Category Archives: Gene Medicine

Ten years ago, a little-known Science paper authoredby Jennifer Doudna, Ph.D., and Emmanuelle Charpentier, Ph.D., proposedusing CRISPR/Cas9 for gene editing. As the first wave of gene-editing-based therapies post clinical data and head to the FDA, biopharma executives at the forefront of the burgeoning field highlighted the innovations and challenges before gene editing is ready for prime time.

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Now, some of the early efforts of turningCRISPRgene editingtechnologyinto viable therapies are coming to fruition. Vertex and partner CRISPR Therapeutics justannouncedtheir plan to file their CRISPR/Cas9-edited cell therapy exagamglogene autotemcel (exa-cel) for a rolling review at the FDA in sickle cell disease and beta thalassemia starting in November. If approved, exa-cel could be the first CRISPR-based therapy available.

Meanwhile, Intellia Therapeutics, after being thefirst to showthat systemic infusion of CRISPR inside the human body could treat disease, recentlyreportedmore positive early data for its in vivo gene editing candidates for transthyretin amyloidosis (ATTR) and hereditary angioedema.

For now, most gene editing projects are focused on rare diseases and some blood or cancer indications with well-established genetic drivers. Those diseases have clear clinical endpoints and risk-benefit understanding to allow for a quick drug development path.

Read more:
10-year CRISPR anniversary: How gene editing revolutionized medicine, and what lies ahead - Genetic Literacy Project

Risky at-birth surgery saves baby with rare disorder

Doctors have performed a dramatic surgery to save a baby who was born with a life-threatening rare disorder that hampered his ability to breathe. (Sept. 21) (AP Video: Emma H. Tobin)

AP

Every baby born in the United States is pricked in the heel shortly after birth. A blood sample is then analyzed to look for one of 20 to 30 inherited diseases.

Early identification of a particular disease meanstreatment can start right away, potentially saving or extending thechild's life.

Now, doctors want to go even further: They want to look not just atblood, but atgenes.

A new effort announced Wednesday by a genetic testing company paired withresearchers at NewYork-Presbyterian/Columbia Universityaims to sequence 100,000 newborns in New York City over the next five years.

The sequencing would look for about 250 diseases that strike before age 5 and for which there are treatments or approaches that can make a difference in a child's life.

A similar effort in the United Kingdom is also examining the genes of 100,000 newborns, looking for diseases for which there is a treatment or a cure.

The programs promiseto bring treatments to babies before symptoms become obvious and at a time when something can be done to help them.

"The appetite for this is growing. The awareness of this is growing. We all see it as inevitable," said Dr. Robert Green, a medical geneticist atBrighamand Women's Hospital and Harvard Medical School, both in Boston."We are grossly underutilizing the life-saving benefits of genetics and we have to get past that."

This week,Green is hosting a conference in Boston, bringing together researchers and industry representatives from the U.S., U.K., European Union and Australia to set standards and discuss the challenges and opportunities presented byscaling upnewborn genetic sequencing.

This kind of early sequencing and treatment is possible now for the first time because of dramatic advances in diagnostics, therapies and digital data storage, as well as a reduction in the cost of sequencing, said Dr. Paul Kruszka, a clinical geneticist and chief medical officer of GeneDx at Sema4, which is leading the new program.

"We're entering the therapeutic era and leaving the diagnostic era," Kruszka said. "This potentially has the opportunity to change the way we practice medicine especially in rare disease."

Right now, families with rare diseases often search for a diagnosis for 5, 10 or even 20 years. If the child could be diagnosed at birth, he said, it would short-circuit that process and treatment could begin much earlier hopefully before the child suffers irreversible damage.

Before deciding whether every family should get access to genetic sequencing for their newborn,large studies like Sema4's are needed to justify the cost, Kruszka said.

The price of gene sequencing has dropped precipitiously, with one company, Illumina. announcing last week that its newest-generation sequencing machinescan run a complete sequence for about $200. Kruszka said Sema4 expects to still payabout $1,000 for each sequence of all 20,000 genes.

Gene sequencing at birth should be able to save money over the child's lifetime by preventing illness, Green said. The costs of sequencing are limited, he said, but the benefits will build up over the child's lifetime and may help family members, too.

Green and his team began analyzing the genetic sequences of newborns in 2013, and has found lots of useful information among the first 320 babies sequenced, he said. He now has funding to expand his sequencing researchto 1,000 newborns.

Large numbers are essential because most of the diseases being diagnosed are extremely rare.

Convincing parents to participate in a sequencing research trial "is not easy," Green said. Many are concerned about privacy and the discrimination their child might face if their genome were made public. And it can be a unpleasant for parents to consider the horrible diseases their perfect newborn might be harboring,he said.

