Boston-based Decibel Therapeutics, in collaboration with Regeneron Pharmaceuticals, has been granted orphan drug designation by the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) for its lead gene therapy product candidate, DB-OTO. This AAV dual-vector-based gene therapy is designed to package OTOF cDNA in two AAV vectors, solving the problem of OTOF gene size exceeding the payload capacity of a single AAV vector. Orphan drug designation is granted to drugs that treat rare conditions affecting no more than five in 10,000 people in the European Union, where there is no satisfactory treatment option or where the medicine could be of significant benefit to those affected. DB-OTO is intended to provide durable, high-quality, physiological hearing to those with profound, congenital hearing loss caused by mutations of the otoferlin gene, which regulates synaptic transmission, facilitating communication between inner ear sensory cells and the auditory nerve. DB-OTO also received orphan drug and rare pediatric disease designations from the U.S. FDA in 2021. Currently, there are no approved pharmacologic treatments for Otoferlin-related hearing loss. Decibel plans to initiate a global Phase 1/2 clinical trial of DB-OTO, named CHORD™, in the first half of 2023, with the goal of restoring hearing in individuals with congenital deafness caused by mutations in the OTOF gene.
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.
Experimental ‘decoy’ protects against SARS-CoV-2 infection
Durable vectored immunoprophylaxis by decoy-expressing AAV vectors. (A) Balb/c mice (n = 3) were injected i.n. with decoy-luciferase fusion protein-expressing AAV vectors (1 × 1012 vg). Luciferase activity was visualized by live imaging over 60 d at the indicated time...
Gene therapy rescues hearing for the first time in aged mouse models
Graphical abstract. Credit: Molecular Therapy (2023). DOI: 10.1016/j.ymthe.2023.05.005By 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic...
Predicting the liver-tropism of AAV vectors based on pre-clinical liver models
Ex vivo results in human and NHP liver models. (a) Schematic of transduction of indicated in vitro and ex vivo models. (b) NGS read contribution (%) for each AAV from extracted DNA. (c) NGS read contribution (%) for each AAV from mRNA-derived complementary DNA. AAV,...
Researchers investigate the protein BVES and its important role in muscular dystrophy
BVES disruption compromises the body weight gain and muscle function in mice. All animal experiments were performed in WT and BVES-KO male mice with the C57BL/6N genetic background. a Immunofluorescence images of GA muscles in WT and BVES-KO mice (4 months of age)...
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