FSHD Therapy

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.

Gene Therapy for FSHD Treatment
EP1-321 is one of Epic Bio’s therapeutic candidates for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare genetic disease that causes the degeneration of the face, shoulder blades, and upper arm muscles. This disorder is caused by the abnormal expression of a gene, DUX4, that is normally expressed during embryogenesis. This abnormal expression of DUX4 causes cell death and tissue degeneration in muscle tissue.

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.

Advancing Toward Clinical Trials
The information showed that EP1-321 repressed DUX4 expression, improved FSHD muscle cell survival, and showed no signs of toxicity in NHP studies. The company stated that they are working hard to introduce this therapy as an option for patients with FSHD. Additionally, they foresee initiating first-in-human trials in 2024. The data from their preclinical trial is promising and will hopefully lead to a treatment for FSHD patients.
From Geneoline: https://www.geneonline.com/promising-clinical-data-supports-clinical-initiation-of-epic-bios-fshd-therapy/
About PackGene

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.

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