"You've gone through all this pregancy and you're sitting there with a healthy baby (and I'm) offering you the opportunity to find out something that's devastating and terrifying," he said. "How fun is that?"

He doesn't think privacy needs to be a major parental concern. Companies can learn more useful information by tracking someone's cell phone or credit card than their genome and most common diseases are the result of many combinations of genes.

"Many people hear 'genetics' and worry somehow that that is a special kind of privacy threat," he said, adding that he doesn't think there is. "We haven't been paying attention to the medical benefits of genetic testing, particularly predictive genetic testing."

if people don't want to know, that's okay, too, Green said. "We canrespect people who don't want to know, but as also respect people who do want to know," he said. "Some families will say 'I treasure the precious ignorance.' Others will say 'If I could have known, I would have poured my heart and soul into clinical trials or spent more time with the child when she was healthy."

In a five-year review of their research, Green and his colleagues found that "terrible things didn't happen" when they sequenced newborn genomes.

Families, he said, "did not in fact have downstream distress," he said. "They did have appropriate medical follow-up and that there were amazing benefits to the babies and the families as a result of the surveillance and treatment."

The baby sequencing identified several parents who had inherited illnesses and received risk-reducing surgery, he said, as well as a baby who had a narrowed aorta that wouldn't have been detected if its genetics hadn't indicated the need for an echocardiogram.

"Even in a small sample we found much to act on," he said.

At Rady Children's Hospital in San Diego, they're trying to rapidly sequence the genomes of babies who already have problems and are being treated in one of 83 children's hospitals acrossCanada and the U.S.

Every morning, samples arrive by Fedex. In some cases, the baby is in such dire shape than an answer is needed immediately. For those children, "we've got to drop what we're doing and go,"said Dr. Stephen Kingsmore, the president and CEO of Rady's Institute for Genomic Medicine."Even a day can cost a child's life or brain function."

For babies who are stable, sequencing still happens rapidly, but a little less so."Every sample gets onto a sequencer the same day," he said.

So far, the institute, which is also collaborating on a newborn sequencing study in Greece,has been able to provide a 1,500 children with a diagnosis in the first weeks of life in addition to a life-saving treatment.

"That idea, that future is where a child never experiences a sick day, even though they have a fatal condition," the institute's former director of marketing,Graciela Sevilla,said earlier this year. "We'd love to see that on a regular basis."

Contact Weintraub at kweintraub@usatoday.com

Health and patient safety coverage at USA TODAY is made possible in part by a grant from the Masimo Foundation for Ethics, Innovation and Competition in Healthcare. The Masimo Foundation does not provide editorial input.

Air pollution could be contributing to millions of premature births

Estimates in a new study say air pollution could be a factor in up to 3.4 million preterm births.Video provided by Newsy

Newslook

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Blood from a baby at birth can be gene sequenced to prevent diseases - USA TODAY

Its natural for women to worry about breast cancer, especially since many people know someone who was touched by the disease. While there is no foolproof way to prevent breast cancer, there are things you can do to lower your risk. Some factors you cant change, but knowing what can help is key to lowering your risk of breast cancer.

Breast cancer is the second most common cancer among women in the U.S.some types of skin cancer are the most common. Between 1989 and 2020, breast cancer death rates decreased by 43%, but racial and ethnic inequities still exist. While breast cancer incidence is lower among Black women than white women, the death rate is 40% higher among Black women than white women, according to the American Cancer Society. That is because about one in five Black women with breast cancer have triple-negative breast cancermore than any other racial or ethnic group. Meanwhile, Asian American and Pacific Islanders have the lowest death rate from breast cancer while Native Americans and Alaska Natives have the lowest rates of developing breast cancer. And while rare, men can get breast cancer too.

The AMAsWhat Doctors Wish Patients Knew series provides physicians with a platform to share what they want patients to understand about todays health care headlines.

In this installment, Jill Jin, MD, an internist at Northwestern Medicine and clinical assistant professor of medicine at Northwestern University Feinberg School of Medicine, took time to discuss what patients need to know about what to do to reduce their risk of breast cancer. She is also a senior physician adviser for the AMA and an associate editor forJAMA.

Know the risk factors

While risk factors for breast cancer are broad, we think primarily about agearound the age of menopause and after menopause is when breast cancer risk goes up in women, said Dr. Jin. Family history, of course, is another big one. That includes genetic mutations that we know of such as BRCA1 and BRCA2.

Theres also this whole concept of estrogen exposure, which can be both endogenouswithin the body or how much your body producesversus exogenous, from medications she said. Then other things like alcohol and smoking are thought to be associated somewhat with breast cancer as well.

