For UCLA-based startup, new muscular dystrophy treatment is a personal mission

Courtney Young helped develop the gene therapy at the heart of a biotech startup,MyoGene Bio, when she was a doctoral student at UCLA from 2013 to 2018. But the kernel of the company ’s endeavor is older still — and definitively personal.In 2008, when Young was in high school, she found out that her cousin, then just a toddler, had been diagnosed with Duchenne muscular dystrophy. She made it her mission to cultivate the skills needed to provide better options for him and others who were suffering with the deadly muscle-wasting disease.“I directed my career toward working on Duchenne,” said Young, MyoGene Bio’s co-founder and CEO. “I came to UCLA specifically because there’s a high concentration of researchers working on the disease. My advisers and I decided to develop a novel treatment that targets the primary cause of disease, because we really wanted to make a meaningful impact on the lives of Duchenne patients.”Young now pursues that goal close to her earlier scientific home, because MyoGene Bio is based at theMagnify incubator in theCalifornia NanoSystems Institute at UCLA. Her company reached the finals of the 2021 University of California Startup Innovation Challenge, a contest for startups with at least one founder affiliated with UC. Young also will be among six entrepreneurs featured at theUC Biotech Investor Demo Day on March 11.Building on all of that momentum, Young is now seeking venture capital funding and conduct...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news

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NEW YORK--(BUSINESS WIRE)--October 01, 2020 -- Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the...
Source: - Clinical Trials - Category: Pharmaceuticals Source Type: clinical trials
Dear Investor, Please find attached a press release from our partner Sarepta Therapeutics: Do not hesitate to contact us for any further questions. With best regards,
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news
Gene therapy for Duchenne muscular dystrophy (DMD) is being evaluated as a therapeutic strategy to restore production of functional dystrophin. Replacement of full-length dystrophin gene is not feasible due to its large size relative to the cloning capacity of adeno-associated virus (AAV)-derived vectors ( ∼5kb). Strategies based on delivery of shortened, but functional forms of dystrophin have emerged as a viable option. Identification of the optimal shortened micro-dystrophin construct that most normalizes muscle function is a key translation question.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Duchenne muscular dystrophy (DMD) is a common and fatal neuromuscular disease caused by loss-of-function mutations in the DMD gene which encodes dystrophin. Such genetic defects are amenable to correction by genome editing. However, DMD can be caused by an extensive list of mutations which will require a large volume of personalized gene therapies to tackle. This demand cannot be met in a reasonable timeframe with current development pipelines. Producing a DMD gene therapy requires assessing the approach's ability to restore the dystrophin protein in vitro prior to in vivo studies.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Growing evidence demonstrates a continuous interaction between the immune system and the skeletal muscle in inflammatory diseases of different pathogenetic origins, in dystrophic conditions such as Duchenne Muscular Dystrophy as well as during normal muscle regeneration. Although one component of the innate immunity, the macrophage, has been extensively studied both in disease conditions and during cell or gene therapy strategies aiming at restoring muscular functions, much less is known about dendritic cells and their primary immunological targets, the T lymphocytes. This review will focus on the dendritic cells and T lym...
Source: Frontiers in Physiology - Category: Physiology Source Type: research
on CL Abstract Spinal Muscular Atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletions or mutations in the survival motor neuron (SMN1) gene. An important hallmark of disease progression is the pathology of neuromuscular junctions (NMJs). Affected NMJs in the SMA context exhibit delayed maturation, impaired synaptic transmission, and loss of contact between motor neurons and skeletal muscle. Protection and maintenance of NMJs remains a focal point of therapeutic strategies to treat SMA, and the recent implication of the NMJ-organizer Agrin in SMA pathology suggests additional NMJ organizi...
Source: Biochemical and Biophysical Research communications - Category: Biochemistry Authors: Tags: Biochem Biophys Res Commun Source Type: research
We describe the design of a non-viral mammalian expression vector in which the primary transgene (a truncated dystrophin gene linked with Duchenne muscular dystrophy (DMD)) named microdystrophin delR4-R23/delCT (MD1) is under the transcriptional control of elements of desmin locus control region (DES-LCR). The designed vector, named as DES-LCR/MD1-EGFP, was constructed by cloning two fragments into the pBluescript backbone. Fragment 1 contains DES-LCR enhancer and DES-LCR promoter region while fragment 2 contains MD1 transgene and reporter EGFP (enhanced green fluorescent protein) gene separated by linker P2A (2A peptide)....
Source: Bioinformation - Category: Bioinformatics Authors: Tags: Bioinformation Source Type: research
Source: NPR Health and Science - Category: Consumer Health News Authors: Source Type: news
Dear Investor, Please find attached a press release by Sarepta Therapeutics: Do not hesitate to contact us for any further questions. With best regards,
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news
Xin Xie Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked dystrophin gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the dis...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Review Source Type: research
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