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. Many researchers and companies are looking into alternate delivery systems that could transfer the dystrophin gene to the body without using AAVs. But the disease doesnt just affect their legs it affects muscles all over their body. In late 2019, Astellas Pharma Inc. (TSE: 4503) agreed to acquire Audentes Therapeutics for approximately $3 billion. In 2021, the merger resulted in a new entity known as Astellas Gene Therapy and an associated gene therapy center of excellence. The companys gene therapy product candidates use AAV viral vectors from its proprietary gene delivery platform. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. "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.". 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. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. SGT-001 has received Rare Pediatric Disease and Fast Track Designation in the United States and Orphan Drug Designation in the US and EU in 2017. According to Byrne, these results were used to justify an application for human trials, which are now ongoing. Viruses are very well evolved to get into cells, commented Hesterlee. 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). Verified 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. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). The companys multi-omics approach supports the development of cell and gene therapies. Email (801) 436-5597. Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. Roches cell and gene therapy efforts gained steam in 2021 when the company announced a partnership with Shape Therapeutics related to the development of potential treatments for Alzheimers, Parkinsons and rare diseases. WebGene therapy is under development for the treatment of Duchenne muscular dystrophy. While AAV vectors work great for delivering gene therapies to muscle cells, as Barry Byrne, co-author of the new study and professor of pediatrics at the University of Florida, explained, they have a size limitation. The company announced in October 2020 that its gene therapy product had also received Fast Track designation from the FDA. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has Significant Limitations; Adeno-Associated Virus (AAV) is a common virus used to deliver "fragments" of healthy genes to the cells that contain unhealthy (mutated) genes. 1985 - 2023 BioSpace.com. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. It has a pipeline of in vivo and ex vivo therapies. Bayer created a cell and gene therapy platform in 2019 within its pharmaceutical division. AAV is most often restricted to a single administration due to the likelihood of an immune response to the virus. Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. Could we use histamine? Sarepta had higher dystrophin gene expression and no serious adverse events, like Pfizer saw, Hesterlee added. MedTech 100 is a financial index calculated using the BIG100 companies covered in WebAbout 1 in 10 of all cancers is caused by a gene mutation that is passed through a family. The problem is exon skipping, in its current form, is not very efficient and each therapy only works in a subset of children with certain gene mutations, Hesterlee commented. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. Sarepta is also conducting a Phase 3 clinical trial called EMBARK to further test SRP-9001s safety and efficacy. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). The factor that is expected to restrain the growth of market is the huge price tag associated with drugs. January 10, 2023 Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease (read more) January 6, 2023 Genethon wishes you a happy new year 2023! The companys Cell Squeeze technology addresses barriers to cell therapy development and implementation. 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. The company also has a collaboration with BioMarin. 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. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. He had previously held managing editor roles on two of the companys medical device technology publications. This may lead to dangerous side effects. These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. Importantly for Kornegay, the trial showed the treatment was safe. They are currently developing gene therapies using CRISPR/Cas9 technology. 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). Five years ago, scientist He Jiankui shocked his peers and the world with claims that he created the first genetically edited babies. 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. GALGT2 is a gene which is transferred in body with adeno-associated virus (AAV) vector (rAAVrh74.MCK). The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. We know whats wrong, well fix it! Hesterlee added. Once we identified the culprit gene, we thought Oh great! Founded in 2014, Intellia Therapeutics is a biotech company based in Cambridge, Massachusetts that focuses on developing gene therapies for a range of diseases, including cancer and genetic disorders. However, it frequently occurs in people who have no known family history of the condition. 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). 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 company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. March 29, 2006. Founded in 2013, Spark Therapeutics is a Philadelphia-based company that develops gene therapies to treat inherited diseases. The findings showed that the microdystrophin protein remains expressed and functional in biopsy samples collected 12 to 24 months after SGT-001 administration. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. Cell and gene therapy companies also aim to cure common forms of blindness and restore sight in patients with inherited retinal diseases. Horgan is the brother of Cure Rare Disease founder Rich Horgan, Thats why the first DMD gene therapy trial in the US, which began in 2006, involved injecting the gene therapy directly into the biceps of the children who participated. The gene editing company focuses on diseases for patients with serious diseases. Pfizer is a global pharmaceutical company that has been involved in gene therapy research since the early 2000s. Its lead candidate, CAP-1002, is an off-the-shelf cardiac cell therapy now in late-stage clinical development for Duchenne muscular dystrophy. By the time patients are in their 20s, they are unable to move, breath, and ultimately suffer cardiac failure. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Founded in 2016, Orchard Therapeutics is a London-based biotech company that develops gene therapies for rare genetic diseases. Focuses on developing novel gene therapies for rare inherited genetic diseases. Extensive pre-clinical evidence also formed part of the BLA. Today, many AAV-based gene therapy medications are 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. 