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. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Sarepta is a market leader in this category, with three out of every five marketed therapies in the US market addressing DMD. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Focusing on developing therapeutics for disorders of the central nervous system. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. 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. 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). 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. Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. These micro-dystrophins might provide only partial improvement of muscle function. (2022, January 10). Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. 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. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The company sells a variety of instruments and consumables. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. Terry Horgan, the primary patient in an N-of-1 clinical trial evaluating a CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has died, according to an announcement from Cure Rare Disease, the nonprofit biotech sponsoring the trial. Unfortunately, their Phase I/II trial (IGNITE DMD) is still on hold by the FDA. Which Countries Top the Chart in Global Pharmaceutical Market? Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. 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. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. Subsequent gene therapy trials have moved to intravenous (IV) administration typically only requiring one fairly quick dose. 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 Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. A Range of Possible Mechanisms. 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. The company aims to develop therapies for neurological disorders and other diseases. The team has several theories as to why and Byrne believes the issue is solvable. The company is working with Roche for commercialization outside the country. The company has scored approvals for several RNAi therapeutics. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. 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, The company also has a collaboration with BioMarin. In this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. Today, many AAV-based gene therapy medications are The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. 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). Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. On the other hand, high cost of gene therapies restrains the growth to some extent. areas The company has a variety of gene and cell therapy programs in the clinic and preclinical programs in genome engineering and off-the-shelf cell therapy. 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. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. 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. 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. Horgan is the brother of Cure Rare Disease founder Rich Horgan, This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. The Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. 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. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. Pharma50: 50 Leading Cell and gene therapy companies. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. 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. EDIT-101, EDIT-103, EDIT-102, EDIT-202, EDIT-301. "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.". CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. 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. This is based on a proprietary algorithm built from the drugs sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalDatas Pharmaceutical Intelligence Center. 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. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. All three companies are using different versions of minidystrophin, explained Hesterlee. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. There are two main types of gene therapy: somatic gene therapy and germline gene therapy. Use tab to navigate through the menu items. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. 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. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. Pfizer is also conducting a Phase III study of the same product, which is being developed globally. 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. [This feature is a part of 2022s Pharma 50 series.]. 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. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Now, researchers had to find the best time during the course of the childrens disease to test the therapy. Muscle weakness and atrophy spread from the trunk and forearms to other muscles throughout the body as the disease advances. Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate why they were all being tripped up by serious safety concerns. It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. Several gene therapy approaches are being explored as treatments for Duchenne muscular dystrophy (DMD). 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. Data are expected to start rolling in late next year. 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. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. July 6, 2022. The company was spun-out from University College London in 2014. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. 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. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. Duchenne Muscular Dystrophy is the most common type of muscular dystrophy.
SRP-9001 (2E14 vg/kg dose) is currently being investigated in open-label Phase I/II study (Study 101). 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. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure. Importantly, our platform is Not Restricted By Gene Size. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. The FDA soon put the study under clinical hold, which it thenliftedearlier this year after the company had addressed the agency's concerns. For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran). The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. The disease is universally fatal. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. In September 2021, Astellas Pharma reported a fourth patient death in the AT132 clinical Whereas Becker Muscular Dystrophy has a longer life expectancy, usually in their 30s. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. The
Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. You can compare outcomes to natural disease due to a rich natural history of DMD. It also has a muscle-specific promoter, which is a DNA element that regulates the activity of a gene called MHCK7. Without this protein, males with Duchenne muscular dystrophy lose muscle cells to damage as they age. 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. He is currently the Scientific Founder and SAB Chairman of Bolden Therapeutics. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. 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. The FDA soon put the Phase Ib study under clinical hold. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. 617). 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. Gene therapy replaces the mutated gene with a copy using whats called a vector to bring a working copy of the gene into a cell. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Allied Market Research (AMR) is a full-service market research and business-consulting wing of Allied Analytics LLP based in Portland, Oregon. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. The companys core focus areas include immuno-oncology and plant sciences. It is currently being investigated in a Phase I/II study in six boys ages 4 and up. Byrne and colleagues now had a therapeutic that would fit in the AAV. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. 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