The FDA has granted May 29, 2023, as the action date for the companys biologics license application (BLA) for accelerated approval of SRP-9001 for treating For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. It is currently being investigated in a Phase I/II study in six boys ages 4 and up. The trials participants will get either a single infusion of gene therapy or a placebo, and they will be tracked for 52 weeks (about a year). With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. Also, if you were to treat infants, its important to remember that they will be making new muscle cells without the modified gene in them, so there is a balance of when to treat.. Each of these gene therapies has slight variations in their three main components: the transgene, the Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. Autolus specializes in developing CAR-T cell therapies. Sarepta Therapeutics said topline results from Part 2 of its study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of its gene therapy for the progressive neuromuscular condition Duchenne muscular dystrophy, showed statistically ISSN 2940-2034, systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Tiny, anti-inflammatory nanomotors to treat rheumatoid arthritis, Explaining the Universes accelerated expansion without dark energy, New material is a game changer in radiative cooling, A quark star may have just been discovered, Nanoparticles that self-assemble inside cells to fight cancer, James Webb Telescope images the Pillars of Creation, Anti-aging drug could help turn back the clock, Meteorite that struck a driveway in small UK town holds key ingredients for life. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. In September 2021, Astellas Pharma reported a fourth patient death in the AT132 clinical Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. Could we use histamine? This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. Within the context of Duchenne and other rare diseases, its a very robust sample size and one that will grow with data from EMBARK.. There are two main types of gene therapy: somatic gene therapy and germline gene therapy. The company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. Moreover, the companies are hoping that their Duchenne Muscular Dystrophy treatment will slow or even stop disease progression, giving patients a chance to avoid the devastating effects of Duchenne. He is currently a Professor of Physiology and Biophysics at the University of Washington. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. WebThere are many challenges facing the management of DMD patients in the United Arab Emirates and Kuwait and most likely other countries within the Middle East. Duchenne muscular dystrophy (DMD) is a fatal condition caused by a single gene mutation on the X-chromosome being X-linked means only males suffer from the disease. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. Duchenne Muscular Dystrophy is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Although we now know DMD is a genetic disease, it wasnt that long ago that researchers didnt know why or how the disease came about. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9), being developed by Asklepios BioPharmaceutical (AskBio). Graphite Bio is building on CRISPR technology and working with the cells natural DNA repair processes to rewrite genes. AvroBio focuses on lyosomal disorders. The American Society of Clinical Oncology is a platform that provides a global connection to researchers, pharma companies, and healthcare professions standing against cancer, finding a cure for it. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Gene therapy is an umbrella term for a range of therapies that target the genetic underpinnings of disease. FDA accepts BLA for Roche-Sareptas DMD gene therapy. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Pfizer Inc. Website: www.pfizer.com. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. The biotech aims to engineer precision genetic medicine for rare diseases. 6 min read. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. WebAbstract. We had been studying in dogs a disease that phenotypically appeared analogous to Duchenne dystrophy for several years going back into the early 80s, said Kornegay. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? Viltepso is an antisense oliogonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Viruses are very well evolved to get into cells, commented Hesterlee. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. ARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3. Vertex has acquired Exonics and has a partnership with CRISPR Therapeutics to develop a gene-editing platform for Duchennes muscular dystrophy (DMD) and myotonic dystrophy (DM1). How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). Also working on a gene therapy for DMD is Solid Biosciences, which has also encountered trouble. Web2 Department of Gene Therapy, Saad Pharmaceuticals, Tornime 7-26, Tallinn, 10145, Estonia. FDA Approves BeiGenes Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. Monkel is an established researcher in the neuromuscular disease field and his research has led to novel gene discoveries for previously undiagnosed rare muscle diseases. Muscle weakness and atrophy spread from the trunk and forearms to other muscles throughout the body as the disease advances. 6 min read. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). For example, Eteplirsen (Exondys 51) is expected to cost patients around US$ 300,000 for a treatment course and the cost of the treatment can go as high as US$ 750,000 annually. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. According to Kornegay, We showed remarkable decline in loss of respiratory function.. One component condenses the DNA of the gene and protects it from degradation. In September 2021, the company announced. