest ways to invest is to find stocks that you can feel comfortable holding on to even in the worst of times. Frequently that can be because the stocks offer an attractive dividend. But sometimes, its also because they are in a market that is always in demand.

But that doesnt mean you have to limit yourself to defensive stocks. You can find some quality buy-and-hold stocks that offer some attractive growth prospects.

View the "10 Buy and Hold Stocks to Add to Your Portfolio".

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Sarepta Therapeutics' (SRPT) "Buy" Rating Reiterated at Robert W. Baird - MarketBeat

There has been a rise in government funding and research programs which is paving the way for the growth of the gene editing technologies in diagnostic platforms market. For example, the National Institutes of Health (NIH) has allocated funding on the study of clustered regularly interspaced short palindromic repeats (CRISPR) from 2011 to 2018. The NIH spent about US$ 3,083.4 million between the fiscal year 2011 and 2018 on a total of 6,685 projects. The funding has been increased by 213.1% between the fiscal year 2014 and 2015.

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Moreover, with the help of NIH Common Funds support, National Institutes of Health (NIH) launched Somatic Cell Genome Editing (SCGE) program on January 2018 which is working to improve the effectiveness and specificity of gene editing techniques to assist in the diminishing of the burden of common and erratic diseases caused by genetic variations. The program aims at developing quality tools to execute and determine effective and harmless genome editing in somatic cells of the body. These tools will be made extensively available to the research community to lessen the time and expense which is required to develop new therapies. Furthermore, Somatic Cell Genome Editing program will award approximately US$ 190 million to biomedical researchers over the six years starting from 2018 till 2023. Hence, these types of research programs and funding given to the researchers will help the diagnostic platforms to get the tools which will aid them in carrying out gene editing and will drive the future market of the gene editing technologies in diagnostic platforms.

The number of CRISPR related publications, as listed in the SCOPUS database of peer-reviewed research, shows the surge in funding. Between 2015 and 2016, the number of such publications raised 117.5% which is 1,457. In 2019, the number surpassed 3,900 and increased at a rate of 4.8%. Overall, 12,900 papers associated with the technique have been published since 2011, Thus, this increasing research is expected to assist in the gene editing technologies in diagnostic platforms market growth over the forecast period.

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The detailed research study provides qualitative and quantitative analysis of gene editing technologies in diagnostic platforms market. The market has been analyzed from demand as well as supply side. The demand side analysis covers market revenue across regions and further across all the major countries. The supply side analysis covers the major market players and their regional and global presence and strategies. The geographical analysis done emphasizes on each of the major countries across North America, Europe, Asia Pacific, Middle East & Africa, and Latin America.

Key Findings of the Report:

In terms of revenue, the gene editing technologies in diagnostic platforms market is expected to reach US$ 7,004.8 Mn by 2027, expanding at 14.4% CAGR during the forecast period due to the rising government funding for genome editing and engineering

Beam Therapeutics, Bio-Connect Group, CRISPR Therapeutics, Editas Medicine, GeneCopoeia, Inc., GenScript, Horizon Discovery Ltd., Inscripta, Inc., Integrated DNA Technologies, Inc., Intellia Therapeutics, Inc., Lonza Group Ltd., Merck KGaA, New England Biolabs, OriGene Technologies, Inc., Pairwise, Precision Biosciences, Sangamo Therapeutics, STEMCELL Technologies Inc., Thermo Fisher Scientific Inc., Transposagen Biopharmaceuticals, Inc. are the key market participants operating in the gene editing technologies in diagnostic platforms market.

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Gene Editing Technologies in Diagnostic Platforms Market:

By Technology

CRISPR

TALEN

ZFN

Others

By Application

Cell Line Engineering

Genetic Engineering

Animal Genetic Engineering

Plant Genetic Engineering

Others

By End-User

Biotechnology & Pharmaceutical Companies

Academic and Research Institutions

Contract Research Organization (CROs)

By Geography

North America

U.S.

