The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. 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). 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. The FDA hasacceptedRocheandSareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD). Four of those are for ocular indications while the other two are for a salivary gland condition and Parkinsons disease. The drug in question, GS-1811 (formerl AbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Published: Nov 28, 2022 The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. 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. Currently, Matthew is a Venture Partner at Medicxi. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. Sometimes called minidystrophins, there are slight variations between different versions of these shortened genes, but the key is they are all small enough to fit into AAV, explained Hesterlee. He had previously held managing editor roles on two of the companys medical device technology publications. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. As part of the FDA's accelerated approval pathway, Roche and Sarepta have also initiated the EMBARK trial, a global, randomized, double-blinded and placebo-controlled study of SRP-9001 in DMD patients aged 4 to 7 years old. Sarepta is headquartered in Cambridge, Massachusetts, the US. Currently these trials are taking place in the US. 1985 - 2023 BioSpace.com. The company raised $40 million in Series A funding in late 2017 and has attracted the likes of Merck & Co.'s Roger Perlmutter and the noted gene therapy For example, Eteplirsen (Exondys 51) is expected to cost patients around US$ 300,000 for a treatment course and the cost of the treatment can go as high as US$ 750,000 annually. Email (801) 436-5597. In addition, most patients calves appear enlarged. Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. Extensive pre-clinical evidence also formed part of the BLA. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. Krystal Biotech specializes in redosable gene therapy. The company sells a variety of instruments and consumables. The factors driving this growth are the newborn screening of DMD, increasing awareness programs, upcoming launches and approvals, and robust pipeline activity in the gene therapy for DMD. 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. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. 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). Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. We dont know exactly why the dog did not predict this severe adverse event, said Kornegay. "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 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. An IND application is expected by the end of 2021. At Qmed, he overhauled the brands news coverage and helped to grow the sites traffic volume dramatically. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the bodys immune responses to the protein expressed by their gene therapeutic. SRP-9001 was safe and well-tolerated up to one-year post-administration. The pharma and biotech editor of WTWH Media, Brian is a veteran journalist with more than 15 years of experience covering an array of life science topics, including clinical trials, drug discovery and development and medical devices. These DMD therapies may, at best, slow the progression of Duchenne. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. All rights reserved. Data are expected to start rolling in late next year. "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. 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. 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. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. The patients body will react to the viral vector just like it would any other virus, creating antibodies to hunt and destroy the gene therapy viruses. It is administered by intravascular and intramuscular routes. The NAV AAV8 vector, which has been used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12) are used in RGX-202 to support the delivery and targeted expression of genes throughout skeletal and heart muscle. 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. REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.. Antibody status can be quite divisive in the DMD community.. March 29, 2006. 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 company aims to develop therapies for neurological disorders and other diseases. The next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. The company is developing CRISPR/Cas9 genome editing technology. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. Pharmaceutical companies see the value too, with one company, Sarepta, expecting approval of a Duchenne muscular dystrophy gene therapy as early as June of this year. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? 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. Get industry leading news, data and analysis delivered to your inbox. Also working on a gene therapy for DMD is Solid Biosciences, which has also been having trouble. MedTech 100 is a financial index calculated using the BIG100 companies covered in Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. Founded in 1992, bluebird bio is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies for rare genetic diseases and cancer. 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. In January, The FDA approved Regenxbios request to conduct a Phase I/II clinical trial in the United States to assess the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne Muscular Dystrophy (DMD). Founded in 1995, Sangamo Therapeutics is a biotech company based in Richmond, California that focuses on developing gene therapies for rare genetic diseases and cancer. Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. Byrne and colleagues now had a therapeutic that would fit in the AAV. The therapeutic candidate acts by targeting Beta-1, 4 N-acetylgalactosaminyltransferase 2 (GALGT2). 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 company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). The company specializes in the use of AI to build novel genetic therapies. Pfizer Inc. Website: www.pfizer.com. 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. The company boasts that its AI Workbench can help manage the complexity in RNA biology while identifying novel targets. All three companies are using different versions of minidystrophin, explained Hesterlee. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. Focuses on developing novel gene therapies for rare inherited genetic diseases. Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. The companys single-course gene editing programs focus on conditions with a genetically driven, life-long and severely elevated LDL-C such as familial hypercholesterolemia (FH). We have developed other critical functions to ensure proper gene delivery. Knowing your family history is the first step to understand and be proactive about your Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. Feb 18, 2022 | Reading Time: 8 minutes. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle PF-06939926 was granted Fast Track designation in 2020. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. 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 . Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. 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. 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. 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. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. LPC Intern, CMHC-I. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. But there is a limit to how much cargo you can stuff inside these tiny viruses, about 5 kb for AAV. Pfizers PF-06939926 was designated as an Orphan Drug and Pediatric Rare Disease by the FDA in May 2017 and an Orphan Medicinal Product Designation by the EMA for the treatment of DMD. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. For example, the dystrophin gene is too large to fit into the adeno-associated viruses, or AAVs, that are commonly used to deliver gene therapies. Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. 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 The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. A gene transfer therapy study to evaluate the safety and efficacy of SRP-9001 in participants with Duchenne Muscular Dystrophy (DMD) [NCT05096221]. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The company previously reported 1-year data for the same measures in March 2021. Viruses are very well evolved to get into cells, commented Hesterlee. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. Microdystrophin expression was seen via muscle biopsies 90 days after treatment (at a dose of 2E14 vg/kg), which stabilized dystrophin-associated proteins and restored activity of a key enzyme (called neuronal nitric oxide synthase, or nNOS) in the muscles. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." Abeona aims to develop therapies for a variety of diseases, including Recessive Dystrophic Epidermolysis Bullosa and Sanfilippo Syndrome Type A. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. WebAbstract. In May 2022, four companies, Pfizer, Sarepta, Genethon and Solid Biosciences, were all observing serious side effects in their gene therapy clinical trials for DMD. 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. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. All functional improvement the boys gained (measured by the NorthStar Ambulatory Assessment (NSAA) rating scale) was also maintained for at least one year post-treatment. The biotech aims to engineer precision genetic medicine for rare diseases. 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