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
Taysha Drops Gene Therapy Candidate as FDA Again Calls for Study Changes
Pictured: FDA sign in its headquarters/iStock, GrandbrothersTaysha Gene Therapies has decided to drop its lead experimental AAV-based gene therapy candidate after the FDA reiterated calls for the company to put the treatment through a randomized, double-blind...
FDA Accepts Orchard’s BLA for Rare Disease Gene Therapy Behind EU Approval
Pictured: Gene therapy illustration showing an injection targeting a specific DNA site/iStock, Ilya Lukichev The FDA on Monday accepted Orchard Therapeutics’ Biologics License Application for OTL-200, an investigational gene therapy for rare disease metachromatic...
Excision Biotherapeutics’s pre-clinical studies testing dual-guide CRISPR strategies for viral excision as part of the company’s efforts to develop a cure for HIV
Dr. Nadia Amrani, a scientist at Excision Biotherapeutics conducts pre-clinical tissue culture studies testing Excision's dual-guide CRISPR strategies for viral excision as part of the company's efforts to develop a cure for HIV. (Photo Credit: Dawn Wessman...
Moderna, Immatics Ink Potential $1.8B Oncology Deal including Cancer Vaccines
Pictured: Moderna sign on building in Cambridge/iStock, hapabapaVaccine giant Moderna and oncology biotech Immatics have inked a research partnership worth a little more than $1.8 billion in potential payments to the latter. The collaboration will combine Moderna’s...
Related Services / Products
Plasmids GMP Services
READ MORE
AAV GMP Services
READ MORE
Technology Platforms
READ MORE