Start screening between 40 and 50

Overall, the recommended age to start screening for breast cancer in average-risk women would be anywhere from 40 to 50 years old, said Dr. Jin. It is important to convey to patients that most professional societies do recommend later than 40, either 45 or 50, as the age to start screening. But most physicians are still starting on the earlier end of this spectrum because it can be a tough sell for patients to say, wait until 50 years old when, to be honest, most people around them are probably getting screened earlier.

Almost everyone knows somebody these days who has had breast cancer, whether its a friend or a family member, and when you have that personal connection its scary, she added. Thats why I usually tell women who are at average riskwho dont have family history of breast cancerthat I am comfortable waiting until 45 years old to start screening.

If they do have a family history of breast cancer or other risk factors, we certainly can and should start screening earlier, Dr. Jin said. Its very individualized at the end of the day.

Different screening tests are available

There are several different screening modalities, said Dr. Jin, noting that a mammogram is the most common one. Other methods of screening include ultrasounds, as well as a breast magnetic resonance imaging.

But for most people, we start with mammograms, she said.

Earlier screening isnt always better

It always begins with getting to know the patient, asking about their history and their lifestyleits definitely an individualized risk assessment first, said Dr. Jin. And if there is nothing that suggests they are at higher risk than average, then you can have a discussion with patients about potentially waiting to start screening.

It comes down to the benefits versus the harms of screening, she added, noting the younger you start screening, the more lives you will save because you will catch more cancers at earlier stages, especially the more aggressive ones.

But on the flip side, the younger people are, the more you pick up things that are not cancer, which is called a false positive finding. Younger women have denser breast tissue, and when breast tissue is dense, it is very hard to differentiate normal tissue from something that may look like cancer, Dr. Jin explained. And then you go down this whole path of follow-up testing which includes additional mammograms and sometimes biopsy, which very often ends up being an unnecessary biopsy because everything will turn out normal.

This causes a lot of anxiety. It upends patients lives for a couple months while this whole process is going on, and that amount of anxiety affects many other parts of patients livesit is not trivial, she added. And then you do it all over again the next year. The younger you start, the more the potential harms of these false positives start to outweigh the potential benefit of earlier diagnosis.

Maintain a healthy lifestyle

For all womenreally for everyoneit is important to maintain a healthy lifestyle, said Dr. Jin. That means eating a balanced diet, not drinking too much alcohol, not smoking, maintaining regular physical activity and a normal body mass index.

All of those things are likely helpful for prevention of not just breast cancer, but other cancers as well, along with cardiovascular diseasea lot of things, she added.

There are medications to reduce risk

Chemoprevention, or the use of medications, is another option to reduce breast-cancer risk, said Dr. Jin. For chemoprevention, there are two classes of medications that are used. One is called selective estrogen receptor modulators, or SERMs.

Tamoxifen is probably the most common one that is used. SERMs medications block the effects of estrogen in the breast, she added. Another class is called aromatase inhibitors. Those are usually used in older women after menopause and stop other hormones in the body from becoming estrogen.

However, both have other side effects. While tamoxifen blocks the effects of estrogen in breast tissue, it can actually enhance estrogen effects in other parts of the body, so we do worry about blood clots as well as uterine cancer, said Dr. Jin. And then aromatase inhibitors can cause other side effects related to low estrogen such as hot flashes, bone pain, decreased bone density, and increased risk of osteoporosis and fractures.

Thats why we dont use these medications in everyone to decrease breast cancer risk, and reserve them for high-risk women only. Again, as with every decision in medicine, we want to make sure the balance of potential benefits versus harms is in favor of benefits, she said.

Surgical prevention is also an option

The other kind of prevention would be surgical prevention, said Dr. Jin. This is also done for women who are high risk, most commonly because of the BRCA gene mutation.

People who have a known BRCA gene mutation, which puts them at an increased risk for both breast and ovarian cancer, are candidates for surgery to remove the breasts. Thats called prophylactic mastectomy, she said. They also may be candidates for surgery to remove the ovaries to decrease the risk of ovarian cancer as well.

Test for the BRCA gene mutation

There are calculators that can be used to calculate whether someone, based on their family history and ethnicity, should get genetic testing for the BRCA gene mutation, which is a blood test said Dr. Jin. If you have a first-degree family membersuch as your mom or siblingwho has breast cancer and is known to have BRCA, then you should get tested for it.

If you just have a family history of breast cancer with unknown BRCA status, thats when the calculators come into play, she added, noting they look at how many first-degree and second-degree relatives, whether you are of Ashkenazi Jewish descent, and certain other risk factors to decide whether you should get the genetic testing.

