He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. But it took another 30 years to be able to apply this knowledge to develop effective drugs., Although corticosteroids can slow the progression of DMD to some extent, they dont address the underlying issue the lack of functional dystrophin. [This feature is a part of 2022s Pharma 50 series.]. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. Gene therapy; Cell Within the context of Duchenne and other rare diseases, its a very robust sample size and one that will grow with data from EMBARK.. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. In addition, RNA has potential in treating brain cancer, asMass General Brighamobserves in its list of one dozen potentially disruptive cell and gene therapy innovations. The Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. Cellectis has more than two decades of experience in gene editing. AvroBio focuses on lyosomal disorders. Powered by Madgex Job Board Software. As a result, SRP-9001 would gain a competitive edge. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. It is usually observed between the ages of three and six. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. WebDuchenne muscular dystrophy is the result of a mutation or deletion of the gene encoding dystrophin, located on the X chromosome. All three companies are using different versions of minidystrophin, explained Hesterlee. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. 617). Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. The FDA hasacceptedRocheandSareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD). In recent years, weve gotten much better at detecting benefits in the boys even when they are in the early stages and improving, so trials have started to skew younger, including children as young as 4 years old.. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Their first gene therapy product, Zynteglo, was approved by the European Medicines Agency in 2019 to treat a form of inherited anemia. Their gene therapy products are based on ex-vivo gene therapy, which involves modifying a patient's own cells outside the body and then reintroducing them. According to the companys press release, preliminary data from nine boys with DMD (ages 6-12) showed the therapy was well-tolerated during intravenous infusion. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. The trick was using higher doses and the right serotypes of AAV to move the vector out of the bloodstream and into muscle, Hesterlee added. Other hurdles of developing a DMD gene therapy. The leading site for news and procurement in the pharmaceutical industry. By Tristan Manalac. This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. WebGene therapy Cell therapy Drug therapy Mutation specific approaches About clinical research Current trials in DMD Current trials in SMA Current trials in LGMD Facing the Challenges of Clinical Trials Overview of therapeutic approaches for SMA The Problem The splicing process Therapeutic strategies for SMA Outcome measures This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. It is difficult and costly to manufacture large quantities of AAV. EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301. One component condenses the DNA of the gene and protects it from degradation. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. In September 2021, the company reported Positive 1.5-year functional data and patient-reported outcome measures (Pediatric Outcomes Data Collection Instrument, or PODCI) for Patients 4-6 in the ongoing IGNITE DMD Phase I/II clinical trial of SGT-001. Each of these gene therapies has slight variations in their three main components: the transgene, the This may lead to dangerous side effects. 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. For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. The tricky part is getting the gene inside the cell. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. Currently, Matthew is a Venture Partner at Medicxi. 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. DMD starts to show its effects during early childhood. That year, Bayer also acquired BlueRock Therapeutics. There are two main types of gene therapy: somatic gene therapy and germline gene therapy. 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. Arising in one of every 3,500 to 5,000 male infants worldwide, DMD is a rare neuromuscular disease caused by mutations in the gene encoding for the protein dystrophin. According to Solids leadership, this would allow it to focus on two other key DMD programs. The FDA soon put the Phase Ib study under clinical hold. Or higher doses to drive the virus into the muscles? Hesterlee added. Jeff is an internationally recognized leader in the gene therapy and muscular dystrophy fields and has been a pioneer in AAV micro-Dystrophin gene therapy research and clinical development for DMD. 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. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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 Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. They finally found the perfect balance, naming the shortened genes microdystrophins.. Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. However, unlike Sarepta, Pfizer does not have any additional candidates that may join the market and earn market share if its gene therapy treatment fails to win approval, implying that the stakes are higher for the latter. SGT-001 is a systemically administered candidate that provides the body with a synthetic dystrophin gene called microdystrophin. The next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. This news closes a tumultuous time for Astellas regarding the therapy. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. In fact, the FDA recently granted SRP-9001 Fast Track designation. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has Significant Limitations; Adeno-Associated Virus (AAV) is a common virus used to deliver "fragments" of healthy genes to the cells that contain unhealthy (mutated) genes. 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 biotech specializes in creating gene therapies for severe genetic disorders and cancer. 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). GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). An IND application is expected by the end of 2021. solutions for life science vertical and offering quintessential advisory services in the In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. It is usually observed between the ages of three and six. While Solid Biosciences SGT-001 and Regenxbios RGX-202 are in the early stage of development for DMD treatment. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. July 6, 2022. Successful introduction of gene therapy to treat DMD will require careful planning, education, capacity building and prioritization of core i Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. Gene therapy is an umbrella term for a range of therapies that target the genetic underpinnings of disease. Viruses are very well evolved to get into cells, commented Hesterlee. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. These micro-dystrophins might provide only partial improvement of muscle function. The problem is exon skipping, in its current form, is not very efficient and each therapy only works in a subset of children with certain gene mutations, Hesterlee commented. Rare Daily Staff. WebGene therapy Cell therapy Drug therapy Mutation specific approaches About clinical research Current trials in DMD Current trials in SMA Current trials in LGMD Facing the Challenges of Clinical Trials Overview of therapeutic approaches for SMA The Problem The splicing process Therapeutic strategies for SMA Outcome measures For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. Stan was elected to the Washington State Academy of Sciences in 2013 and selected as a Fellow of the American Association for the Advancement of Sciences in 2017. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Despite the risks mentioned above, which may result in lower uptake than Sareptas product, Pfizer could still capture a significant market share and see a return on its investment before more gene therapies enter the market. Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. MDA gave research grants to four labs tasked with finding the cause. The company is developing CRISPR/Cas9 genome editing technology. