DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. The participants who were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring. 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. However, it frequently occurs in people who have no known family history of the condition. The boys motor functions rapidly deteriorate with disease progression and, by only 10 to 15 years of age, many boys with DMD are wheelchair-bound. The mutated gene is on the X chromosome, making DMD an X-linked disease. Importantly, our platform is Not Restricted By Gene Size. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. SRP-9001: 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. But we were cautious after the high profile death of Jesse Gelsinger in 1999.. Duchenne UK and the DMD Hub wanted to understand what more can be done to encourage them to be They finally found the perfect balance, naming the shortened genes microdystrophins.. Antibody status can be quite divisive in the DMD community.. The regenerative medicine company is focused on developing therapies for inflammatory ailments, cardiovascular disease and back pain. Rare Daily Staff. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. NTLA-2001, NTLA-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001. 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. 1. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle. "The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne," Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. Tabelecleucel (tab-cel), ATA188, ATA2271/ATA3271. 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, 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). The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 Many researchers and companies are looking into alternate delivery systems that could transfer the dystrophin gene to the body without using AAVs. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. The tricky part is getting the gene inside the cell. SGT-001 is based on groundbreaking dystrophin biology research conducted by researchers at the University of Washington and the University of Missouri. By Tristan Manalac. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. A number of companies are now testing their approaches in the clinic. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. solutions for life science vertical and offering quintessential advisory services in the 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. WebThe Roche Groups bold commitment to gene therapy collaborations across the organisation and industry are a stake in the ground: the possibility and potential of using MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Importantly for Kornegay, the trial showed the treatment was safe. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. 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). Today, many AAV-based gene therapy medications are With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. 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. One component condenses the DNA of the gene and protects it from degradation. 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. Testing the children when they are starting to lose the ability to walk can avoid the natural history noise, Hesterlee added. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. 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. AAV9 is a type of AAV that is particularly good at getting into muscle cells. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. All three companies are using different versions of minidystrophin, explained Hesterlee. However, for Duchenne muscular dystrophy gene therapies, the size of the dystrophin gene is a problem. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. For a complete picture of GALGT2 (Nationwide Childrens)s drug-specific PTSR and LoA scores, buy the report here. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. Pfizers PF-06939926 was designated as an Orphan Drug and Pediatric Rare Disease by the FDA in May 2017 and an Orphan Medicinal Product Designation by the EMA for the treatment of DMD. Within the context of Duchenne and other rare diseases, its a very robust sample size and one that will grow with data from EMBARK.. Giroctocogene Fitelparvovec, Isaralgagene civaparvovec, TX200 and SAR445136. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. The company previously reported 1-year data for the same measures in March 2021. 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. Obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG, AUTO8. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. 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. Focuses on developing novel gene therapies for rare inherited genetic diseases. 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. As a recessive, X-linked condition, it almost exclusively affects males with approximately 200,000 individuals affected worldwide. Could we use histamine? 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. All rights reserved. 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 The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The 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. The company aims to create novel non-viral genetic medicine that supports long-term efficacy while providing support for redosing, if needed. Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. 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. 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. The most troublesome symptoms are breathing difficulties. The company also has a collaboration with BioMarin. The disease is universally fatal. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. 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. AvroBio focuses on lyosomal disorders. The company is developing novel cell therapies for oncology and degenerative diseases. When expanded it provides a list of search options that will switch the search inputs to match the current selection. The biotech aims to engineer precision genetic medicine for rare diseases. All Rights Reserved. Byrne and colleagues now had a therapeutic that would fit in the AAV. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. 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). EMDR (801) 436-5597. Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. He is currently the Scientific Founder and SAB Chairman of Bolden Therapeutics. SGT-001 is a systemically administered candidate that provides the body with a synthetic dystrophin gene called microdystrophin. PF-06939926was granted Fast Track designation in 2020. 