Breastfeeding may reduce risk

While there are no clinical trials on this topic, there is observational data that does suggest that breastfeeding is protective against breast cancer, said Dr. Jin. The same goes for having children versus not having children; pregnancy does seem to be protective as well.

Were not saying go get pregnant and breastfeed to reduce your risk of having breast cancerits not practical, she added. But it does seem to be an association.

Birth control is OK to take

This is also somewhat controversial, but overall, the link between birth control and breast cancer is very small to none, Dr. Jin said. When I talk with my patients about this, I share that using birth control pills most likely does not increase the risk of breast cancer in a clinically significant way.

Furthermore, this very small potential increase in risk is limited to the time that youre actually taking birth-control pills, she said. So, its not a permanent effect. Its temporary.

Be cautious with self-breast exams

There has not been any good evidence to show that self-screening has any overall benefit in mortality, said Dr. Jin. Breasts are just lumpy to begin with and a lot of people end up feeling lumps that just end up being normal breast tissue.

And you may end up, again, going down that path of all the imaging and the biopsies and in the end, it is nothing, she added. So, self exams are not recommended by clinical guidelines.

However, some people are going to be wanting to do that anyways and that is fine. If someone really wants to stay on top of their body, I will explain that breasts can feel lumpy or bumpy, and what they are looking for is a change from baseline. At the end of the day, you still know your own breasts and your own body the best, said Dr. Jin. So, if you feel something that is different, that you have not felt before, then you should let me know and we can decide at that point what to do,

If they are in the office with me, I am happy to do a quick exam of the breast and tell them this is what your normal breast tissue feels like, dont be alarmed if you feel this or if you feel this. It is just normal.

Dont hesitate to talk to your doctor

While screening is recommended for between 40 and 50 years old, if at any point you do notice something like a lump or you see something weird on the skin or if you have pain or any symptoms that are different than normal, that takes you out of the typical screening category, she emphasized. As with all cancer screening, when a symptom is detected that is different, and you should never hesitate to bring that up to your doctor.

Read the rest here:
What doctors wish patients knew about breast-cancer prevention - American Medical Association

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

Pharmaceutical TechnologyVolume 46, Number 10October 2022Pages: 54-55

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," Pharmaceutical Technology 46 (10) 5455 (2022).

View post:
Growth in Cell and Gene Therapy Market - Pharmaceutical Technology Magazine

The Gene Editing Service Market research report forecast 2022 -2029 provides in-depth information on market trends, market capacity, industry size, growth factors, share, innovations, competitive environment, business problems, and more. This reports historical data confirms demand growth on a global, national, and regional scale. The research of the Gene Editing Service also aids in the understanding of industry prospects and growth chances. This report leverages advanced tools such as SWOT analysis and Porters Five Forces analysis to accurately estimate market and revenue growth. The report also provides an extensive analysis of the impact of the COVID-19 pandemic and how it contributed to market progress.

Market research reports from WMR include a competitive landscape, in-depth vendor selection methodology, and analysis based on qualitative and quantitative research to properly Gene Editing Service Market growth. In this Research Report, by analyzing key aspects such as profit, pricing, competition, and promotions, as well as examining, synthesizing, and summarising data from many sources, the analyst produces a comprehensive picture of the Gene Editing Service market. It shows a variety of market elements by identifying the top industry influencers. The market study further also draws attention to crucial industry factors such as global clients, potential customers, and sellers, which instigates positive company growth. In order to gauge the turning point of the businesses, significant market key players are also enlisted in order to deliver readers an in-depth analysis of industry strategies.

% ( : $ ) :https://www.worldwidemarketreports.com/promobuy/817034

Caribou Biosciences CRISPR Therapeutics Merck KGaA Editas Medicine Thermo Fisher Scientific Horizon Discovery Genscript Biotech GeneCopoeia Integrated DNA Technologies Eurofins Genomics DNA 2.0 (ATUM) BBI Life Sciences Genewiz Gene Oracle SBS Genetech Bio Basic

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Global markets are presented by Gene Editing Service type, along with growthforecasts. Estimates of production and valueare based on the price in the supply chain at which the Gene Editing Service are procured by the manufacturers.

This report has studied every segment and provided the market size using historical data. They have also talked about the growth opportunities that the segment may pose in the future. This study bestows production and revenue data by type, and during the historical period and forecast period.

Ex-Vivo In-Vivo

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This report has provided themarket size (production and revenue data) by application, during the historical period and forecast period.

This report also outlines the market trends of each segment and consumer behaviors impacting the Gene Editing Service market and what implications these may have on the industrys future. This report can help to understand the relevant market and consumer trends that are driving the Gene Editing Service market.