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. Cell and gene therapies promise to enable broad changes in the healthcare system over the next decade, prompting a growing number of cell and gene therapy companies to join the space. Now, after serving three years in a Chinese prison for practicing medicine without a license, he faces obstacles and critics as he tries to re-enter science. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. 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 . Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). 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. Verified The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. It is very likely that one or both of these gene therapies could be approved., This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation, Hesterlee concluded. Sarepta is currently the leading gene therapy player in the DMD space. Today, many AAV-based gene therapy medications are The Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. This button displays the currently selected search type. The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. 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.. Eventually, they will need ventilation to help them breathe. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. 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. Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. As per DelveInsight Duchenne Muscular Dystrophy Epidemiology Report, the total DMD prevalent population was more than 30K in the 7MM in 2020, which is further expected to increase by 2032. In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. These results have paved the way for ongoing human trials, which have shown a promising ability of this therapy to slow the progression of the disease. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. Once inside the cell, the viral vector behaves like a virus and makes the cell produce the protein encoded by the working gene it is carrying, compensating for the original mutated copy. This loss adds up to about 50 billion yen, or about $390 million (U.S.). Click for Index There are currently four companies who have DMD gene therapy products that have been given to boys with Duchenne, and three of the companies have ongoing studies in the USA. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. The mutated gene is on the X chromosome, making DMD an X-linked disease. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. Pfizer Inc. Website: www.pfizer.com. USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. The company is working with Roche for commercialization outside the country. March 29, 2006. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. First, the good news for Solid Biosciences: the new process put in place for its Duchenne muscular dystrophy gene therapy project SGT-001 looks safe. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. For example, Eteplirsen (Exondys 51) is expected to cost patients around US$ 300,000 for a treatment course and the cost of the treatment can go as high as US$ 750,000 annually. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. WebWhilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. Check out the MDAs Facebook Live Q&A event MDA Frontline COVID-19 Response: Back-to-School in the Midst of COVID-19 Concerns for the Neuromuscular Disease Community with Dr. Christopher Rosa and Justin Moy. Tune in live this Friday, July 31 at 3pm ET to join the discussion. The Food and Drug Administration approved the therapies after studying a few dozen boys. 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. Antibody status can be quite divisive in the DMD community.. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). But we were cautious after the high profile death of Jesse Gelsinger in 1999.. It has a pipeline of in vivo and ex vivo therapies. 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. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by involvement of the shoulder muscles. Gene therapy; Cell therapy; Drug therapy; Mutation specific approaches; TREAT-NMD Services Limited is a wholly owned subsidiary of TREAT-NMD Alliance Limited, a registered charity in England & The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. Moreover, a thorough clinical assessment, a complete patient history, and a number of specialist diagnostics, including molecular genetic tests, are used for Duchenne Muscular Dystrophy diagnosis. GlobalDatas Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). The company also has a collaboration with BioMarin. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Sarepta has three products marketed for the treatment of DMD: Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen). SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. EMDR (801) 436-5597. 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. 1985 - 2023 BioSpace.com. We are able to directly target the muscle cells. With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). Details >>, provide genotype and phenotype data from the same cell across thousands of single cells, 25 novel therapies set to shape the landscape of medicine in 2023, Genascence believes gene therapy can transform the treatment of knee osteoarthritis, Drug Discovery & Developments top stories of 2022. 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. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). The major goal is to demonstrate safety. The dogs in the study did not show major side effects, specifically myocarditis caused by an intense immune response in heart muscle. WebDr Paul Benson is an oral and facial surgeon, serial entrepreneur and business coach with a diverse portfolio of companies in a variety of industries including healthcare, beauty, 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. Most boys stop walking and need a wheelchair between 9 and 14 years old.. Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. 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. RGX-202 is intended to deliver a transgene encoding a novel microdystrophin with functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin. 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. 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. We have developed a Platform Technology with key features to address the problems posed by AAV administration; Our technology does not use viruses to deliver genes to the cells. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. NIH, U.S. National Library of Medicine, ClinicalTrials.gov. RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. Vyondys 53 (golodirsen) Injection. It also has a muscle-specific promoter, which is a DNA element that regulates the activity of a gene called MHCK7. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Its pipeline product includes SRP-5051, SRP-9001, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Recently Serepta released the topline results from Part 2 of Study SRP-9001-102 (Study 102), an ongoing, double-blind, randomized, placebo-controlled clinical study to assess the safety, efficacy, and tolerability of a single dose of SRP-9001 (delandistrogene moxeparvovec) in 41 patients with Duchenne Muscular Dystrophy, 21 of whom were in the placebo crossover cohort. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. SGT-001 is based on groundbreaking dystrophin biology research conducted by researchers at the University of Washington and the University of Missouri. SGT-001 has received Rare Pediatric Disease and Fast Track Designation in the United States and Orphan Drug Designation in the US and EU in 2017. A gene transfer therapy study to evaluate the safety and efficacy of SRP-9001 in participants with Duchenne Muscular Dystrophy (DMD) [NCT05096221]. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. SRP-9001 is also being studied in a randomized, placebo-controlled Phase II trial (Study 102) in 41 boys ages 4-7 years with results expected in early 2021. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) The company then opened U.S. enrollment for a Phase III trial of the therapy that was already underway in the U.K., Canada and other countries. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. Use tab to navigate through the menu items. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. WebAbstract. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells.