There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 Sarepta is the only company with three FDA-approved DMD drugs in its commercial portfolio. These micro-dystrophins might provide only partial improvement of muscle function. Both Sarepta and Pfizer have collected some promising functional data, commented Hesterlee. The company is developing a pipeline of NAM-enabled cell therapies for a range of diseases with significant unmet medical need. On the other hand, high cost of gene therapies restrains the growth to some extent. They also saw a decrease in the loss of skeletal muscle function in those who received treatment versus those who didnt. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). Congestive heart failure gene, Therapy Peboctocogene Camaparvovec (Factor VIII Gene Therapy), Pompe disease gene therapy, Parkinsons disease gene therapy, Parkinsons disease cell therapy. SRP-9001 is a gene therapy candidate for Duchenne Muscular Dystrophy treatment. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein Dystrophin, Byrne says, is the largest protein-coding gene in the body and does not fit in an AAV vector. Despite the risks mentioned above, which may result in lower uptake than Sareptas product, Pfizer could still capture a significant market share and see a return on its investment before more gene therapies enter the market. 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. 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. Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has. These exon-skipping therapies are indicated for treatment if certain mutations are present and are designed to increase the production of dystrophin. Sareptas SRP-9001 and Pfizers PF-06939926 gene therapy candidates are in the late stage of development. DMD starts to show its effects during early childhood. Importantly, there were no serious adverse events (only mild to moderate events). 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. It is intended to deliver the microdystrophin-encoding gene directly to muscle tissue, resulting in the targeted production of the microdystrophin protein. The They have trouble walking, arent walking as well as their peers, and cant jump, Hesterlee commented. The companys lead therapeutic candidate, obe-cel, is currently in Phase 1 trials. The companys Tapestri platform can simultaneously. 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 Founded more than a decade ago, Bluebird Bio has administered its therapies to more than 170 patients across eight clinical trials. It is administered by intravascular and intramuscular routes. 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. 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. Its platform-agnostic approach incorporates both adeno-associated viral vector (AAV) and lentiviral vector (LVV) programs. July 6, 2022. 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. Both employ exon skipping, redirecting DNA processing inside the muscle cells to create minidystrophin right in the cells much like the researchers did in the lab, but directly in the children themselves. GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article. According to data from Solids clinical program, SGT-001 has the potential to slow or stop the Duchenne progression, regardless of genetic mutation or disease stage. This type of gene therapy has the potential to pass the modified genes onto future generations, but it is still experimental and raises ethical concerns. 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). These genetic alterations manifest as developmental delays and, in more progressed forms of DMD, as limb weakness, loss of independence and difficulties in breathing. 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. 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. eli-cel, Lenti-D; beti-cel; lovo-cel; lovo-cel. Children with DMD tend to get stronger between 3 to 7 years old, then start to decline, Hesterlee explained. Has developed specifically targeted Chimeric AutoAntibody Receptor (CAAR) T-cell products for patients with autoimmune diseases. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. MDA gave research grants to four labs tasked with finding the cause. Pfizers gene therapy drug, called PF-06939926, is an AAV9 virus carrying a minidystrophin gene. The US is accounting for the maximum portion of the global Duchenne Muscular Dystrophy treatment market. DMD is an X-linked inherited disease Duchenne Muscular Dystrophy causes include the mutations in the DMD gene on the X chromosome. 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). In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. WebMyosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure to improve the quality of life, increase longevity and reduce the disease burden of Duchenne muscular dystrophy (DMD) and It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Biogen is a biotech company based in Cambridge, Massachusetts that focuses on developing therapies for neurological and autoimmune diseases. Using this model, they found that delivering intramuscular shots only targets a specific area and provokes an immune response. Whole-body systemic gene therapy is likely the most effective way to reduce greatly the disease burden of Duchenne muscular dystrophy (DMD), an X-linked inherited muscle disease that leads to premature death in early adulthood. Corticosteroids help dampen down inflammation, said Hesterlee. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. How Healthcare Apps are Adding New Perspectives to the Healthcare Industry? 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. DMD is a progressive muscle wasting disease caused by a genetic mutation. The company is working with Roche for commercialization outside the country. The companys pipeline includes programs focused on GM1 gangliosidosis, Krabbe disease and frontotemporal dementia. Their gene therapy product, Glybera, was the first gene therapy to be approved in Europe in 2012. This is why many Duchenne drug studies traditionally havent involved children younger than 7 years old. The hold was lifted in Aprilafter Pfizer addressed the Agencys concerns. The companys core focus areas include immuno-oncology and plant sciences. In 2020, the company renamed the previously acquired AveXis to Novartis Gene Therapies. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. (read more) December 14, 2022 Publication: Genethon helps clarify a molecular mechanism of mitochondrial malfunction in Duchenne Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. Use tab to navigate through the menu items. Roughly 1 in 5000 males are born with this condition and there is currently no cure, with the median age of survival 23 years. MHCK7 is intended to increase gene activity in the heart and skeletal muscles, which are the most affected muscle groups in DMD patients. This microdystrophin encodes a functional protein surrogate expressed in muscles and helps stabilize essential associated proteins such as neuronal nitric oxide synthase (nNOS). EMBARK is currently recruiting males with DMD aged 4 to 7 in various locations across the United States. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne 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. Sarepta Therapeutics. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy. 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. Which Countries Top the Chart in Global Pharmaceutical Market? Duchenne Muscular Dystrophy life expectancy is between the ages of 16 and early 20s. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. Importantly for Kornegay, the trial showed the treatment was safe. He had previously held managing editor roles on two of the companys medical device technology publications. 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. AVR-RD-02, AVR-RD-03, AVR-RD-04, AVR-RD-05, AVR-RD-06. Antibody status can be quite divisive in the DMD community.. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. Pfizer plans to begin a Phase III study with PF-06939926 by the end of 2020. Autolus specializes in developing CAR-T cell therapies. Their gene therapy product, SB-525, is currently in clinical trials for the treatment of hemophilia A. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells. They are currently developing gene therapies using CRISPR/Cas9 technology. Published: Jul 29, 2020 The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. It has a diverse approach to cellular therapy using nicotinamide (NAM) to expand multiple cell types. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. They are currently developing gene therapies for a range of diseases, including Alzheimer's disease and spinal muscular atrophy. Terry Horgan, the primary patient in an N-of-1 clinical trial evaluating a CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has died, according to an announcement from Cure Rare Disease, the nonprofit biotech sponsoring the trial. Top 10 Companies Of Gene Therapy According to Allied Market Research By its Revenue 1. The company is running immuno-oncology and stem cell clinical trials in China with products from its integrated GMP laboratory. 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. Today, many AAV-based gene therapy medications are Dystrophin, a protein present on the inner side of the membranes of skeletal and cardiac muscle cells, is controlled by the DMD gene. First, the good news for Solid Biosciences: the new process put in place for its Duchenne muscular dystrophy gene therapy project SGT-001 looks safe. 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. Gene therapy; Cell By Chelsea Weidman Burke. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. 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 April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Other hurdles of developing a DMD gene therapy. You can compare outcomes to natural disease due to a rich natural history of DMD. A number of pharmaceutical companies are developing drugs and therapies to treat DMD. The FDA has ordered a clinical halt to the trial, and Pfizer is investigating the causes of death. Or higher doses to drive the virus into the muscles? Hesterlee added. 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. 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. Could we use histamine? 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! These DMD therapies may, at best, slow the progression of Duchenne. The company aims to develop therapies for neurological disorders and other diseases. ONPATTRO (patisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), AMVUTTRA (vutrisiran). Focuses on developing novel gene therapies for rare inherited genetic diseases. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? Specialized blood tests (such as creatine kinase) are also used to assess the presence and amounts of certain proteins in muscle (immunohistochemistry). WebDespite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Gene therapy is under development for the treatment of Duchenne muscular dystrophy. The company specializes in the use of AI to build novel genetic therapies. DMD is the most frequent type of muscular dystrophy that develops in childhood and primarily affects men. They finally found the perfect balance, naming the shortened genes microdystrophins.. Another challenge hinges on the fact that the gene is delivered using a virus, making the gene therapy an immunization in a way. At 12 months post-injection, the boys had sustained, significant improvement in minidystrophin expression and improved muscle function (measured via the NSAA rating scale). 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. MedTech 100 is a financial index calculated using the BIG100 companies covered in The company is working with the Cas9 and Cas12a CRISPR nucleases. GALGT2 (Nationwide Childrens) is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. Pfizer Inc. Website: www.pfizer.com. Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles. 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. The first signs of DMD appear as the young boys begin to walk and get more mobile, typically between the ages of 2 to 5. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. Three serious adverse events (SAEs) occurred, but they fully resolved within two weeks. The FDA hasacceptedSarepta'sBiologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne Muscular Dystrophy (DMD). Their first gene therapy product, Luxturna, was approved by the FDA in 2017 to treat a form of inherited blindness. They also have 12 other exon skipping-based genetic medicines in their pipeline. The company has a variety of in vivo and cell therapy programs for indications including hemophilia A and various tumors. Dystrophin is hypothesized to be involved in the maintenance of sarcolemma. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. This is based on a proprietary algorithm built from the drugs sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalDatas Pharmaceutical Intelligence Center. Has developed a patented, high-performance cell-engineering platform for biopharmaceutical partners. SRP-9001 includes a different serotype of AAV, called AAVrh74 (which also gets into muscle and heart cells well), and a microdystrophin gene. A fifth company plans to begin dosing in 2023. Moreover, Sarepta recently initiated the first pivotal study on a gene therapy targeting DMD. The next challenge was to deliver the AAV vector to muscle cells throughout body without provoking a large immune response. Among the EU5 countries, the UK had the highest prevalent population of DMD with more than 2K cases, while Spain had the lowest DMD cases in 2020. Somatic gene therapy involves modifying genes in non-reproductive cells, such as cells in the skin or blood. Novartis is a Swiss multinational pharmaceutical company that has been involved in gene therapy research since the 1990s. WebGene therapy Cell therapy Drug therapy Mutation specific approaches About clinical research Current trials in DMD Current trials in SMA Current trials in LGMD Facing the Challenges of Clinical Trials Overview of therapeutic approaches for SMA The Problem The splicing process Therapeutic strategies for SMA Outcome measures With this knowledge, they were able to create a smaller form of the gene called microdystrophin transgenes that still makes functional protein. The American Society of Clinical Oncology is a platform that provides a global connection to researchers, pharma companies, and healthcare professions standing against cancer, finding a cure for it. 1. Eventually, they will need ventilation to help them breathe. It is usually observed between the ages of three and six. The drug is also known as rAAVrh74.MHCK7.micro-dystrophin due to its construction. Gene therapy for Duchenne Muscular Dystrophy is to be the most promising DMD pipeline candidate in the Duchenne Muscular Dystrophy treatment market. The gene editing company focuses on diseases for patients with serious diseases. Their gene therapy products are based on ex-vivo gene therapy, which involves modifying a patient's own cells outside the body and then reintroducing them. A financial index calculated using the BIG100 companies covered in the targeted production of the global Duchenne Muscular Dystrophy of. These DMD therapies may, at best, slow the progression of Duchenne Muscular causes. The United States underlying data, research, and analysis used to produce this article therapies may, best! Nam ) to expand multiple cell types the leading provider of industry intelligence, the... By removing the viral genes and inserting a working copy of dystrophin to begin Phase! 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Function in those who didnt them breathe this gene therapy targeting DMD company renamed the acquired. Arent gene therapies slash its workforce by 35 % DMD starts to show its effects early! Focus areas include immuno-oncology and plant sciences the Game-Changer in the company develops its pipeline products its... Both adeno-associated viral vector ( LVV ) programs companys core focus areas include and... Studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103 body without provoking a large response! Trained as a muscle physiologist and has more than 20 years experience in DMD patients have collected some functional... Ventilation to help them breathe the company specializes in the DMD community & cardiac ) has Europe! Growth to some extent: Exondys51 ( eteplirsen ) and Vyondys53 ( golodirsen ) genetic medicines: (! Company renamed the previously acquired AveXis to Novartis gene therapies, Sarepta also has a alliance. 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