Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Importantly, our platform is Not Restricted By Gene Size. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. An IND application is expected by the end of 2021. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The first signs of DMD appear as the young boys begin to walk and get more mobile, typically between the ages of 2 to 5. AAV-based gene therapies for x-linked myotubular myopathy (XLMTM), Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. 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 The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry. The mutated gene is on the X chromosome, making DMD an X-linked disease. Once we identified the culprit gene, we thought Oh great! Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). Duchenne muscular dystrophy (DMD) is a rare, fatal The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. Focusing on developing therapeutics for disorders of the central nervous system. Specializes in developing next-generation AAV capsids for gene therapies. Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). Solid Biosciences therapy, called SGT-001, involves a microdystrophin gene carried by an AAV9 viral vector. July 6, 2022. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. 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). Increase in the prevalence of chronic disorders, rise in government support, and ethical acceptance of gene therapy for cancer treatment drive the growth of the global gene therapy market. 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. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock late last year after a treated patient died. 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 disease is universally fatal. 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. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. That worked great for small genes, but not so well for dystrophin.. Likelihood of Approval and Phase Transition Success Rate Mod IMT-009 by Immunitas Therapeutics for Squamous Non-Small Cell Lung Cancer: Likelihood of Approval, IMT-009 by Immunitas Therapeutics for Diffuse Large B-Cell Lymphoma: Likelihood of Approval, IMS-001 by Imstem Biotechnology for Relapsing Remitting Multiple Sclerosis (RRMS): Likelihood of Approval, IMS-001 by Imstem Biotechnology for Primary Progressive Multiple Sclerosis (PPMS): Likelihood of Approval. Vast improvements have been made in managing patients with DMD, but one stubborn 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 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). Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. All Rights Reserved. The Food and Drug Administration approved the therapies after studying a few dozen boys. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. Byrne and colleagues now had a therapeutic that would fit in the AAV. "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.". This type of gene therapy has the potential to pass the modified genes onto future generations, but it is still experimental and raises ethical concerns. 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. 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). AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. This loss adds up to about 50 billion yen, or about $390 million (U.S.). The company was spun-out from University College London in 2014. While Solid Biosciences SGT-001 and Regenxbios RGX-202 are in the early stage of development for DMD treatment. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). In addition, Brian covered the medical device sector for 10 years at UBM. 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, That year, Bayer also acquired BlueRock Therapeutics. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. Summer Zemp. That allowed researchers to test the gene therapy proof-of-concept in DMD patients without worrying about systemic administration right off the bat. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. All rights reserved. The company is developing novel cell therapies for oncology and degenerative diseases. Now, researchers had to find the best time during the course of the childrens disease to test the therapy. "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. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by involvement of the shoulder muscles. A fifth company plans to begin dosing in 2023. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. 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. Importantly, there were no serious adverse events (only mild to moderate events). 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. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. 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. 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. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Duchenne Muscular Dystrophy is caused by a dystrophin gene mutation (a protein in the muscles). It employs a non-lethal modified virus (AAVrh74) with a high affinity for muscle tissue, allowing for targeted delivery. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. Could we use histamine? They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. 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. USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." According to Kornegay, We showed remarkable decline in loss of respiratory function.. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an All rights reserved. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. Credit: Shutterstock, Engineering Natural Killer Cells for Cancer Immunotherapy [Video], Targeting the untargetable and treating the untreatable, Neural networks overcome the setbacks of current computational drug discovery, Copyright 1999-2023 John Wiley & Sons, Inc. All rights reserved. WebDuchenne muscular dystrophy is the result of a mutation or deletion of the gene encoding dystrophin, located on the X chromosome. Email (801) 436-5597. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. 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. 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.. The companys allogeneic CAR-T program targets B-cell malignancies. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. 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. solutions for life science vertical and offering quintessential advisory services in the SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. Myosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure to improve the quality of life, increase longevity and reduce the disease burden of Duchenne muscular dystrophy (DMD) and other neuromuscular diseases. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. 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. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. Gene therapy is an umbrella term for a range of therapies that target the genetic underpinnings of disease. They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. Feb 18, 2022 | Reading Time: 8 minutes. 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. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. We have developed several monoclonal antibodies against a specific muscle target protein that is present in both skeletal and cardiac muscle. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. 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. Cellectis has more than two decades of experience in gene editing. The two nucleases give it access to a variety of genetic mutations and develop targeted and durable gene edited medicines. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. 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. As part of the FDAs 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. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. Despite all the challenges faced over the years, there are a handful of gene therapies being developed for DMD currently, with a few pivotal Phase III trials close on the horizon. 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. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. The collaboration could be worth more than $3 billion. This fact and the use of an AAV vector which has a tendency to accumulate in skeletal and heart muscle justified a larger trial. Both employ exon skipping, redirecting DNA processing inside the muscle cells to create minidystrophin right in the cells much like the researchers did in the lab, but directly in the children themselves. Buy the report here. 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. 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. 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. Data are expected to start rolling in late next year. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. Published: Jul 29, 2020 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. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. 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. 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. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. Data are expected to start rolling in late next year. WebDMD gene therapy aims to deliver a working version of the dystrophin gene, so that the body can produce functioning dystrophin. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. It is administered by intravascular and intramuscular routes. These DMD therapies may, at best, slow the progression of Duchenne. Sarepta is headquartered in Cambridge, Massachusetts, the US. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. 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. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. One component condenses the DNA of the gene and protects it from degradation. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. Proudly created with Wix.com. Within the context of Duchenne and other rare diseases, its a very robust sample size and one that will grow with data from EMBARK.. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). The disease is universally fatal. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Despite this progress, most DMD patients pass away in their 20s to 30s due to respiratory failure, infection, or cardiomyopathy (dilation of the heart due to overwork). It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. BioSpace sat down with Sharon Hesterlee, Ph.D., chief research officer at the Muscular Dystrophy Association (MDA), to talk about the history and challenges of developing gene therapy for DMD and the DMD gene therapy field as a whole, including Pfizers and Sarepta Therapeutics latest clinical data. Gene expression and no serious adverse events ( only mild to moderate )... 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