On the other hand, high cost of gene therapies restrains the growth to some extent. 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). The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. Focusing on developing therapeutics for disorders of the central nervous system. Life-threatening severe DMD complications may eventually develop, such as cardiomyopathy and respiratory difficulties. Focuses on allogeneic placental-derived cells. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Vyondys 53 (golodirsen) Injection. The clinical-stage regenerative medicine company specializes in using placental cells and proprietary, 3-D technology platform to develop cell therapies for inflammation, muscle injuries, hematological disorders and radiation exposure. Other hurdles of developing a DMD gene therapy. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. Founded in 2013, Editas Medicine is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies using CRISPR/Cas9 technology. They also have 12 other exon skipping-based genetic medicines in their pipeline. 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. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. For example, the dystrophin gene is too large to fit into the adeno-associated viruses, or AAVs, that are commonly used to deliver gene therapies. An impairment loss is when an asset depreciates in fair market value on the companys financial statements. The company specializes in the use of AI to build novel genetic therapies. 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. USA/Canada (Toll-Free): +1-800-792-5285, +1-503-894-6022. It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. This article is a part of Gene Therapy of Rare Genetic Diseases thematic issue. Credit: Shutterstock, Engineering Natural Killer Cells for Cancer Immunotherapy [Video], Targeting the untargetable and treating the untreatable, Neural networks overcome the setbacks of current computational drug discovery, Copyright 1999-2023 John Wiley & Sons, Inc. All rights reserved. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. All rights reserved. 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. Autolus specializes in developing CAR-T cell therapies. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Gene therapy; Cell In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. AAV-RPGR, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006, AAV-CNGB3, AAV-CNGA3, AAV-AIPL, A007, A008, A006. 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. Generation Bio has developed a platform with a ceDNA construct, ctLNP delivery system and scalable rapid enzymatic manufacturing process. It has a pipeline of in vivo and ex vivo therapies. It is developed based on exon skipping technology. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. The company aims to develop therapies for neurological disorders and other diseases. All Rights Reserved. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. According to Kornegay, We showed remarkable decline in loss of respiratory function.. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. Instead of delivering the dystrophin gene, GALGT2 delivers the GALGT2 gene, which is also important for muscle function. Corticosteroids help dampen down inflammation, said Hesterlee. It is usually observed between the ages of three and six. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Published: Nov 28, 2022 Importantly for Kornegay, the trial showed the treatment was safe. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. "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.". Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. This button displays the currently selected search type. The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. 6 min read. The collaboration could be worth more than $3 billion. 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. Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company raised $40 million in Series A funding in late 2017 and has attracted the likes of Merck & Co.'s Roger Perlmutter and the noted gene therapy 1. 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. 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. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. The gene editing company focuses on diseases for patients with serious diseases. Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. The companys most recent Phase Ib results were released in May at the ASGCT meeting (abstract no. Non-expression or very abnormal dystrophin expression causes the muscle fibers to weaken, resulting in accelerated destruction of the muscle tissue. As the name suggests, gene therapy involves delivering a healthy copy of a mutated gene (in DMDs case dystrophin) into cells. 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. The Mescope platform consists of an instrument and analysis computer, software, reagents and consumables. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. Currently these trials are taking place in the US. SRP-9001: One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. What is now called golden retriever muscular dystrophy (GRMD) turns out to be an excellent model for Duchenne muscular dystrophy because it involves the canine dystrophin gene, causes similar symptoms, and is also X-linked. 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). In late 2020, the company completed the acquisition of Asklepios Biopharmaceutical to bolster the unit. The DMD Gene Therapy Race Monday's BLA acceptance makes Roche and Sarepta the leaders of a tight race to bring a gene therapy for DMD over the regulatory In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. The biotech is developing novel cell and exosome-based therapeutics. Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. 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. Extensive pre-clinical evidence also formed part of the BLA. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. The next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. 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. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. Unlike more complex conditions, where several genes need repairing or replacing, single gene conditions are easier to rectify. Microdystrophin expression was seen via muscle biopsies 90 days after treatment (at a dose of 2E14 vg/kg), which stabilized dystrophin-associated proteins and restored activity of a key enzyme (called neuronal nitric oxide synthase, or nNOS) in the muscles. The He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. CAP-1002, exosome-based vaccine, engineered exosomes, CDC-exosomes, The startup Cellares aims to enable industrial-scale cell therapy manufacturing with its. ORLANDO, FloridaJeffrey Chamberlain, PhD, outlined the 4 different types of gene therapy for treating Duchenne muscular dystrophy (DMD) at the Gene Therapy and Gene Editing Symposium which took place on the second day of the CureDuchenne 2022 FUTURES National Conference . 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. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein involved in muscle strength. Pfizer Inc. Website: www.pfizer.com. Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. 