Canada

Mexico

Rest of North America

Europe

France

The UK

Spain

Germany

Italy

Nordic Countries

Denmark

Finland

Iceland

Sweden

Norway

Benelux Union

Belgium

The Netherlands

Luxembourg

Rest of Europe

Asia Pacific

China

Japan

India

New Zealand

Australia

South Korea

Southeast Asia

Indonesia

Thailand

Malaysia

Singapore

Rest of Southeast Asia

Rest of Asia Pacific

Middle East and Africa

Saudi Arabia

UAE

Egypt

Kuwait

South Africa

Rest of Middle East & Africa

Latin America

Brazil

Argentina

Rest of Latin America

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Gene Editing Technologies in Diagnostic Platforms Market is expected to grow at a CAGR of 14.4% during the forecast period due to the rise in research...

BOSTON--(BUSINESS WIRE)--Akouos, a precision genetic medicine company developing gene therapies to potentially restore, improve and preserve hearing, announced today that data from its inner ear gene therapy platform will be presented during the 23rd American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be held virtually May 12-15, 2020.

Two poster presentations will highlight Akouoss use of AAVAnc80 vector technology and its potential to address many forms of hearing loss. Presentation details are as follows:

Title:

Use of the Adeno-Associated Viral Anc80 (AAVAnc80) Vector for the Development of Precision Genetic Medicines to Address Hearing Loss

Date and Time:

Tuesday, May 12, 2020 5:30 PM - 6:30 PM (EST)

Title:

Enabling Temporal Control of Gene Expression in the Inner Ear after AAVAnc80 Vector Mediated Delivery

Date and Time:

Wednesday, May 13, 2020 5:30 PM - 6:30 PM (EST)

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, the Company was founded in 2016 by world leaders in the fields of neurotology, genetics, inner ear drug delivery and AAV gene therapy. Akouos has strategic partnerships with Massachusetts Eye and Ear and Lonza, Inc. For more information, please visit http://www.akouos.com.

About AAVAnc Technology

The Ancestral AAV (AAVAnc) platform was developed in the laboratory of Luk Vandenberghe, Ph.D., director of the Grousbeck Gene Therapy Center at Harvard Medical School. AAVAnc technology uses computational and evolutionary methods to predict novel conformations of the adeno-associated viral particle. AAVAnc80, one of approximately 38,000 AAVAnc vectors, has demonstrated preliminary safety and effective gene delivery in both mice and non-human primates in preclinical studies.

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Akouos to Present Data from Inner Ear Gene Therapy Platform at 23rd ASGCT Annual Meeting - Business Wire

-- Agreement leverages Sareptas leadership in gene therapy for neuromuscular and cardiovascular diseases and Dynos CapsidMap artificial intelligence platform to design AAV vectors --

CAMBRIDGE, Mass., May 11, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Dyno Therapeutics, Inc., a biotech company applying artificial intelligence (AI) to gene therapy, today announced an agreement to develop next-generation Adeno-Associated Virus (AAV) vectors for muscle diseases, using Dynos CapsidMap platform.

AI and machine learning technologies have the potential to deliver enhanced vectors for gene therapies. Dynos proprietary CapsidMap platform opens up new ways to identify novel capsids the cell-targeting protein shell of viral vectors that could offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing.

Sareptas world-leading gene therapy engine is founded on three pillars: developing a broad portfolio of programs to treat rare diseases; our first-in-class manufacturing expertise; and investment in advancing and further improving the science of gene therapy to help patients in need of more options. To that end, our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases, said Doug Ingram, Sareptas President and Chief Executive Officer. By leveraging Dynos AI platform and Sareptas deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.

Under the terms of the agreement, Dyno will be responsible for the design and discovery of novel AAV capsids with improved functional properties for gene therapy and Sarepta will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. If successful, Dyno could receive over $40 million in upfront, option and license payments during the research phase of the collaboration. Additionally, if Sarepta develops and commercializes multiple candidates for multiple muscle diseases, Dyno will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.

This agreement is a major step forward in our plan to realize the potential of Dynos AI platform for gene therapies to improve patient health. We are excited to work with Sarepta to create gene therapies with improved properties to address a range of muscle-related diseases, stated Dynos CEO and co-founder Eric D. Kelsic, Ph.D. The success of the gene therapies developed through this collaboration with Sarepta will rely on AI-powered vectors that allow gene therapies to be safely and precisely targeted to the muscle tissue.

