The 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 this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. One of those labs, Louis Kunkels lab, identified the dystrophin gene first in 1986.. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. 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. The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. 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. 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. 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. Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. 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. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. 2020 by Myosana Therapeutics, Inc.. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. WebAbstract. This button displays the currently selected search type. 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.. 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. 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. We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. 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. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). WebDuchenne muscular dystrophy is the result of a mutation or deletion of the gene encoding dystrophin, located on the X chromosome. 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. For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. 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. DelveInsight is a Business Consulting and Market research company, providing expert business 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. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. The company sells a variety of instruments and consumables. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. 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.. Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). When expanded it provides a list of search options that will switch the search inputs to match the current selection. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). 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 companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. 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. Summer Zemp. Roughly 1 in 5000 males are born with this condition and there is currently no cure, with the median age of survival 23 years. All rights reserved. WebThe Roche Groups bold commitment to gene therapy collaborations across the organisation and industry are a stake in the ground: the possibility and potential of using Allied Market Research provides global enterprises as well as medium and small businesses with unmatched quality of Market Research Reports and Business Intelligence Solutions. AMR has a targeted view to provide business insights and consulting to assist its clients to make strategic business decisions and achieve sustainable growth in their respective market domain. Contact information and locations are not yet available, but initial trial sites are expected to open in the United States, with sites in Canada and Europe to follow. He has extensive research experience in DMD. In addition, Brian covered the medical device sector for 10 years at UBM. James is an expert in CMC drug development, with decades of experience in the Pharmaceutical 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. Importantly for Kornegay, the trial showed the treatment was safe. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. The FDA hasacceptedSarepta'sBiologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne Muscular Dystrophy (DMD). Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. All three companies are using different versions of minidystrophin, explained Hesterlee. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. Proudly created with Wix.com. Get industry leading news, data and analysis delivered to your inbox. They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. Five pharmaceutical companies, namely Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio, are currently working on gene therapy for Duchenne Muscular Dystrophy. Rare Daily Staff. Based in California, Audentes Therapeutics is a biotechnology company that employs gene therapy technology to develop treatments for people with rare muscle The DMD Gene Therapy Race Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. Currently these trials are taking place in the US. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. Which Countries Top the Chart in Global Pharmaceutical Market? The company also has a collaboration with BioMarin. 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. They can help slow down disease progression, but tackling inflammation only addresses one downstream effect.. Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. 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. These micro-dystrophins might provide only partial improvement of muscle function. Krystal Biotech specializes in redosable gene therapy. The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. Focuses on clinical-stage gene therapy. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Clinical researchers at UC Davis Health are using a gene therapy approach for Duchenne muscular dystrophy (DMD), the rare genetic disease that mainly occurs in 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. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. 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. Dystrophin, the largest gene in the human body, encodes a muscle protein responsible for keeping muscle cells from pulling themselves apart when the muscle is working, like a shock absorber for the cell, as Hesterlee described. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Published: Jul 29, 2020 The patient was a part of the studys non-ambulatory arm. By Tristan Manalac. Powered by Madgex Job Board Software. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Without dystrophin, the muscle cells suffer from microtears, leading to their demise and progressive muscle wasting. Currently, Matthew is a Venture Partner at Medicxi. Pharma50: 50 Leading Cell and gene therapy companies. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock late last year after a treated patient died. 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). 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 next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. On the other hand, high cost of gene therapies restrains the growth to some extent. Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. It is a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a shortened version of the human dystrophin gene (mini-dystrophin) controlled by a human muscle specific promotor. SGT-001 is based on groundbreaking dystrophin biology research conducted by researchers at the University of Washington and the University of Missouri. 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. 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. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. 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. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy. But there is a limit to how much cargo you can stuff inside these tiny viruses, about 5 kb for AAV. The boys motor functions rapidly deteriorate with disease progression and, by only 10 to 15 years of age, many boys with DMD are wheelchair-bound. Within the context of Duchenne and other rare diseases, its a very robust sample size and one that will grow with data from EMBARK.. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne 6 min read. In September 2021, the company announced. The disease is universally fatal. Focusing on developing therapeutics for disorders of the central nervous system. The company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. 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. The clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. The leading site for news and procurement in the pharmaceutical industry. It employs a non-lethal modified virus (AAVrh74) with a high affinity for muscle tissue, allowing for targeted delivery. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. Gene therapy; Cell 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. 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. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. That year, Bayer also acquired BlueRock Therapeutics. The disease is universally fatal. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. We dont know exactly why the dog did not predict this severe adverse event, said Kornegay. Now that the dystrophy gene was brought down to a useful size, the next challenge researchers faced was getting the gene therapy from the blood stream into the muscle. PF-06939926was granted Fast Track designation in 2020. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. 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 addition, RNA has potential in treating brain cancer, asMass General Brighamobserves in its list of one dozen potentially disruptive cell and gene therapy innovations. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. Muscle weakness and atrophy spread from the trunk and forearms to other muscles throughout the body as the disease advances. Sarepta is currently the leading gene therapy player in the DMD space. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. Throughout the late 1990s and early 2000s, researchers tinkered with the dystrophin gene, figuring out what parts were needed and how much they could trim out to still have a functional protein. 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. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. In addition, most patients calves appear enlarged. The approved DMD therapies received a positive response and helped the patients. According to Byrne, these results were used to justify an application for human trials, which are now ongoing. 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. The two nucleases give it access to a variety of genetic mutations and develop targeted and durable gene edited medicines. Eventually, they will need ventilation to help them breathe. Importantly, there were no serious adverse events (only mild to moderate events). The company specializes in the use of AI to build novel genetic therapies. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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). 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. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. 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Viruses are very well evolved to get into cells, commented Hesterlee. 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. REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. 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. His innovative Muscle-Targeted, Non-Viral platform has the potential to provide a novel gene therapy treatment for DMD a wide-range of other neuromuscular and cardiac disorders. 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