Commercial Academic Research

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The Gene Editing Service Market engineering process uses a top-down and bottom-up approach and several data triangulation methods to evaluate and validate the size of the entire market and other dependent sub-markets listed in this research report. The major players in the market were identified through the second survey and the market rankings were determined through the first and second surveys.

To analyze actual Gene Editing Service market sales and their breakdowns, primary and secondary approaches were used. The Gene Editing Service assessment comprised extensive primary searches, such as surveys, expert opinions, profiles, and secondary ratings to business magazines, industry directories, paid venues, and others. In addition, the industry research examines data acquired from a range of sector analysts and significant market participants along the industrys value chain to provide a succinct quantitative and qualitative analysis.

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North America (U.S., Canada, Mexico) Europe (U.K., Italy, Germany, France, Rest of the EU) Asia-Pacific (India, Japan, China, South Korea, Australia, Rest of APAC) Latin America (Chile, Brazil, Argentina, Rest of Latin America) Africa and the Middle East (Saudi Arabia, U.A.E., South Africa, Rest of MEA)

% : $ ) :https://www.worldwidemarketreports.com/promobuy/817034

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This research contains detailed information on the factors that are projected to impact Gene Editing Service market growth and share in the future.

The report examines the present situation of the Gene Editing Service market as well as future prospects for a variety of geographic locations.

It can be used as a SWOT and competitive landscape study when combined with Porters Five Forces analysis.

It gives an in-depth examination of the industry, highlighting its growth rates and expansion potential.

The research contains a wealth of information, including Gene Editing Service market dynamics and opportunities for the forecast period.

Quantitative, qualitative, value (USD Million), and volume (Units Million) data are among the segments and sub-segments.

Data on demand and supply forces, as well as their impact on the Gene Editing Service market, may be found at the regional, sub-regional, and country levels.

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Mr. ShahWorldwide Market Reports,Tel: U.S. +1-415-871-0703U.K. +44-203-289-4040Japan +81-50-5539-1737Email: [emailprotected]Website:https://www.worldwidemarketreports.com/

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Gene Editing Service Market 2022 : Top Players to Reflect Impressive Growth Rate till 2029: Caribou Biosciences, CRISPR Therapeutics, Merck KGaA,...

SEATTLE Oct. 4, 2022 Below are summaries of recent Fred Hutchinson Cancer Center research findings and other news.

Join us for our next virtual science event focused on precision oncology. The discussion will be 10 a.m. - 11 p.m. PT on Wednesday, Oct. 12 and will feature Drs. Thomas Lynch Jr., Christina Baik, Sujata Jana, Jeff Leek and Colin Pritchard. Please RSVP HERE by Monday, Oct. 10.

And if youre looking for sources for Octobers Breast Cancer Awareness Month, please see our breast cancer page for a list of projects, experts and the latest breast cancer news.

Fred Hutch organizational news

Fred Hutch begins rollout of new brandFred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance formally merged in April 2022 to officially become Fred Hutchinson Cancer Center. In October the organizations new brand was officially launched, with updates to websites, social media channels and banners as well as updated signs around the South Lake Union campus. Patients and providers will see temporary signage and other updates at Fred Hutch clinical care sites around the Seattle region in the coming months.Media contact: Kat Wynn, kwynn@fredhutch.org

Precision oncology

Transformative $78M gift to establish new precision oncology instituteStuart and Molly Sloan have pledged $78 million to support Fred Hutch. The gift will further advance Fred Hutchs work in precision oncology, which integrates fundamental biology, technology, immunology, data science and clinical experience into strategies to prevent, detect and treat cancer.Media contact: Kat Wynn, kwynn@fredhutch.org

Infectious disease

Researchers refine experimental gene therapy for herpesTwo years after scientists showed that an experimental gene therapy for herpes which affects billions of people around the world can knock out most latent infection in mice, new tests reveal that it also suppresses the amount of transmissible virus shed by the treated animals. In a paper posted on bioRxiv, Fred Hutch virologists Drs. Keith Jerome and Martine Aubert report the treatment dramatically reduced or even eliminated viral shedding in treated mice compared to controls.Media contact: Molly McElroy, mwmcelro@fredhutch.org

Health equity

Spokane Regional Health District: Gaps in cancer care experienced in SpokaneFor their podcast Cancer Health Equity Now, members of Fred Hutchs Office of Community Outreach and Engagement in Spokane spoke with Spokane Regional Health District Health Officer Dr. Frank Velazquez. They discussed gaps in access to cancer care in Spokane, collaboration efforts by local organizations, and the feasibility of a sustainable Mobile Mammogram Program locally.Media contact: Kat Wynn, kwynn@fredhutch.org

On Twitter

Follow Dr. Aakansha Singhvi (@SinghviLabGlia) as the Singhvi lab works towards understanding how glia-neurons interact with the nervous system and the key role worms play.