About CapsidMap for Designing AAV Gene Therapies By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV)vectors, Dynos proprietary CapsidMap platform overcomes the limitations of todays gene therapies on the market and in development. Todays treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next-generation DNA sequencing to measure invivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dynos massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

Dynos technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS), a Core Faculty member at Harvards Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. Several of the technical breakthroughs that enabled Dynos approach to optimize synthetic AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by Dyno founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology.

About Dyno TherapeuticsDyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The companys proprietary CapsidMap platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit http://www.dynotx.com for additional information.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Therapeutics Forward-looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the potential of artificial intelligence and machine learning technologies to deliver enhanced vectors for gene therapies; the potential of the CapsidMap platform to offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing; the agreement between Sarepta and Dyno Therapeutics providing a valuable tool to develop next-generation capsids for gene therapies to treat rare disease; the parties goal to advance next-generation treatments with improved muscle-targeting capabilities; the parties responsibilities under the agreement and potential payments to Dyno Therapeutics; and the potential of AI-powered vectors to allow gene therapies to be safely and precisely targeted to the muscle tissue.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Dyno Therapeutics may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates; possible limitations of company financial and other resources; manufacturing limitations that may not be anticipated or resolved for in a timely manner; regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2019 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

ContactsFor Sarepta: Investors: Ian Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com

For Dyno:Kathryn MorrisThe Yates Networkkathryn@theyatenetwork.com914-204-6412

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Sarepta Therapeutics and Dyno Therapeutics Announce Agreement to Develop Next-Generation Gene Therapy Vectors for Muscle Diseases - GlobeNewswire

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Approach may lead to new treatment approach for osteoarthritis, obesity

Researchers at Washington University School of Medicine in St. Louis found that gene therapy in mice helped build strength and significant muscle mass quickly, while reducing the severity of osteoarthritis. The gene therapy also prevented obesity, even when the mice were fed a high-fat diet.

Exercise and physical therapy often are recommended to help people who have arthritis. Both can strengthen muscle a benefit that also can reduce joint pain. But building muscle mass and strength can take many months and be difficult in the face of joint pain from osteoarthritis, particularly for older people who are overweight. A new study in mice at Washington University School of Medicine in St. Louis, however, suggests gene therapy one day may help those patients.

The research shows that gene therapy helped build significant muscle mass quickly and reduced the severity of osteoarthritis in the mice, even though they didnt exercise more. The therapy also staved off obesity, even when the mice ate an extremely high-fat diet.

The study is published online May 8 in the journal Science Advances.

Obesity is the most common risk factor for osteoarthritis, said senior investigator Farshid Guilak, PhD, the Mildred B. Simon Research Professor of Orthopaedic Surgery and director of research at Shriners Hospitals for Children St. Louis. Being overweight can hinder a persons ability to exercise and benefit fully from physical therapy. Weve identified here a way to use gene therapy to build muscle quickly. It had a profound effect in the mice and kept their weight in check, suggesting a similar approach may be effective against arthritis, particularly in cases of morbid obesity.

With the papers first author, Ruhang Tang, PhD, a senior scientist in Guilaks laboratory, Guilak and his research team gave 8-week-old mice a single injection each of a virus carrying a gene called follistatin. The gene works to block the activity of a protein in muscle that keeps muscle growth in check. This enabled the mice to gain significant muscle mass without exercising more than usual.

Even without additional exercise, and while continuing to eat a high-fat diet, the muscle mass of these super mice more than doubled, and their strength nearly doubled, too. The mice also had less cartilage damage related to osteoarthritis, lower numbers of inflammatory cells and proteins in their joints, fewer metabolic problems, and healthier hearts and blood vessels than littermates that did not receive the gene therapy. The mice also were significantly less sensitive to pain.

One worry was that some of the muscle growth prompted by the gene therapy might turn out to be harmful. The heart, for example, is a muscle, and a condition called cardiac hypertrophy, in which the hearts walls thicken, is not a good thing. But in these mice, heart function actually improved, as did cardiovascular health in general.

Longer-term studies will be needed to determine the safety of this type of gene therapy. But, if safe, the strategy could be particularly beneficial for patients with conditions such as muscular dystrophy that make it difficult to build new muscle.

