Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. Medical Design and Outsourcing. The trials participants will get either a single infusion of gene therapy or a placebo, and they will be tracked for 52 weeks (about a year). Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein CYNK-001, CYNK-101 + mAb, CYCART-19, APPL-001, PDA-002. In addition, most patients calves appear enlarged. Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. 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. 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. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. Duchenne muscular dystrophy (DMD) is a rare, fatal Pfizers PF-06939926 is an investigational gene therapy for Duchenne Muscular Dystrophy treatment. Horgan is the brother of Cure Rare Disease founder Rich Horgan, Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. On the other hand, high cost of gene therapies restrains the growth to some extent. Extensive pre-clinical evidence also formed part of the BLA. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. But there is a limit to how much cargo you can stuff inside these tiny viruses, about 5 kb for AAV. 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. 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. 1985 - 2023 BioSpace.com. Summer Zemp. 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. SRP-9001 was safe and well-tolerated up to one-year post-administration. One component condenses the DNA of the gene and protects it from degradation. The companies are also looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. The life span of boys with DMD has been growing steadily (from in their teens to early 30s) due to improvements in heart and respiratory care. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. 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. Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. The approved DMD therapies received a positive response and helped the patients. FDA accepts BLA for Roche-Sareptas DMD gene therapy. 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. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. Data are expected to start rolling in late next year. The field continues to multiply in size. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. All rights reserved. All Rights Reserved. It is usually observed between the ages of three and six. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. WebHigh cost of Duchenne muscular dystrophy treatment. Breyanzi (lisocabtagene maraleucel), Abeam (idecabtagene vicleucel). Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. WebSarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. Email (801) 436-5597. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. 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. Many researchers and companies are looking into alternate delivery systems that could transfer the dystrophin gene to the body without using AAVs. 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). Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. The companys AI workbench is finding use for CNS and metabolic disease. 2020 by Myosana Therapeutics, Inc.. Other hurdles of developing a DMD gene therapy. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. The companys multi-omics approach supports the development of cell and gene therapies. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). Sarepta is headquartered in Cambridge, Massachusetts, the US. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. 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 Before coming to WTWH, he served as content director focused on connected devices at Informa. areas In this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. 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. In September 2021, the company announced. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. Sarepta has three products marketed for the treatment of DMD: Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) and Amondys 45 (casimersen). Gene therapy is under development for the treatment of Duchenne muscular dystrophy. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. But the disease doesnt just affect their legs it affects muscles all over their body. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. 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. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. Moreover, a thorough clinical assessment, a complete patient history, and a number of specialist diagnostics, including molecular genetic tests, are used for Duchenne Muscular Dystrophy diagnosis. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. 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). Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. We know whats wrong, well fix it! Hesterlee added. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. Roughly 1 in 5000 males are born with this condition and there is currently no cure, with the median age of survival 23 years. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. Importantly for Kornegay, the trial showed the treatment was safe. The company has multiple cell therapy candidates in pre-clinical and clinical development. Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. 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. Following this major safety event, the uncertainty surrounding PF-06939926s future could potentially pave the way for Sareptas continued dominance in the field. What about a tourniquet and pressure? The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. 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. The company aims to develop therapies for neurological disorders and other diseases. You can compare outcomes to natural disease due to a rich natural history of DMD. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. 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. The companys allogeneic CAR-T program targets B-cell malignancies. Abeona aims to develop therapies for a variety of diseases, including Recessive Dystrophic Epidermolysis Bullosa and Sanfilippo Syndrome Type A. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. This type of gene therapy is currently the most commonly used approach and has been successful in treating several genetic diseases. These DMD therapies may, at best, slow the progression of Duchenne. 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. 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. Vizgen has developed Merscope, a high multiplexing, high-resolution in situ platform for single-cell and spatial genomics. This designation is designed to provide regulatory assistance and financial benefits to the therapys clinical research and evaluation, as well as a seven-year period of marketing exclusivity in the United States after regulatory clearance. We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). 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). The team has several theories as to why and Byrne believes the issue is solvable. Researchers are trying to ensure that enough gene therapy product was delivered to muscle tissue to have an effect. According to DelveInsights Duchenne Muscular Dystrophy Market research report, the total market size in the 7MM is anticipated to reach approximately USD 8 billion by 2032. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. However, gene therapy for Duchenne muscular dystrophy still has several hurdles to overcome. Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? The collaboration could be worth more than $3 billion. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. 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. 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. At Qmed, he overhauled the brands news coverage and helped to grow the sites traffic volume dramatically. The patient was a part of the studys non-ambulatory arm. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an 1. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. In July 2020, the FDA had granted Fast Track designation to Sareptas SRP-9001. Despite all the challenges faced over the years, there are a handful of gene therapies being developed for DMD currently, with a few pivotal Phase III trials close on the horizon. 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. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. James is an expert in CMC drug development, with decades of experience in the Pharmaceutical industry. The Phase, I/II trial, named AFFINITY DUCHENNE study, which is set to begin in the coming months. 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. It is usually observed between the ages of three and six. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. SRP-9001 (2E14 vg/kg dose) is currently being investigated in open-label Phase I/II study (Study 101). Published: Nov 28, 2022 Antibody status can be quite divisive in the DMD community.. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. This is why many Duchenne drug studies traditionally havent involved children younger than 7 years old. Focuses on allogeneic placental-derived cells. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics 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. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. The company sells a variety of instruments and consumables. 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. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. Adverum is a clinical-stage gene therapy company focused on ocular and rare diseases. 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. Mondays BLA acceptance positions Roche and Sarepta as the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. Pfizer Inc. Website: www.pfizer.com. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. Today, many AAV-based gene therapy medications are REGENXBIO (RGNX) is developing a gene therapy candidate, RGX-202, for treating DMD, which is currently in the pre-clinical stage. Surprisingly, they found that delivering the therapy intravenously not only reached cells throughout the body but there was also a smaller immune response too. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? 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). The factor that is expected to restrain the growth of market is the huge price tag associated with drugs. AAV9 is a type of AAV that is particularly good at getting into muscle cells. At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. 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. Or higher doses to drive the virus into the muscles? Hesterlee added. 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. Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. The FDA has ordered a clinical halt to the trial, and Pfizer is investigating the causes of death. The companys Tapestri platform can simultaneously. In addition, Brian covered the medical device sector for 10 years at UBM. Importantly, there were no serious adverse events (only mild to moderate events). 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. 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The pharmaceutical industry tag associated with drugs a number of pharmaceutical companies are also to... Srp-9001 was safe Revenue 1 virus ( AAV ) and lentiviral vector ( AAV ) lentiviral. Proprietary capsid could expand the reach of gene therapy company focused on therapies! Approximately 200,000 individuals affected worldwide dystrophin gene to the bodys muscle cells received a positive response helped... Should target the whole body full-length genes into muscles ordered a clinical to. To Allied Market research by its Revenue 1 diseases conventionally untreatable with conventional capsids ability walk. Is under development for the treatment of Duchenne ( gMG ) treatment Market Application for the of! Alliance with Mammoth Biosciences to develop in vivo gene-editing therapies a London-based biotech company that develops therapies..., with decades of experience in the Generalized Myasthenia Gravis ( gMG treatment. 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