The biotechnology company Epic Bio presented preclinical data supporting the potential clinical progress of EPI-321 in treating facioscapulohumeral muscular dystrophy (FSHD). The data presented supports EP1-32’s development as a FSHD patient’s one-time gene therapy treatment. Their data provided comprehensive information on the pharmacokinetics, safety, and biodistribution of EPI-321 in non-human primates (NHP). They intend to file an IND and CTA by the end of this year and are committed to the rapid progression of EP1-231 as a candidate for clinical studies.
The DUX4 gene is found on chromosome 4 and is normally methylated, but in the case of FSHD patients, there is a loss of methylation at the gene. Consequently, the expression of the DUX4 gene results in the activation of apoptotic pathways that lead to progressive muscle wasting. EP1-321 is an AAV vector that encodes a catalytically inactive Cas protein fused to gene-suppressing modulators. These modulators function by permanently inhibiting the expression of DUX4.
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
Related News
Sangamo Therapeutics Secures Accelerated Approval Pathway for Gene Therapy in Fabry Disease
Sangamo Therapeutics has announced a major advancement in its gene therapy program for Fabry disease, as the U.S. FDA has provided a clear pathway for Accelerated Approval. This decision could potentially speed up approval timelines by three years, with a Biologics...
[2024/10/18] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
GSK sues Moderna over mRNA vaccine patents, seeks ‘reasonable royalty’
GSK on Tuesday unveiled a lawsuit filed against Moderna in Delaware federal court, alleging that its patented inventions provide the “foundation” for Moderna’s mRNA vaccine portfolio. GSK said it’s looking to recover “a reasonable royalty” for Moderna’s tens of...
Gene Therapy Automatically Converts Omega-6 to Omega-3 Fatty Acids in the Body
Shriners Children's Develops New Technology to Prevent Childhood Obesity ST. LOUIS, Oct. 16, 2024 /PRNewswire/ -- According to the Centers for Disease Control, nearly 20% of children and teens are considered obese. Research shows it can have a dramatic impact on a...
Related Services
Plasmids GMP Services
READ MORE
AAV GMP Services
READ MORE
Technology Platforms
READ MORE