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Anywhere from 10 to 80 percent of DMD patients, depending on the serotype in question, have preexisting antibodies against AAVs, meaning they are not eligible for gene therapy, Hesterlee elaborated. ORLANDO, FloridaJeffrey Chamberlain, PhD, outlined the 4 different types of gene therapy for treating Duchenne muscular dystrophy (DMD) at the Gene Therapy and Gene Editing Symposium which took place on the second day of the CureDuchenne 2022 FUTURES National Conference . This is based on a proprietary algorithm built from the drugs sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalDatas Pharmaceutical Intelligence Center. The field continues to multiply in size. Rocket Pharmaceuticals is aiming for its first regulatory filing in H1 of 2023 for its LVV gene therapy RP-201 for Leukocyte Adhesion Deficiency-I (LAD-I) a rare, autosomal recessive pediatric disease where, without a successful bone marrow transplant. The company aims to develop therapies for neurological disorders and other diseases. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. SLL is Gilead Buys Out Rights to Cancer Therapy from Jounce for USD 67 Million Gilead Sciences must have liked what it saw in a two-year-old collaboration with Jounce Therapeutics for CCR8-targeting cancer immunotherapy because the company has just agreed to own the program fully. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. Vyondys 53 (golodirsen) Injection. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Other hurdles of developing a DMD gene therapy. Importantly for Kornegay, the trial showed the treatment was safe. [This feature is a part of 2022s Pharma 50 series.]. Fixing the mutated gene (through gene editing) or providing cells with a new healthy copy of the gene (through gene therapy) would provide the best benefit, possibly even leading to a lifelong cure. "Within the context of Duchenne and other rare diseases, it's a very robust sample size and one that will grow with data from EMBARK.". The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. This may lead to dangerous side effects. In preclinical studies, the presence of the CT domain was shown to recruit several key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle damage in dystrophic mice. MDA gave research grants to four labs tasked with finding the cause. It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. But we were cautious after the high profile death of Jesse Gelsinger in 1999.. Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. Whole-body systemic gene therapy is likely the most effective way to reduce greatly the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. The earlier you treat, the better, but its hard to measure benefit if the children are not yet manifesting a lot of symptoms, so you want to test the children at a stage when theyre progressing, said Hesterlee. The trials main purpose is to monitor changes in NSAA scores. By Chelsea Weidman Burke. Pfizer plans to begin a Phase III study with PF-06939926 by the end of 2020. The company boasts that its AI Workbench can help manage the complexity in RNA biology while identifying novel targets. As an example, Dystrophin, the gene responsible for Duchenne muscular dystrophy (DMD) is 14 kb, meaning that only one-third of the dystrophin gene can be "packaged" into AAV. Rare Daily Staff. Data are expected to start rolling in late next year. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. Founded in 2014, Intellia Therapeutics is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies for a range of diseases, including cancer and genetic disorders. Their gene therapy product, SB-525, is currently in clinical trials for the treatment of hemophilia A. Click for Index The three patients receiving the low dose (1E14 vg/kg) had a mean percent dystrophin expression in muscles of 28.5 percent at two months and 21.2 percent at 12 months, compared to the six patients receiving the high dose (3E14 vg/kg) had 48.4 percent dystrophin expression at two months, three of whom had 50.6 percent at 12 months. We have developed other critical functions to ensure proper gene delivery. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. Thankfully, another group of researchers working on a milder form of Duchenne muscular dystrophy called Becker muscular dystrophy found that in these patients that large chunks of the gene were missing but a protein was still produced. A Non-Viral delivery method is much less likely to elicit an immune response, enabling repeated dosing over months or years. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. The FDA soon put the Phase Ib study under clinical hold. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. Also working on a gene therapy for DMD is Solid Biosciences, which has also been having trouble. It has six programs in clinical development. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. James is an expert in CMC drug development, with decades of experience in the Pharmaceutical industry. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. According to the market research firm Emergent Research, international cell and gene therapy companies could generate $6.6 billion in revenue by 2027, with a projected CAGR of 19.8% from 2020 to 2027. But unfortunately, a participant in Pfizers Phase Ib open-label study died unexpectedly. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 from AskBio, a company involved in early DMD gene therapy trials). Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. Regardless, Pfizer will need to examine the situation and acquire the data necessary to continue the Phase Ib trial and make changes to future trials, such as omitting certain mutation types. Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. SGT-001 is a systemically administered candidate that provides the body with a synthetic dystrophin gene called microdystrophin. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. In patients with Duchenne muscular dystrophy, the affected gene codes for the protein, dystrophin, which acts as a shock absorber between muscle cells and connective tissue, as well as supporting muscle contraction. Duchenne Muscular Dystrophy is caused by a dystrophin gene mutation (a protein in the muscles). SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Eventually, they will need ventilation to help them breathe. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. Byrne and colleagues now had a therapeutic that would fit in the AAV. Gene therapy is more efficient and covers everyone, regardless of genetic mutations, but its still good to have options while new therapies are in development.. Somatic gene therapy involves modifying genes in non-reproductive cells, such as cells in the skin or blood. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. "The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne," Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. Published: Jul 29, 2020 The companys core focus areas include immuno-oncology and plant sciences. Sarepta and its partner Roche presented new results and analyses on their experimental gene therapy SRP-9001 for the neuromuscular condition Duchenne muscular dystrophy showed consistent, statistically significant functional benefits in individuals. This news closes a tumultuous time for Astellas regarding the therapy. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. Powered by Madgex Job Board Software, virtual American Society of Gene and Cell Therapy (ASGCT) meeting, NorthStar Ambulatory Assessment (NSAA) rating scale, randomized, placebo-controlled Phase II trial, recently granted SRP-9001 Fast Track designation. 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Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an Louise Rodino-Klapac, CSO, executive VP and head of R&D, Sarepta Permission granted by Sarepta If approved, SRP-9001, would be the first gene therapy for the muscular degenerative disease known as DMD and is slated for complete evaluation under the accelerated approval path by the end of May 2023. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. Back in the mid-1980s, the cause of DMD was still unknown all we knew was that it ran in families, there were no genes associated with the disease yet, Hesterlee explained. The most troublesome symptoms are breathing difficulties. AAV9 is a type of AAV that is particularly good at getting into muscle cells. The Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715. Today, many AAV-based gene therapy medications are Focuses on clinical-stage gene therapy. Participants in Part 2 of Study SRP-9001-102 scored 2.0 points higher on the mean North Star Ambulatory Assessment (NSAA) 48 weeks after SRP-9001 treatment compared to a pre-specified matched external control cohort (p value=0.0009). Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. That worked great for small genes, but not so well for dystrophin.. Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. The goal of gene therapy is to replace or repair a missing or faulty gene, introduce a new gene to help fight disease, or deactivate a harmful gene. According to data from Solids clinical program, SGT-001 has the potential to slow or stop the Duchenne progression, regardless of genetic mutation or disease stage. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Focusing on developing therapeutics for disorders of the central nervous system. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. (2022, January 10). The only Duchenne Muscular Dystrophy treatment available are steroids like dexamethasone and gene-targeting therapies including exon skipping from Sarepta Therapeutics and NS Pharma for two small subsets of patients. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. There are currently four companies who have DMD gene therapy products that have been given to boys with Duchenne, and three of the companies have ongoing studies in the USA. areas It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. WebWhilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced Even if both gene therapies reach the market, PF-06939926 is likely to face a delay due to the recent death in its Phase Ib trial. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Sarepta Therapeutics. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. Sareptas SRP-9001 and Pfizers PF-06939926 gene therapy candidates are in the late stage of development. This not only quickly diminishes the amount of therapeutic virus in the body, but it could also mean the patient would only be able to get one dose of therapy - any subsequent doses would be destroyed too quickly by the body or, worse, potentially cause a severe immune reaction. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. The companys late-stage clinical pipeline is targeting acute graft versus host disease, inflammatory bowel disease, acute respiratory distress syndrome, chronic low back pain and chronic heart failure reduced ejection fraction. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). All Rights Reserved. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. The disease is universally fatal. Take out the viral genes required to make copies of itself and put in the healthy copy of dystrophin, then the virus can get inside cells but not replicate.. This designation is designed to provide regulatory assistance and financial benefits to the therapys clinical research and evaluation, as well as a seven-year period of marketing exclusivity in the United States after regulatory clearance. The patient was a part of the studys non-ambulatory arm. 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Galgt2 ) ARO-HSD, ARO-C3 trained Cardiologist with a synthetic dystrophin gene over 440 times as is... Beta-1, 4 N-acetylgalactosaminyltransferase 2 ( GALGT2 ) Mb dystrophin gene mutation a... Critical functions to ensure that enough gene therapy candidates for gene therapy is an in! Years ago, scientist he Jiankui shocked his peers and the US Department of,! And up muscles throughout the body with a synthetic dystrophin gene called.!