Virus evolution expert Dr. Jesse Bloom (@jbloom_lab) recently shared a study on the evolution of antibody immunity following Omicron BA. He said, Although response to Omicron dominated by preexisting B-cells, antibodies from these cells better at 6 months than 1 month.

Science spotlightScience Spotlight is a monthly installment of articles written by postdoctoral fellows at Fred Hutch that summarize new research papers from Hutch scientists. If youre interested in learning more or covering these topics, contact: media@fredhutch.org

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Fred Hutchinson Cancer Center unites comprehensive care and advanced research to provide the latest cancer treatment options and accelerate discoveries that prevent, treat and defeat cancer and infectious diseases worldwide.

Based in Seattle, Fred Hutch is an independent, nonprofit organization and the only National Cancer Institute-designated cancer center in Washington. We have earned a global reputation for our track record of discoveries in cancer, infectious disease and basic research, including important advances in bone marrow transplantation, HIV/AIDS prevention, immunotherapy and COVID-19 vaccines. Fred Hutch operates eight clinical care sites that provide medical oncology, infusion, radiation, proton therapy and related services and has network affiliations with hospitals in four states. Fred Hutch also serves as UW Medicines cancer program.

Please note that our organization was renamed Fred Hutchinson Cancer Center in April 2022, following the merger of long-time partners, Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance.

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Tip Sheet: $78 million to support new precision oncology institute, update on experimental gene therapy for herpes and the launch of Fred Hutch's new...

Cell and gene therapies seek to correct the root cause of an illness at the molecular level. These game-changing medicines are reshaping how we address previously untreatable illnesses transforming peoples lives.

Cell and gene therapy represent overlapping fields of research with similar therapeutic goals developing a treatment that can correct the underlying cause of a disease, often a rare inherited condition that can be life-threatening or debilitating and has limited treatment options.

While these technologies were initially developed in the context of treating rare diseases caused by a single faulty gene, they have since evolved towards tackling more common diseases, says Professor Rafael J. Yez-Muoz, director of the Centre of Gene and Cell Therapy (CGCT) at Royal Holloway University of London.

A powerful example is the chimeric antigen receptor (CAR) T-cell therapies, which have been approved for treating certain blood cancers. The approach involves genetically modifying a patients T cells in the laboratory before reintroducing them into the body to fight their disease.

For the first time, we had an example of gene therapy to treat a more common disease demonstrating that the technology has wide applicability, enthuses Yez-Muoz.

To date, 24 cellular and gene therapy products have received approval from the US Food and Drug Administration (FDA) including life-changing treatments for patients with rare diseases, such as inherited forms of blindness and neuromuscular conditions. A variety of gene and cell-based therapies for both rare and common diseases are also currently in development across many therapeutic areas, offering hope for many more families in coming years.

This webinar will provide an introduction to the regulatory framework for cell and gene therapies and highlight the importance of chemistry, manufacturing and controls. Watch to learn about regulatory concerns, safety and quality testing throughout the product lifecycle and key acronyms and terminology.

Gene therapies seek to introduce specific DNA sequences into a patients body to treat, prevent or potentially cure a disease. This may involve the delivery of a functional gene into cells to replace a gene that is missing or causing a problem or other strategies using nucleic acid sequences (such as antisense oligonucleotides or short interfering RNAs [siRNAs]) to reduce, restore or modify gene expression. More recently, scientists are also developing genome-editing technologies that aim to change the cells DNA at precise locations to treat a specific disease.

The key step in successful gene therapy relies on the safe and efficient delivery of genetic material into the target cells, which is carried out by packaging it into a suitable delivery vehicle (or vector). Many current gene therapies employ modified viruses based on adenoviruses, adeno-associated viruses (AAV), and lentiviruses as vectors due to their intrinsic ability to enter cells. But non-viral delivery systems such as lipid nanoparticles (LNPs) have also been successfully employed to deliver RNA-based therapeutics into cells.

A big advantage of using viral vectors for gene delivery is they are longer lasting than non-viral systems, states Dr. Rajvinder Karda, lecturer in gene therapy at University College London. Many of the rare diseases were aiming to tackle are severe and we need to achieve long-term gene expression for these treatments to be effective.

While improved technological prowess empowers the development of CRISPR-edited therapies, supply-chain and manufacturing hurdles still pose significant barriers to clinical and commercialization timelines. Watch this webinar to learn more about the state of CRISPR cell and gene therapies, challenges in CRISPR therapy manufacturing and a next-generation manufacturing facility.

