Category Archives: Genetic Therapy

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Cutting-edge research on rewriting the genes responsible for Tay-Sachs disease, sickle-cell anemia, and other disorders will be presented at this weeks online annual meeting of the American Society for Gene and Cell Therapy. Originally planned as a Boston gathering, the scientific groups get-together became a virtual one because of the Covid-19 pandemic.

From Tuesday through Friday, academic researchers will be presenting their latest data online, along with updates from firms like Bluebird Bio (ticker: BLUE), Voyager Therapeutics (VYGR), Fate Therapeutics (FATE), Beam Therapeutics (BEAM), Axovant Gene Therapies (AXGT), and many others. Patients and their families have found their way to clinical trials through the societys website.

Bluebird plans presentations on its cell therapy against the blood cancer known as multiple myeloma. Using a technology known as CAR-T, the company creates supercharged versions of a patients immune cells that have halted disease progression in some of the 18 patients enrolled in a continuing Phase 1 trial.

Featured on Friday will be reports on the first babies treated with gene therapy for the debilitating neurodegenerative disorder Tay-Sachs. The treatment is being developed by the London-based Axovant under license from the University of Massachusetts Medical School.

Voyager will discuss its preclinical mouse studies on treating neurological disorders like amyotrophic lateral sclerosis and Huntingtons disease by using techniques that block the rogue signals generated by defective genes.

Fate Therapuetics is scheduled to show a new off-the-shelf CAR-T technology that it hopes will allow the immune system to target a broad range of solid tumors as well as multiple myeloma. The approach is licensed from Harvard Universitys Dana-Farber Cancer Institute.

Beam, meanwhile, will detail success it has shown in preclinical editing of the genetic defect that causes sickle-cell anemia. The company is developing a sharper-edged way of rewriting faulty genes than the widely used Crispr technology that Beam licensed from researchers at the Broad institute of Harvard and MIT. Beam founder and Crispr pioneer Feng Zhang will give a featured lecture as part of the online meeting on Thursday.

Write to Bill Alpert at william.alpert@barrons.com

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Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering - Barron's

Trying to hack fitness is a multi-million-dollar industry; weve all seen at least one ad featuring a purported miracle product that claims it can make people lose weight and look greatwithout even trying. From low-effort exercise machines to strange-ingredient diets to fat-burning belts and bands, theres no shortage of attempts to make it easy to be fit.

A gene therapy trial performed on mice may foreshadow yet another way to hack fitness. In a study done by a team at Washington University in St. Louis medical school, mice quickly built muscle mass and reduced obesity after receiving the therapy, even while eating a diet high in fat and not exercising. The results were published last week in a paper in Science Advances.

Sound appealing? Heres how it worked.

The gene targeted was FST, which is responsible for making a protein called follistatin. In humans and most other mammals, follistatin helps grow muscle and control metabolism by blocking a protein called myostatin, which acts to restrain muscle growth and ensure muscles dont get too big.

The researchers injected eight-week-old mice with a virus carrying a healthy FST gene (gene therapy involves adding healthy copies of a gene to cells, usually using a virus as a deliveryman).

Over a period of 18 weeks, or about 4 months, the team observed that the muscle mass of the treated mice more than doubled, as did their strength level. They also experienced reduced damage related to osteoarthritis, less inflammation in their joints, and had healthier hearts and blood vessels than mice that didnt receive the gene therapyeven though all the mice ate the same high-fat diet and did the same amount of exercise.

Going into the study, the researchers worried the muscle growth catalyzed by the gene therapy could harm the heart, mainly through thickening of the hearts walls. Surprisingly, though, heart function and cardiovascular health of the treated mice actually improved. In subsequent studies, the team will continue to monitor the treatments effect on the heart, as complications could emerge over time.

Talk about a fitness hack; imagine being able to build muscle and maintain a healthy metabolism while lounging on the couch eating burgers and fries. There have been similar studies to replicate the effects of exercise by commandeering the genetic instructions that control the way cells interact with proteins; though various exercise pills have successfully mimicked the effects of regular cardiovascular activity in mice, scientists still dont fully understand how, at a molecular level, exercise has the effects it does on the human body.

