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Find Clinical Drug Pipeline Developments & Deals by Benitec Biopharma
BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized PABPN1 & two siRNAs against mutant PABPN1. It is being evaluated for the treatment of oculopharyngeal muscular dystrophy.
The Company intends to use the net proceeds from the financing to fund the clinical development and related commercialization of BB-301, which is being evaluated in early-stage clinical trial studies for treating oculopharyngeal muscular dystrophy.
BB-301 is a Cell and Gene therapy drug designed for Oculopharyngeal Muscular Dystrophy Related Dysphagia, utilizing the PABPN1 gene as a replacer and administered via intramuscular injection.
The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.
The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.
BB-301 is a novel, modified AAV9 capsid expressing bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 and two small inhibitory RNAs against mutant PABPN1 is currently in development for Oculopharyngeal Muscular Dystrophy.
BB-301 is currently in development for Oculopharyngeal Muscular Dystrophy. BB-301 results in robust inhibition of mutant PABPN1 and concomitant replacement of the codon-optimized PABPN1 protein. The treatment restores muscle strength and muscle weight.
The Company intends to use the net proceeds from this financing for the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB301 treatment study.
The company intends to use the proceeds for development of BB-301, including the natural history lead-in study and Phase 1b/2a BB-301 treatment study, for continued advancement of development activities, for general corporate purposes and for strategic growth opportunities.
The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall goal of providing data to submit an investigational new drug application to the U.S. Food and Drug Administration, a mandatory step to conduct clinical trials.
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