Viral-vector gene therapies are either administered directly into the patients body (in vivo), or cells harvested from a patient are instead modified in the laboratory (ex vivo) and then reintroduced back into the body. Major challenges for in vivo gene delivery approaches are with the safe and efficient targeting of the therapeutic to the target cells and overcoming any potential immune responses to the vectors.

As well as getting the genetic material into the affected cells, we also need to try and limit it reaching other cells as expressing a gene in a cell where its not normally active could cause problems, explains Dr. Gerry McLachlan, group leader at the Roslin Institute in Edinburgh.

For example, the liver was identified as a major site of toxicity for an AAV-based gene therapy approved for treating spinal muscular atrophy (SMA), a type of motor neuron disease that affects people from a very young age.

Unfortunately, these viruses are leaky as theyre also going to organs that dont need therapy meaning you can get these off-target effects, says Karda. Theres still work to be done to develop and refine these technologies to make them more cell- and organ-specific.

It is also important to ensure the gene is expressed at the right level in the affected cells too high and it may cause side effects and too little may render the treatment ineffective. In a recent major advancement in the field, scientists developed a dimmer switch system Xon that enables gene expression to be precisely controlled through exposure to an orally delivered small molecule drug. This novel system offers an unprecedented opportunity to refine and tailor the application of gene therapies in humans.

Download this whitepaper to discover an electroporation system that resulted in CAR transfection efficiencies as high as 70% in primary human T cells, can avoid the potential risks associated with viral transduction and is able to produce CAR T cells at a sufficient scale for clinical and therapeutic applications.

In 1989, a team of researchers identified the gene that causes the chronic, life-limiting inherited disease cystic fibrosis (CF) the cystic fibrosis transmembrane conductance regulator (CFTR). This was the first ever disease-causing gene to be discovered marking a major milestone in the field of human genetics. In people with CF, mutations in the CFTR gene can result in no CTFR protein, or the protein being made incorrectly or at insufficient levels all of which lead to a cascade of problems that affect the lungs and other organs.

Our team focuses on developing gene therapies to treat respiratory diseases in particular, were aiming to deliver the CTFR gene into lung cells to treat CF patients, says McLachlan.

The results of the UK Respiratory Gene Therapy Consortiums most recent clinical trial showed that an inhaled non-viral CTFR gene therapy formulation led to improvements in patient lung function.

While this was encouraging, the effects were modest and we need to develop a more potent delivery vehicle, explains McLachlan. Weve also been working on a viral-based gene therapy using a lentiviral vector to introduce a healthy copy of the CTFR gene into cells of the lung.

Kardas team focuses on developing novel gene therapy and gene-editing treatments for incurable genetic diseases affecting the central and peripheral nervous system and Yez-Muoz is aiming to develop new treatments for rare neurodegenerative diseases that affect children, including SMA and ataxia telangiectasia (AT).

But a significant barrier for academic researchers around the world is accessing the dedicated resources, facilities and expertise required to scale up and work towards the clinical development and eventually the commercial production of gene and cell therapies. These challenges will need to be addressed and overcome if these important advancements are to successfully deliver their potentially life-changing benefits to patients.

Download this app note to discover how electron activated dissociation can obtain in-depth structural characterization of singly charged, ionizable lipids and related impurities, decrease risk of missing critical low abundance impurities and increase confidence in product quality assessment.

After many decades of effort, the future of gene and cell therapies is incredibly promising. A flurry of recent successes has led to the approval of several life-changing treatments for patients and many more products are in development.

Its no longer just about hope, but now its a reality with a growing number of rare diseases that can be effectively treated with these therapies, describes Yez-Muoz. We now need to think about how we can scale up these technologies to address the thousands of rare diseases that exist and even within these diseases, people will have different mutations, which will complicate matters even further.

But as more of these gene and cell-based therapies are approved, there is a growing urgency to address the challenge of equitable access to these innovative treatments around the world.

Gene therapies have the dubious honor of being the most expensive treatments ever and this isnt sustainable in the longer term, says Yez-Muoz. Just imagine being a parent and knowing there is an effective therapy but your child cant access it that would be absolutely devastating.

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Cell and Gene Therapy: Rewriting the Future of Medicine - Technology Networks

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

BioPharm InternationalVol. 35, No. 10October 2022Pages: 4951

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," BioPharm International 35 (10) 4951 (2022).

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Growth in Cell and Gene Therapy Market - BioPharm International

ZUG, Switzerland and BOSTON, Sept. 28, 2022 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX130, the Companys wholly-owned allogeneic CAR T cell therapy targeting CD70, for the treatment of Mycosis Fungoides and Szary Syndrome (MF/SS).