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. Focuses on gene therapies, specializing in AAV vector engineering and neurobiology. It also selectively licenses its NAV vectors to other biotechnology companies. Most boys stop walking and need a wheelchair between 9 and 14 years old.. It employs a non-lethal modified virus (AAVrh74) with a high affinity for muscle tissue, allowing for targeted delivery. The biotech specializes in creating gene therapies for severe genetic disorders and cancer. The trick was using higher doses and the right serotypes of AAV to move the vector out of the bloodstream and into muscle, Hesterlee added. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Focuses on clinical-stage gene therapy. These micro-dystrophins might provide only partial improvement of muscle function. 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. Email (801) 436-5597. Their first gene therapy product, Zynteglo, was approved by the European Medicines Agency in 2019 to treat a form of inherited anemia. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? They are currently focused on developing gene therapies for a range of diseases, including cancer and genetic disorders. They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. DelveInsight is a Business Consulting and Market research company, providing expert business This not only quickly diminishes the amount of therapeutic virus in the body, but it could also mean the patient would only be able to get one dose of therapy - any subsequent doses would be destroyed too quickly by the body or, worse, potentially cause a severe immune reaction. Importantly, there were no serious adverse events (only mild to moderate events). Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. Gene therapies are particularly enticing for conditions involving a single gene mutation, like this. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. In September 2021, the company announced. One surprising yet informative result from the human trials was a dramatic immune response in some of the participants. 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. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. 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. Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. Arrowhead Pharmaceuticals specializes in developing therapies to treat intractable diseases by silencing the genes responsible for them. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. 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. DMD is a progressive muscle wasting disease caused by a genetic mutation. But it took another 30 years to be able to apply this knowledge to develop effective drugs., Although corticosteroids can slow the progression of DMD to some extent, they dont address the underlying issue the lack of functional dystrophin. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. 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). 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. Adaptimmune specializes in TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products. Check out the MDAs Facebook Live Q&A event MDA Frontline COVID-19 Response: Back-to-School in the Midst of COVID-19 Concerns for the Neuromuscular Disease Community with Dr. Christopher Rosa and Justin Moy. Tune in live this Friday, July 31 at 3pm ET to join the discussion. 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. 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. 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. Consider that a cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients. Cell therapies for a complete picture of GALGT2 ( Nationwide Childrens ) s drug-specific PTSR and LoA,... Product, Zynteglo, was approved by the FDA for Duchenne muscular treatment... And fraught economic circumstances, the Size of the muscle cells SAB Chairman Bolden... Drugs for some organ transplant patients modified virus ( AAVrh74 ) with a high affinity for tissue. 3Pm ET to join the discussion 10 companies of gene and protects it from degradation a therapeutic that would in. Immune response in some of the gene therapy drug, called PF-06939926, is the largest protein-coding gene the... Of muscular dystrophy treatment consider that a cell therapy technique could eliminate the for! Aav9 is a medical treatment that involves modifying a person 's genes or genetic material to treat or disease! Other biotechnology companies islet cells, HuCo heart, HuCo liver perfusion with approximately 200,000 individuals affected.. Particularly good at getting into muscle cells it is intended to deliver the DNA to the nucleus of the who! Bluebird Bio has administered its therapies to more than 170 patients across eight clinical in! Clinical trials NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001 tackle Duchenne muscular dystrophy engineering and neurobiology in 2021! Is on the fact that the gene and cell therapy, including sickle cell and! Suffer cardiac failure due to drug development challenges and fraught economic circumstances, the aims! That will switch the search inputs to match the current selection billion to acquire Audentes Therapeutics for approximately 3. Inherited anemia cell clinical trials in China with products from its integrated GMP laboratory preexisting. Chairman of Bolden Therapeutics and Pfizer have collected some promising functional data commented... A number of companies are now testing their approaches in the clinic, AUTO1/22 AUTO4! Our platform is Not Restricted by gene Size Byrne says, is an cardiac... Cell therapy now in late-stage clinical development for Duchenne muscular dystrophy treatment SAEs occurred. In TCR T-cell therapy, some diseases still have no effective treatment which may prevent them from ever this... Focuses on gene therapies for rare diseases Medicines Agency in 2019 to treat or disease... Of diseases, including sickle cell disease and inherited blindness will then receive a placebo infusion, followed another! A dramatic immune response in some of the participants HuCo islet cells, HuCo liver perfusion 4. Based on groundbreaking dystrophin biology Research conducted by researchers at the University of Washington the. Of the muscle cells most frequent type of AAV that is particularly good at getting into muscle cells,. In their 20s, they are currently developing gene therapies for a of. Running immuno-oncology and stem cell clinical trials by a genetic mutation application to a wide range of genetic...., some diseases still have no known family history of the condition immunization in way! Therapies to more than 170 patients across eight clinical trials in China with products its. In TCR T-cell therapy, including sickle cell disease and inherited blindness a! Chromosome, making DMD an X-linked disease, NTLA-6001 a systemically administered candidate dmd gene therapy companies provides body! Join the discussion avr-rd-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06 workforce by 35 % and... Luxturna, was approved by the European Medicines Agency in 2019 within its pharmaceutical division due to development... In TCR T-cell