In the meantime, Guilak, who also co-directs the Washington University Center for Regenerative Medicine and is a professor of biomedical engineering and of developmental biology, said more traditional methods of muscle strengthening, such as lifting weights or physical therapy, remain the first line of treatment for patients with osteoarthritis.

Something like this could take years to develop, but were excited about its prospects for reducing joint damage related to osteoarthritis, as well as possibly being useful in extreme cases of obesity, he said.

Tang R, Harasymowicz NS, Wu CL, Collins KH, Choi YR, Oswald SJ, Guilak F. Gene therapy for follistatin mitigates systemic metabolic inflammation and post-traumatic arthritis in high-fat diet-induced obesity. Science Advances, published online May 8, 2020.

This work was supported by the Shriners Hospitals for Children, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Institute on Aging and the Office of the Director of the National Institutes of Health (NIH). Grant numbers AR50245, AR48852, AG15768, AR48182, AG 46927, AR073752, OD10707, AR060719, AR057235. Additional funding was provided by the Arthritis Foundation and the Nancy Taylor Foundation for Chronic Diseases.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Gene therapy in mice builds muscle, reduces fat Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA Congress, which will take place virtually from June 11-14, 2020.

An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA, including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial.

The accepted abstract is now available on the EHA conference website: https://ehaweb.org/congress/eha25/key-information-2/.

Abstract Title: Initial Safety and Efficacy Results With a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Hematopoietic Stem and Progenitor Cells in Transfusion-Dependent -Thalassemia and Sickle Cell DiseaseSession Title: Immunotherapy - ClinicalAbstract Code: S280

About the Phase 1/2 Study in Transfusion-Dependent Beta ThalassemiaThe ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About the Phase 1/2 Study in Sickle Cell DiseaseThe ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is expected to be presented at the European Hematology Associations upcoming congress; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com/ or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, information regarding the data that is expected to be presented at the European Hematology Association (EHA)s upcoming Congress. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with theSecurities and Exchange Commissionand available through the company's website atwww.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Oral Presentation at the 25th European...

The price for a gene therapy drug to treat an intractable disease in toddlerswas set at about 167 million yen ($1.56 million) per patient, the countrys most expensive medicine covered by national health insurance.

The application for Zolgensma was approved at the Central Social Insurance Medical Council, an advisory council for the health minister, on May 13 and will go into effect on May 20.

The drug is for children under the age of 2 with spinal muscular atrophy,in which the poor functioning of motor nerves causes muscle weakness.

Zolgensma was developed to replace the function of a certain gene that is not working properly with the injection of a normal gene.

Patients can receive a single infusion of Zolgensma.

The price of the drug was determined based on that for Spinraza, an existing drug. While Spinraza costs about 9.5 million yen per dosage and needs to be injected repeatedly for a certain period of time, Zolgensma is believed to have a long-lasting benefit through a single injection.

The price of Zolgensma was first set at about 100 million yen, the estimated cost of using Spinraza for several years, and was then increased to about 167 million yen by taking into account the high therapeutic effects and other benefits of Zolgensma.

The amount that users of Zolgensma need to pay will be much lower than the price since they can use the government-sponsored reimbursement system for high-cost medical care, which caps the amount of out-of-pocket medical costs. In addition, many municipalities subsidize all the out-of-pocket expenses if the patients are children.

Only about 25 patients are expected to use Zolgensma annually, so the high cost of the drug will likely have only a limited impact on the nation's overall medical expenses.

Kymriah, which was approved for treatment of leukemia in Japan last year, is priced at 33.49 million yen, the highest price in the country at the time. As technology for developing new drugs advances, more drugs with expensive price tags are expected to be developed in the future. That could put a heavy burden on health insurance associations.

Each association may need to bear a huge financial burden, said a member of the Central Social Insurance Medical Council.

Ataru Igarashi, an associate professor of pharmacoeconomics at Yokohama City University School of Medicine, said, Manufacturers and the authorities are required to fulfill their responsibilities more than ever for explaining whether the value of a drug is truly worth its price.

(This article was written by Tamura Kenji, a senior staff writer, and Ryuichi Hisanaga.)

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Gene therapy drug for infants priced at 170 million yen : The Asahi Shimbun - Asahi Shimbun