The RMAT designation is an important milestone for the CTX130 program that recognizes the transformative potential of our cell therapy in patients with T-cell lymphomas based upon encouraging clinical data to date, said Phuong Khanh (P.K.) Morrow, M.D., FACP, Chief Medical Officer of CRISPR Therapeutics. "We continue to work with a sense of urgency to bring our broad portfolio of allogeneic cell therapies to patients in need.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

About CTX130 and COBALT TrialsCTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR T investigational therapy targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1 single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 in adult patients. The COBALT-LYM trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory T or B cell malignancies. The COBALT-RCC trial is evaluating the safety and efficacy of CTX130 for the treatment of relapsed or refractory renal cell carcinoma. CTX130 has received Orphan Drug and Regenerative Medicine Advanced Therapy designations from the FDA.

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 Boston, 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 made by Dr. Morrow in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics including, without limitation, expectations regarding the benefits of RMAT designation; and (ii) 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 for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; 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, quarterly report on Form 10-Q 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.

CRISPR THERAPEUTICS standard character mark and design logo, CTX130 and COBALT are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

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

Media Contact:Rachel Eides+1-617-315-4493rachel.eides@crisprtx.com

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CRISPR Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX130 for the Treatment of Cutaneous T-Cell...

BLA Includes Substantial Body of Data from Pivotal Phase 3 and Ongoing Phase 1/2 Studies

If Approved, Would Be 1st Gene Therapy in U.S. for Treatment of Severe Hemophilia A

SAN RAFAEL, Calif., Sept. 29, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Company resubmitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The resubmission incorporates the Company's response to the FDA Complete Response (CR) Letter for valoctocogene roxaparvovec gene therapy issued on August 18, 2020, and subsequent feedback, including two-year outcomes from the global GENEr8-1 Phase 3 study and supportive data from five years of follow-up from the ongoing Phase 1/2 dose escalation study.

BioMarin anticipates an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure. If approved, valoctocogene roxaparvovec would be the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017.

In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN on August 24, 2022 and endorsed the recommendation from the European Medicines Agency (EMA) to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity in the European Union.

"We are pleased to reach this point in the development program for valoctocogene roxaparvovec and look forward to working with the FDA with the goal of bringing a potentially transformative therapy to people with severe hemophilia A in the United States," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "This large and robust data set provided in this BLA resubmission shows an encouraging efficacy profile. We remain committed to sharing these data with the public, along with even longer-term data generated through our ongoing clinical trials and any post-approval studies, to further our understanding of AAV gene therapy in severe hemophilia A and of gene therapies more broadly."

The resubmission includes a substantial body of data from the valoctocogene roxaparvovec clinical development program, the most extensively studied gene therapy for severe hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data package included supportive evidence from five years of follow-up from the 6e13 vg/kg dose cohort in the ongoing Phase 1/2 dose escalation study. The resubmission alsoincludesaproposedlong-term extension studyfollowingall clinicaltrialparticipantsfor up to 15years, as well astwo post-approval registry studies.

Robust Clinical Program

BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).

Safety Summary

Overall, to date, a single 6e13 vg/kg dose of valoctocogene roxaparvovec has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with valoctocogene roxaparvovec have occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation, a laboratory test of liver function, has remained the most common adverse drug reaction. Other adverse reactions have included aspartate aminotransferase (AST) elevation (101 participants, 63%), nausea (55 participants, 34%), headache (54 participants, 34%), and fatigue (44 participants, 28%). No participants have developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with valoctocogene roxaparvovec.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (Factor VIII 1-5%) or mild (Factor VIII 5-40%) disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week (100-150 infusions per year) or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month (12-48 injections or shots per year). Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visitwww.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin anticipating an FDA response by the end of October on whether the BLA resubmission is complete and acceptable for review, BioMarin's expectations regarding the duration of the review procedure, valoctocogene roxaparvovec being the first commercially-available gene therapy in the U.S. for the treatment of severe hemophilia A, if approved, BioMarin's commitment to sharing longer-term data generated through its ongoing clinical trials and any post-approval studies. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec; additional data from the continuation of the clinical trials of valoctocogene roxaparvovec, any potential adverse events observed in the continuing monitoring of the participants in the clinical trials; the content and timing of decisions by the FDA and other regulatory authorities, including decisions to grant additional marketing registrations based on an EMA license; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec for the clinical trials and commercially; and those and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc and ROCTAVIAN is a trademark of BioMarin Pharmaceutical Inc.

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BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA - PR...