There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 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. 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. 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. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. LPC Intern, CMHC-I. 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. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. 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. Byrne and colleagues now had a therapeutic that would fit in the AAV. The approved DMD therapies received a positive response and helped the patients. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics Importantly, our platform is Not Restricted By Gene Size. For dogs receiving the treatment, there was an increase in microdystrophin protein in skeletal and heart muscle as well as the diaphragm. 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. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. The company has multiple cell therapy candidates in pre-clinical and clinical development. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. 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. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. 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 Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. That year, Bayer also acquired BlueRock Therapeutics. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. FDA accepts BLA for Roche-Sareptas DMD gene therapy. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. It has a pipeline of in vivo and ex vivo therapies. Powered by Madgex Job Board Software. 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. Extensive pre-clinical evidence also formed part of the BLA. It is developed based on exon skipping technology. They are currently developing gene therapies using CRISPR/Cas9 technology. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. The disease is universally fatal. They finally found the perfect balance, naming the shortened genes microdystrophins.. The Food and Drug Administration approved the therapies after studying a few dozen boys. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. 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. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. All three companies are using different versions of minidystrophin, explained Hesterlee. 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. 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 Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). Now, researchers had to find the best time during the course of the childrens disease to test the therapy. UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. This loss adds up to about 50 billion yen, or about $390 million (U.S.). Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. The company previously reported 1-year data for the same measures in March 2021. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. According to Kornegay, We showed remarkable decline in loss of respiratory function.. It is administered by intravascular and intramuscular routes. 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. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. 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. The company aims to develop therapies for neurological disorders and other diseases. These findings showed a significant improvement in patient-reported outcomes and provided encouraging evidence of functional benefit 1.5 years after treatment when compared to natural history data. 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). SRP-9001 (2E14 vg/kg dose) is currently being investigated in open-label Phase I/II study (Study 101). Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. July 6, 2022. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. "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. As a result, SRP-9001 would gain a competitive edge. Eventually, they will need ventilation to help them breathe. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. ARO-AAT, ARO-APOC3, ARO-ANG3, ARO-HSD, ARO-C3. exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310. Knowing your family history is the first step to understand and be proactive about your 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 Data are expected to start rolling in late next year. Verified The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. 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. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. 6 min read. Thats why the first DMD gene therapy trial in the US, which began in 2006, involved injecting the gene therapy directly into the biceps of the children who participated. The most common type of vector is a virus called adeno-associated virus (AAV), which works using the natural ability of viruses to enter cells and hijack cellular machinery to produce viral proteins encoded by viral genes. Gene therapy offers a potentially exciting treatment approach for patients with Duchenne Muscular Dystrophy. Data are expected to start rolling in late next year. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. WebWhilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. FDA Approves BeiGenes Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. 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. 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. 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. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. The companys NEXI-001 and NEXI-002 programs are in Phase 1/2 clinical studies for relapsed acute myeloid leukemia after allogeneic stem cell transplantation and multiple myeloma refractory to >3 prior lines of therapy, respectively. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. The mutated gene is on the X chromosome, making DMD an X-linked disease. 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). Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. 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. 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. 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. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. The trials main purpose is to monitor changes in NSAA scores. 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. 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. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. He is currently providing CMC advice for Myosana. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. We are able to directly target the muscle cells. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. 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