This may change in the next couple years, though; a National Institutes of Health consortium called the Molecular Transducers of Physical Activity is in the midst of an in-depth study on the molecular effects of exercise on tissues and organs in 3,000 people.

If the muscle-building gene therapy eventually reaches a point where it can be used in humans, though, the research team isnt viewing it as a quick-fix health hack. Rather, it would be used to help get people with conditions like muscular dystrophy or severe obesity to a baseline from which they could adopt tried-and-true muscle-building practices like weight lifting or physical therapy.

In cases of severe obesity or muscle loss, it is extremely difficultif not impossibleto lose weight or improve muscle strength through normal exercise and diet, said Farshid Guilak, orthopedic surgery professor and director of research at Shriners Hospitals for Children in St. Louis. The goal of this study was to show the importance of muscle strength in overriding many of the harmful effects of obesity on the joint.

If every condition, process, and trait in our bodies is tightly linked to our genes, its conceivable that almost any aspect of our health could be manipulated using gene therapy and related tools. Maybe one day there will indeed be a pill we can take or a shot we can get to give us svelte, muscular bodies without any of the effort.

The fact that this would ruin the pleasure and satisfaction of a good workout is another conversationand one not everybody would be interested in having. But even if genetic or chemical exercise-replacement tools become safe to use in humans in the foreseeable future, theyll likely be limited, at least at first, to those who need them due to debilitating health conditions.

That saidfor the time being, keep hitting the treadmill, the weight room, or your other off-the-couch, effort-intensive workout of choice.

Image Credit: Aberro Creative from Pixabay

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Forget ExerciseThese Mice Got Ripped With Gene Therapy - Singularity Hub

- Molecular Methods Quantified Precision and Efficiency of Nuclease-Free Gene Editingfor PKU -

- Manufacturing Enhancements Led to Improved Productivity, Quality and Scalability of Commercial Process, Confirmed in 2,000L Bioreactor -

- Data Highlight Unique Characteristics of AAVHSC Genetic Medicines Platform -

BEDFORD, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of data at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. Among Homologys seven presentations are data from its in vivo nuclease-free gene editing program for phenylketonuria (PKU) and in vivo gene therapy program for metachromatic leukodystrophy (MLD), both of which are in IND-enabling studies. Presentations also focus on the Companys commercial manufacturing platform, as well as data on the differentiating characteristics of Homologys family of AAVHSC vectors, particularly when compared to other AAVs, which highlight the potential of the Companys dual gene therapy and editing platform.

Homology has made substantial progress in understanding the unique properties of our AAVHSC-based technology and this enables us to move our dual genetic medicines platform forward to develop potential treatments, or cures, for patients, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. We are pleased to share data here that describe the molecular methods we have developed to characterize in vivo, nuclease-free gene editing efficiency and precision at the DNA level. Additional data from our in vivo MLD gene therapy program demonstrates the impact on key biomarkers in two species, as well as the durability of effect in the murine model of disease with data out to 52 weeks. Underpinning all our programs is our internal GMP process and manufacturing capabilities, where we have now confirmed our commercial HEK293 suspension platform at the 2,000-liter scale, bringing our total internal capacity to 3,500 liters. Additionally, we are presenting data showing improved AAVHSC packaging as compared to the non-Clade F vector AAV5.

Highlights from Homologys 2020 ASGCT Presentations

The presentation, Molecular Characterization of Precise In Vivo Targeted Gene Editing in Human Cells using AAVHSC15, a New AAV Derived from Hematopoietic Stem Cells (AAVHSC), describes quantitative molecular methods to measure efficiency and precision of nuclease-free, homologous recombination-based gene editing. The studies, which used a single I.V. administration of a gene editing construct to insert the human PAH gene, which is mutated in people with phenylketonuria (PKU), in a humanized liver murine model, show:

Two posters related to Homologys internal commercial manufacturing platform will be presented.In Molecular Design and Characterization of Packaging Plasmid Sequences for Improved Production of Novel Clade F AAVHSCs, the data demonstrate:

In Development and Scalability of Transfection-Based Production and Purification of Novel Clade F Adeno-Associated Viruses Isolated from Human Hematopoietic Stem Cells (AAVHSCs), Homology describes high-quality productivity and scalability of its mammalian, suspension-based manufacturing, including:

Related to Homologys HMI-202 investigational gene therapy for MLD, the presentation, Gene Therapy for Metachromatic Leukodystrophy (MLD) That Crosses the Blood-Nerve and Blood-Brain Barriers in Mice and Non-Human Primates, details that a single I.V. administration:

In collaboration with Childrens Hospital of Philadelphia (CHOP), Homology also presents, In Vivo Transduction of Murine Hematopoietic Stem Cells after Intravenous Injection of AAVHSC15 and AAVHSC17, which shows:

As Homology has advanced its AAVHSC technology, it is presenting mechanistic data on the platform, including the following two presentations.In Role of Terminal Galactose in Cellular Uptake, Intracellular Trafficking, and Tissue Tropism Using Adeno-Associated Viruses Isolated from Human Stem Cells (AAVHSCs), the data show:

In AAVHSCs Transduction Does Not Significantly Elicit p53-Mediated Apoptosis or Alter Cell Cycle in Human iPSCs and Primary Cells When Compared to Non-Clade F AAV Vectors, the studies demonstrate that AAVHSCs:

For more information about the presentations, visit Homologys website at http://www.homologymedicines.com/publications.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; our beliefs regarding our manufacturing capabilities; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

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Homology Medicines Announces Presentations on its In Vivo Gene Therapy and Gene Editing Programs and Commercial Manufacturing Platform at the American...

Japan's national health insurance will cover a gene therapy drug for a rare childhood genetic disorder that costs 167 million yen ($1.56 million) per treatment, making it the most expensive medication funded by the public system, government officials said Wednesday.

An advisory panel to the health minister approved provision of Swiss pharmaceutical giant Novartis AG's drug Zolgensma for spinal amyotrophy patients under the age of 2 starting as early as May 20, the officials said.

The drug, which costs over 200 million yen in the United States, is known as the world's most expensive medication.

Supplied photo shows samples of Swiss pharmaceutical giant Novartis AG's gene therapy drug "Zolgensma" for spinal amyotrophy patients. (Photo courtesy of Novartis)

It is a one-time therapy for the genetic disorder, which causes motor neuron loss and muscle wasting, and its coverage by insurance will offer hope for patients of the disease and their families.

With Japan's social security expenses ballooning amid the rapid aging of its population, some experts have expressed concerns over the burden on the insurance system of inclusion of the treatment. They also note that new drugs have tended to be highly expensive in recent years.

But a senior health ministry official said that given the small number of patients of the rare illness, "the fiscal impact is going to be limited."

The previous most expensive drug in Japan was Kymriah, approved last May to treat leukemia and other hematologic cancers, at 33.49 million yen.

Novartis Pharma K.K., a Tokyo-based unit of the Swiss pharmaceutical company, produces and sells both Kymriah and Zolgensma.

Spinal amyotrophy affects one or two infants out of every 100,000 and can lead to severe respiratory problems and early death. Without use of an artificial respirator, it is said that most die within 18 months.

Zolgensma will be given as a one-time infusion into the vein, which can introduce normal genes into human cells to recover motor function.

Novartis expects that the drug will be administered to about 25 patients per year in Japan, estimating annual sales of 4.2 billion yen.

Under the Japanese insurance system, out-of-pocket medical expenses for those under 2 are set at 20 percent of the total. But the real payment is minimal as the central and municipal governments cover almost all the expense under subsidy programs.

Related coverage:

Japan health insurance to cover new leukemia therapy worth 33 mil. yen

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Japan's health insurance to cover $1.5 million gene therapy drug - Kyodo News Plus

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Key player focused on this market are: Advantagene, Amarna Therapeutics, AnGes MG, Inc., Applied Genetic Technologies Corporation, AskBio, Avalanche, Bluebird bio, Celladon Corporation, Dimension Therapeutics, American Gene Technologies International Inc,

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Market Revenue (M USD) and Growth Rate 2015-2023a

Sales and Growth Rate 2015-2023a

Major Players Revenue (M USD) in 2020

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ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 14, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (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.

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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...