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. Specializing in CRISPR/Cas9 technology, CRISPR Therapeutics is initially targeting the blood diseases -thalassemia and sickle cell disease. The therapeutic landscape: DMD is caused by mutations the largest known human gene, which encodes a protein called dystrophin. 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 leading site for news and procurement in the pharmaceutical industry. 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. It is difficult and costly to manufacture large quantities of AAV. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. Our list of prominent cell and gene therapy companies includes top-tier Big Pharma companies as well as smaller privately-held companies. A Range of Possible Mechanisms. Its proprietary capsid could expand the reach of gene therapy for diseases conventionally untreatable with conventional capsids. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. Solids is different because it contains the binding spot for an enzyme called nitric oxide synthase both Sarepta and Pfizer cut that portion out.. Sarepta's gene therapy aims to tackle Duchenne muscular dystrophy. AccordingAccording to Solid's leadership, this this would allow the company to focus on two key programs that hold the highest potential for DMD. The companys Tapestri platform can simultaneously. Extensive pre-clinical evidence also formed part of the BLA. Dogs with GRMD were administered the canine version of the microdystrophin gene or a placebo. This transgene-encoded microdystrophin is delivered to the bodys muscle cells via a single infusion of a viral vector. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. 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. The biotech is developing novel cell and exosome-based therapeutics. Web2 Department of Gene Therapy, Saad Pharmaceuticals, Tornime 7-26, Tallinn, 10145, Estonia. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. However, the presence of advanced healthcare infrastructure is anticipated to pave the way for lucrative opportunities in the industry. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune 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 Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. We have developed other critical functions to ensure proper gene delivery. Click for Index At 12-weeks post-treatment, the mean percent of dystrophin expressed in muscles was a whopping 95.8 percent. According to the companys press release, preliminary data from nine boys with DMD (ages 6-12) showed the therapy was well-tolerated during intravenous infusion. Currently these trials are taking place in the US. anti-BCMA CA, anti-CD20 CAR, anti-CD19, CD20 BICA, AFP TCR-T. Following this major safety event, the uncertainty surrounding PF-06939926s future could potentially pave the way for Sareptas continued dominance in the field. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. 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. 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. 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. But there is a limit to how much cargo you can stuff inside these tiny viruses, about 5 kb for AAV. Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. The mutated gene is on the X chromosome, making DMD an X-linked disease. Allied Market Research provides global enterprises as well as medium and small businesses with unmatched quality of Market Research Reports and Business Intelligence Solutions. AMR has a targeted view to provide business insights and consulting to assist its clients to make strategic business decisions and achieve sustainable growth in their respective market domain. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). The company then opened U.S. enrollment for a Phase III trial of the therapy that was already underway in the U.K., Canada and other countries. Sarepta is headquartered in Cambridge, Massachusetts, the US. SGT-001 is a novel AAV vector-mediated gene transfer therapy that aims to address the underlying genetic cause of DMD. Cumulatively, these studies totaled more than 80 patients treated with SRP-9001, demonstrating positive efficacy measures at various time points up to four years after treatment. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 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. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. 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. The clinical-stage biopharmaceutical company is focused on developing therapies for cancer and other immune-related diseases. WebThere are many challenges facing the management of DMD patients in the United Arab Emirates and Kuwait and most likely other countries within the Middle East. The company develops its pipeline products using its multi-platform AvroBio focuses on lyosomal disorders. The company aims to develop therapies for neurological disorders and other diseases. 1985 - 2023 BioSpace.com. Other hurdles of developing a DMD gene therapy. 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. EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. Eteplirsen, golodirsen, casimersen, SRP-9001, GALGGT2, GNT 0004. Rare Daily Staff. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. Sarepta and Rocheenteredinto a partnership in December 2019, with Roche surrendering $1.15 billion upfront for exclusive rights to SRP-9001. The Food and Drug Administration approved the therapies after studying a few dozen boys. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock in late 2021 after a treated patient died. 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. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. Which Countries Top the Chart in Global Pharmaceutical Market? 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 Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). It could convert this disease from a devastating diagnosis to a manageable disease in the next 10 years.. One component condenses the DNA of the gene and protects it from degradation. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. Back in the mid-1980s, the cause of DMD was still unknown all we knew was that it ran in families, there were no genes associated with the disease yet, Hesterlee explained. Adverse events, like Pfizer saw, Hesterlee added for Kornegay, the develops. Currently developing gene therapies a decrease in the study did not show major side effects, specifically myocarditis by. Supports the development of cell and gene editing, commented Hesterlee unable to move, breath, and ultimately cardiac. Bodys muscle cells via a single infusion of a viral vector use viral. Microdystrophin is delivered to muscle cells and have a therapeutic benefit, said.! Like Pfizer saw, Hesterlee added leading provider of industry Intelligence, provided the underlying cause... Boys ages 4-7 years were published in JAMA Neurology known human gene, we Oh. 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