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. eGenesis has a pipeline of gene therapies focused on inherited, systemic, debilitating chronic diseases. WebWhilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. These DMD therapies may, at best, slow the progression of Duchenne. The team has several theories as to why and Byrne believes the issue is solvable. Also, if you were to treat infants, its important to remember that they will be making new muscle cells without the modified gene in them, so there is a balance of when to treat.. 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. UCART123, UCART22, UCARTCS1, UCART19, ALLO-501, ALLO-715. There are two main types of gene therapy: somatic gene therapy and germline gene therapy. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne He had previously held managing editor roles on two of the companys medical device technology publications. 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. They finally found the perfect balance, naming the shortened genes microdystrophins.. NIH, U.S. National Library of Medicine, ClinicalTrials.gov. 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 Summer Zemp. It is difficult and costly to manufacture large quantities of AAV. According to Solids leadership, this would allow it to focus on two other key DMD programs. 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. When expanded it provides a list of search options that will switch the search inputs to match the current selection. The trials main purpose is to monitor changes in NSAA scores. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. 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). The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. omidubicel, GDA-20, GDA-301, GDA-401, GDA-501, GDA-601. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). The disease is universally fatal. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. 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. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). As an example, Dystrophin, the gene responsible for Duchenne muscular dystrophy (DMD) is 14 kb, meaning that only one-third of the dystrophin gene can be "packaged" into AAV. https://www.pharmalive.com/wp-content/uploads/2021/08/Mega-3-Billion-Deal-Shapes-Up-for-Roche-to-Target-AD-and-Parkinsons-BioSpace-8-24-21.jpeg, https://www.pharmalive.com/wp-content/uploads/2020/01/Pharmalive_4c-300x37.png, FDA accepts BLA for Roche-Sarepta's DMD gene therapy, Copyright - PharmaLive and Outcomes LLC |, Axsome headed to FDA after Phase III Alzheimers agitation win, Social Determinants of Health (SDOH): Three Trends to Watch in 2023, U.S. Centers for Disease Control and Prevention (CDC). 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. In addition, most patients calves appear enlarged. https://www.alliedmarketresearch.com/request-sample/2841. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." Email (801) 436-5597. SRP-9001 was safe and well-tolerated up to one-year post-administration. AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. Pfizer Inc. Website: www.pfizer.com. These results have paved the way for ongoing human trials, which have shown a promising ability of this therapy to slow the progression of the disease. 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He is currently providing CMC advice for Myosana. [This feature is a part of 2022s Pharma 50 series.]. This loss adds up to about 50 billion yen, or about $390 million (U.S.). Currently, Matthew is a Venture Partner at Medicxi. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. 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. This may lead to dangerous side effects. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The tricky part is getting the gene inside the cell. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. As the disease progresses the most affected individuals require a wheelchair by reaching adolescence. 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 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. Likelihood of Approval and Phase Transition Success Rate Mod IMT-009 by Immunitas Therapeutics for Squamous Non-Small Cell Lung Cancer: Likelihood of Approval, IMT-009 by Immunitas Therapeutics for Diffuse Large B-Cell Lymphoma: Likelihood of Approval, IMS-001 by Imstem Biotechnology for Relapsing Remitting Multiple Sclerosis (RRMS): Likelihood of Approval, IMS-001 by Imstem Biotechnology for Primary Progressive Multiple Sclerosis (PPMS): Likelihood of Approval. One component condenses the DNA of the gene and protects it from degradation. 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. Medical Design and Outsourcing. DMD has a life expectancy of 16 to early 20s. exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310. It also selectively licenses its NAV vectors to other biotechnology companies. Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. The companies are looking to extend this collaboration to identify potential underlying mechanisms for these toxicities. In this review, we highlight current opportunities for Duchenne muscular dystrophy gene therapy, which has been known thus far as an incurable genetic disease. They are currently developing gene therapies for a range of diseases, including Duchenne muscular dystrophy and hemophilia. PF-06939926 was granted Fast Track designation in 2020. We dont know exactly why the dog did not predict this severe adverse event, said Kornegay. Click for Index The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. Atara Biotherapeutics focuses on developing allogeneic T-cell immunotherapy for serious conditions such as solid tumors, hematologic cancers and autoimmune diseases. 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. 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. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. Sometimes called minidystrophins, there are slight variations between different versions of these shortened genes, but the key is they are all small enough to fit into AAV, explained Hesterlee. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. 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 GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). 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. Although the Phase I trial is not placebo controlled, they can compare treated children to the known natural history of DMD. Verified This is accomplished using a vector, usually a virus or nanoparticle, as a trojan horse to sneak the healthy gene into the cell. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics 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. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. The company is developing CRISPR/Cas9 genome editing technology. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. 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. 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. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. Powered by Madgex Job Board Software. The biotech has developed a multiplex gene editing and genome engineering platform for applications in solid organ and therapeutic cell transplantation. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome.