PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. Specializes in developing next-generation AAV capsids for gene therapies. 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. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). 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. 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. 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 & Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. What about a tourniquet and pressure? Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). 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. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. 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. 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. 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. UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. 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 WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. 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. 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. The companys core focus areas include immuno-oncology and plant sciences. Sarepta and its partner Roche presented new results and analyses on their experimental gene therapy SRP-9001 for the neuromuscular condition Duchenne muscular dystrophy showed consistent, statistically significant functional benefits in individuals. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." Pfizer Inc. Website: www.pfizer.com. The disease is universally fatal. It also selectively licenses its NAV vectors to other biotechnology companies. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, 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. solutions for life science vertical and offering quintessential advisory services in the 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. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Sarepta is currently the leading gene therapy player in the DMD space. [This feature is a part of 2022s Pharma 50 series.]. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne 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). 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). Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. The field continues to multiply in size. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. March 29, 2006. 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. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. Summer Zemp. This fact and the use of an AAV vector which has a tendency to accumulate in skeletal and heart muscle justified a larger trial. These DMD therapies may, at best, slow the progression of Duchenne. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. 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. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. 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. The disease is universally fatal. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic 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. 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. 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. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. 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. A gene transfer therapy study to evaluate the safety and efficacy of SRP-9001 in participants with Duchenne Muscular Dystrophy (DMD) [NCT05096221]. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. 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. By Tristan Manalac. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. They also have 12 other exon skipping-based genetic medicines in their pipeline. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. The companys platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. Byrne and colleagues now had a therapeutic that would fit in the AAV. Knowing your family history is the first step to understand and be proactive about your The team has several theories as to why and Byrne believes the issue is solvable. Feb 18, 2022 | Reading Time: 8 minutes. 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. 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. Today, many AAV-based gene therapy medications are Importantly, our platform is Not Restricted By Gene Size. This may lead to dangerous side effects. WebAbstract. Could we use histamine? Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. 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 Once we identified the culprit gene, we thought Oh great! Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. The Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. Duchenne Muscular Dystrophy is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Founded in 1998, uniQure is a Dutch biotech company that develops gene therapies for a variety of diseases, including hemophilia, Huntington's disease, and congestive heart failure. Autolus specializes in developing CAR-T cell therapies. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. 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). Each of these gene therapies has slight variations in their three main components: the transgene, the Cellectis has more than two decades of experience in gene editing. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 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. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. A fifth company plans to begin dosing in 2023. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. DelveInsight is a Business Consulting and Market research company, providing expert business The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. According to Solids leadership, this would allow it to focus on two other key DMD programs. He is currently a Professor of Physiology and Biophysics at the University of Washington. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. Duchenne muscular dystrophy (DMD) is a rare, fatal As a result, SRP-9001 would gain a competitive edge. In September 2021, the company announced. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 WebDMD gene therapy aims to deliver a working version of the dystrophin gene, so that the body can produce functioning dystrophin. 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. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics While AAV vectors work great for delivering gene therapies to muscle cells, as Barry Byrne, co-author of the new study and professor of pediatrics at the University of Florida, explained, they have a size limitation. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. Louise Rodino-Klapac, CSO, executive VP and head of R&D, Sarepta Permission granted by Sarepta If approved, SRP-9001, would be the first gene therapy for the muscular degenerative disease known as DMD and is slated for complete evaluation under the accelerated approval path by the end of May 2023. Also working on a gene therapy for DMD is Solid Biosciences, which has also been having trouble. Focuses on allogeneic placental-derived cells. 6 min read. 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. 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. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. The company is working with Roche for commercialization outside the country. This is why many Duchenne drug studies traditionally havent involved children younger than 7 years old. In addition, Brian covered the medical device sector for 10 years at UBM. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. 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. Genetically, DMD is due to null mutation of the dystrophin gene, one of the largest genes in the genome. Eventually, they will need ventilation to help them breathe. 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. The gene editing company focuses on diseases for patients with serious diseases. Gene therapy is more efficient and covers everyone, regardless of genetic mutations, but its still good to have options while new therapies are in development.. This loss adds up to about 50 billion yen, or about $390 million (U.S.). The company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the body's immune responses to the protein expressed by their gene therapeutic. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. Among the EU5 countries, the UK had the highest prevalent population of DMD with more than 2K cases, while Spain had the lowest DMD cases in 2020. 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. Which Countries Top the Chart in Global Pharmaceutical Market? The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. According to Kornegay, We showed remarkable decline in loss of respiratory function.. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response.