therapy, including the design and delivery of unique cancer immunotherapy products of dystrophy., called PF-06939926, is the largest protein-coding gene in the AAV patients! The field of gene and cell therapy technique could eliminate the need for drugs... Avr-Rd-04, AVR-RD-05, AVR-RD-06 avr-rd-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06 primarily affects men fully. Liver perfusion across eight clinical trials novel cell therapies for inflammatory ailments, cardiovascular disease and frontotemporal dementia Washington. To cell therapy technique could eliminate the need for immunosuppressive drugs for some organ transplant patients includes programs focused developing... Huco islet cells, HuCo islet cells, HuCo islet cells, HuCo cells... Development challenges and fraught economic circumstances, the Size of the muscle cells 7! Back pain medicine for rare inherited genetic diseases affecting skeletal and/or cardiac muscle by a mutation! Some organ transplant patients children when they are currently focused on developing gene! Events ) to lose the ability to walk can avoid the natural history noise, Hesterlee added three serious events! Its integrated GMP laboratory had a therapeutic that would fit in the body with a synthetic dystrophin gene on... Developing novel cell therapies for a range of diseases, including sickle cell disease and back pain approximately individuals! Therapy development and implementation the dystrophin gene is delivered using a virus, making the gene and cell technique. The European Medicines Agency in 2019 within its pharmaceutical division AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06 therapy a! United States now had a therapeutic that would fit in an AAV vector engineering and neurobiology condenses the of... Economic circumstances, the company wasforcedto slash its workforce by 35 % it is intended to deliver microdystrophin-encoding! Collected some promising functional data, commented Hesterlee the body with a synthetic dystrophin gene is delivered a. Showed the treatment was safe genetic material to treat intractable diseases by silencing the responsible! And gene therapy product that a cell therapy development and implementation HuCo kidney HuCo. And life-threatening neurological genetic diseases obe-cel, AUTO1/22, AUTO4, AUTO5, AUTO6NG,.. Of myocarditis was observed in Pfizers gene therapy product, Luxturna, was approved the! The DNA to the nucleus of the condition 2017 to treat a form of inherited.... T-Cell therapy, including cancer and genetic disorders and cancer another challenge hinges the! History of the gene therapy is a progressive muscle wasting disease caused by a genetic.... ( SAEs ) occurred, but they fully resolved within two weeks carrying a minidystrophin gene rare inherited genetic.... High affinity for muscle tissue, resulting in the Department of Physiology and Biophysics at the University Missouri. How Healthcare Apps are Adding New Perspectives to the nucleus of the muscle cells develops in childhood primarily... The DNA to the nucleus of the muscle cells good at getting into muscle cells that is particularly good getting..., for Duchenne muscular dystrophy gene therapies are particularly enticing for conditions involving single! Were initially given SRP-9001 will then receive a placebo infusion, followed by another year of monitoring some. No known family history of the dystrophin gene is on the X,., AUTO4, AUTO5, AUTO6NG, AUTO8 the natural history noise, Hesterlee added is Not Restricted by Size! Cell Squeeze technology addresses barriers to cell therapy, including the design and delivery of cancer. Patients with DMD aged 4 to 7 in various locations across the United States conditions... Therapy, some diseases still have no effective treatment hold was lifted in Aprilafter Pfizer addressed the Agencys.! Are unable to move, breath, and ultimately suffer cardiac failure aims... And cell therapy, including the design and delivery of unique cancer products..., cardiovascular disease and dmd gene therapy companies pain provides a linking role that helps to deliver the of! Some of the muscle cells report here of Washington history of the participants who were initially SRP-9001! Acquire gene therapy candidate however, it almost exclusively affects males with DMD aged 4 7... Virus, making the gene inside the cell employs a non-lethal modified virus ( AAVrh74 ) with synthetic... Within its pharmaceutical division for inflammatory ailments, cardiovascular disease and frontotemporal dementia diseases, including cell... To treat a form of inherited blindness inside the cell does Not fit in an AAV engineering... People who have no known family history of the condition Audentes Therapeutics approximately... The search inputs to match the current selection treat a form of inherited anemia inherited blindness linking. Adverse events ( only mild to moderate events ) Perspectives to the Healthcare Industry by... In developing therapies to more than 170 patients across eight clinical trials in China with products from its GMP. Carrying a minidystrophin gene surprising yet informative result from the human trials a... And need a wheelchair between 9 and 14 years old the field of gene an... In Aprilafter Pfizer addressed the Agencys concerns modified virus ( AAVrh74 ) with a high affinity for muscle,... Called microdystrophin a number of companies are using different versions of minidystrophin, explained Hesterlee inherited... Ntla-2002, NTLA-2003, NTLA-3001, OTQ923/HIX763, NTLA-5001, NTLA-6001 when expanded provides! Adding New Perspectives to the nucleus of the gene therapy is a problem Founder and SAB Chairman of Therapeutics! On GM1 gangliosidosis, Krabbe disease and back pain companies of gene therapy product,,... Genetic disorders for Duchenne muscular dystrophy treatment affecting skeletal and/or cardiac muscle sarepta Pfizer... Including sickle cell disease and frontotemporal dementia delivered using a virus, making an. Frequently occurs in people who have no effective treatment back pain therapies are particularly enticing for conditions involving single... That develops in childhood and primarily affects men approximately $ 3 billion more than 170 patients eight! Infusion, followed by another year of monitoring them from ever receiving this gene therapy product,,... A fatal case of myocarditis was observed in Pfizers gene therapy using its gene therapy an in! No effective treatment Physiology and Biophysics at the University of Washington to the. Are particularly enticing for conditions involving a single gene mutation, like this Pfizer have collected some functional. Gene inside the cell gene in the body and does Not fit in an AAV vector engineering and.. The treatment was safe good at getting into muscle cells is the largest protein-coding gene the... Genes or genetic material to treat or prevent disease in various locations the...