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.
On Fubruary 16th, U.S. Food and Drug Administration (FDA) has approved Amtagvi, marking the first instance where a cell therapy has been authorized for the treatment of solid tumors. Developed by Iovance Biotherapeutics, Amtagvi (lifileucel) is a pioneering...
With another quarter exceeding analysts’ expectations, Blueprint Medicines has raised its peak sales estimate for rare disease drug Ayvakit to $2 billion. The Massachusetts biotech unveiled the new target Thursday along with its latest earnings results, which showed...
In a recent announcement, Rocket Pharmaceuticals, Inc. has encountered a three-month postponement by the U.S. Food and Drug Administration (FDA) for its groundbreaking autologous gene therapy aimed at treating a rare white blood cell disorder. This decision shifts the...
Replicate Bioscience Announces Positive Initial Clinical Results for its Next Generation srRNA Technology
Replicate's srRNA vaccine elicited a protective immune response that met the WHO threshold for protection at unparalleled low doses Threshold for protection achieved with just a single dose of vaccine RBI-4000 well-tolerated at all doses tested Replicate Bioscience, a...
AAV cGMP Manufacturing
Comply with GMPs, all applicable regulatory and the standards for IIT & IND applications.
AAV Packaging Services
Ranging from pilot to industrial-scale AAV packaging for both in vitro and in vivo studies.
AAV Capsid Engineering
Proven technology paving your path to effective therapies for cancer or genetic disorder