They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. The drug in question, GS-1811 (formerl AbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. 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). With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. Also working on a gene therapy for DMD is Solid Biosciences, which has also been having trouble. 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. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. All rights reserved. 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. The company is developing CRISPR/Cas9 genome editing technology. 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. The disease is universally fatal. 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. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. 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. 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. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. The company also has a collaboration with BioMarin. In preclinical studies, the presence of the CT domain was shown to recruit several key proteins to the muscle cell membrane, resulting in improved muscle resistance to contraction-induced muscle damage in dystrophic mice. In May, Pfizer, Sarepta, Solid and Genethonjoined armsto investigate their mutual experience with serious safety concerns. It is usually observed between the ages of three and six. 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. These DMD therapies may, at best, slow the progression of Duchenne. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media Privacy Policy | Advertising | About Us. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. We dont know exactly why the dog did not predict this severe adverse event, said Kornegay. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. SRP-9001 (2E14 vg/kg dose) is currently being investigated in open-label Phase I/II study (Study 101). 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. Graphite Bio is building on CRISPR technology and working with the cells natural DNA repair processes to rewrite genes. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. Vyondys 53 (golodirsen) Injection. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Corticosteroids help dampen down inflammation, said Hesterlee. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. It is developed based on exon skipping technology. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The patients body will react to the viral vector just like it would any other virus, creating antibodies to hunt and destroy the gene therapy viruses. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. EMDR (801) 436-5597. DMD starts to show its effects during early childhood. 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. 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. Pharma50: 50 Leading Cell and gene therapy companies. 617). Published: Jul 29, 2020 Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.. 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, they will need ventilation to help them breathe. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. They also have 12 other exon skipping-based genetic medicines in their pipeline. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. omidubicel, GDA-20, GDA-301, GDA-401, GDA-501, GDA-601. 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). Cell and gene therapies promise to enable broad changes in the healthcare system over the next decade, prompting a growing number of cell and gene therapy companies to join the space. Horgan is the brother of Cure Rare Disease founder Rich Horgan, These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. They can help slow down disease progression, but tackling inflammation only addresses one downstream effect.. One component condenses the DNA of the gene and protects it from degradation. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. Allied Market Research (AMR) is a full-service market research and business-consulting wing of Allied Analytics LLP based in Portland, Oregon. The Food and Drug Administration approved the therapies after studying a few dozen boys. The goal of gene therapy is to replace or repair a missing or faulty gene, introduce a new gene to help fight disease, or deactivate a harmful gene. 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. WebHigh cost of Duchenne muscular dystrophy treatment. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. Feb 18, 2022 | Reading Time: 8 minutes. The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. Published: Nov 28, 2022 Autolus specializes in developing CAR-T cell therapies. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. 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. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. The He is currently the Scientific Founder and SAB Chairman of Bolden Therapeutics. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. 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. They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. 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. PF-06939926 is among the two gene therapies in late-stage development for DMD, with Sarepta Therapeutics SRP-9001 serving as its main competitor. The leading site for news and procurement in the pharmaceutical industry. Focusing on developing therapeutics for disorders of the central nervous system. The company is working with Roche for commercialization outside the country. Sarepta and Pfizer are evaluating their lead candidates for gene therapy in the late stages. 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. Importantly for Kornegay, the trial showed the treatment was safe. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. The platform supports the engineering of almost all cell types, including human primary cells and with any molecule. Cellectis has more than two decades of experience in gene editing. 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 company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. 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. The companys allogeneic CAR-T program targets B-cell malignancies. An IND application is expected by the end of 2021. 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. The company is using a stem cell biology and genomics platform to develop a novel autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy for Parkinsons Disease. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. PF-06939926was granted Fast Track designation in 2020. areas 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. The company has multiple cell therapy candidates in pre-clinical and clinical development. Connect with him on LinkedIn or email at [emailprotected], Copyright 2023 WTWH Media LLC. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. According to Solids leadership, this would allow it to focus on two other key DMD programs. The most troublesome symptoms are breathing difficulties. The company has scored approvals for several RNAi therapeutics. Its important to realize that the major goal of an animal study is not necessarily to show efficacy, he said. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. 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. Focuses on clinical-stage gene therapy. In September 2021, the company announced. For this next step, Byrne teamed up with Joe Kornegay, now retired, at the Texas A&M University College of Veterinary and Biomedical Sciences. He has extensive research experience in DMD. It is administered by intravascular and intramuscular routes. Buy the report here. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. That worked great for small genes, but not so well for dystrophin.. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Sarepta Therapeutics has two DMD gene therapies, SRP-9001 Micro-dystrophin and GALGT2 (Nationwide Childrens), in clinical trials and one therapy, GNT0004 Micro-dystrophin (Genethon), in preclinical development. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). 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. The NAV AAV8 vector, which has been used in numerous clinical trials, and a well-characterized muscle-specific promoter (Spc5-12) are used in RGX-202 to support the delivery and targeted expression of genes throughout skeletal and heart muscle. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. This is why many Duchenne drug studies traditionally havent involved children younger than 7 years old. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. The companys multi-omics approach supports the development of cell and gene therapies. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. After almost 15 years since the first gene therapy trial for Duchenne muscular dystrophy (DMD) began, the dream of a DMD gene therapy drug is getting closer to a reality. 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. The United States accounted for approximately 16K prevalent cases of DMD in 2020 which was the maximum in the 7MM. 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. Pfizer Inc. Website: www.pfizer.com. He had previously held managing editor roles on two of the companys medical device technology publications. On the other hand, high cost of gene therapies restrains the growth to some extent. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. Gene therapy; Cell The tricky part is getting the gene inside the cell. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. A fifth company plans to begin dosing in 2023. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be 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 next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. 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. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Use tab to navigate through the menu items. 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. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. 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. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. The FDA has ordered a clinical halt to the trial, and Pfizer is investigating the causes of death. Each of these gene therapies has slight variations in their three main components: the transgene, the Most boys stop walking and need a wheelchair between 9 and 14 years old.. Many researchers and companies are looking into alternate delivery systems that could transfer the dystrophin gene to the body without using AAVs. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. It is usually observed between the ages of three and six. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. SRP-9001 is also being studied in a randomized, placebo-controlled Phase II trial (Study 102) in 41 boys ages 4-7 years with results expected in early 2021. Today, many AAV-based gene therapy medications are The company sells a variety of instruments and consumables. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran). DMD is a progressive muscle wasting disease caused by a genetic mutation. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. 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). 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 Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. AAV9 is a type of AAV that is particularly good at getting into muscle cells. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. Without dystrophin, the muscle cells suffer from microtears, leading to their demise and progressive muscle wasting. 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. 6 min read. Viruses are very well evolved to get into cells, commented Hesterlee. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. Reference: Barry Byrne, Joe Kornegay, et al., Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Science Translational Medicine (2023), DOI: 10.1126/scitranslmed.abo1815, Feature image: The protein dystrophin. 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). GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. 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