Benitec Biopharma Inc. is an emerging biotechnology firm dedicated to advancing groundbreaking genetic medicines. The company specializes in DNA-directed RNA interference (RNAi) based treatments, which it develops to address severe and enduring human conditions. Its pipeline features BB-301, an adeno-associated virus (AAV) gene therapy aimed at treating oculopharyngeal muscular dystrophy, and BB-103, designed to combat chronic hepatitis B virus infection. Established in 1995, Benitec Biopharma's main operations are situated in Hayward, California.
Benitec Biopharma Announces Oral Presentation of Interim Phase 1b/2a Clinical Study Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 at the 2026 American Society of Gene and Cell Therapy Annual Meeting
Benitec Biopharma announced that interim Phase 1b/2a clinical results for high dose and low dose BB-301 have been accepted for oral presentation at the ASGCT Annual Meeting.
The data set includes 12-month follow-up for four Cohort 1 completers, 24-month results for the first Cohort 1 patient, and interim results for the first Cohort 2 patient, demonstrating continued durable improvements.
BB-301 utilizes the company's proprietary Silence and Replace platform and holds Orphan Drug and Fast Track designations for OPMD, a rare disease with no approved therapies affecting approximately 15,000 patients in North America, Europe, and Israel.