Tuesday, June 27, 2023
Benitec Biopharma Inc., a clinical-stage biotechnology company focused on gene therapy, has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for BB-301. BB-301 is a gene therapy designed to treat Oculopharyngeal Muscular Dystrophy-related Dysphagia, a chronic and life-threatening genetic disorder that affects approximately 15,000 patients in several regions.
OPMD patients experience difficulty swallowing liquids and solids, leading to malnutrition, aspiration, and potentially fatal episodes of aspiration pneumonia. Currently, there are no approved therapeutic agents for this condition. The clearance of the IND for BB-301 marks a significant milestone for both Benitec and OPMD patients, offering a potential treatment option for the debilitating and life-threatening symptoms associated with OPMD.
BB-301 utilizes Benitec's proprietary DNA-directed RNA interference (ddRNAi) platform. It is a modified AAV9 capsid that expresses a unique, single bifunctional construct. This construct enables the co-expression of codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) that target the mutant PABPN1. The siRNAs silence the expression of the faulty mutant protein, while the codon-optimized PABPN1 facilitates the production of a functional replacement protein.
The next step for Benitec is to conduct a Phase 1b/2a clinical study of BB-301 for the treatment of OPMD-related Dysphagia. Eligible subjects from Benitec's ongoing NH study will have the opportunity to participate in this clinical study after six months of baseline data collection. The study will assess the safety and efficacy of BB-301 using various radiographic and clinical outcome measures, including quantitative radiographic swallowing studies. Interim safety and efficacy data will be collected approximately every 90 days following the administration of BB-301 to each subject.
The clearance of BB-301's IND provides hope for OPMD patients and signifies a significant advancement in the development of a potential treatment for this debilitating condition.