Benitec Biopharma has secured an oral presentation slot at the 2026 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Boston, Massachusetts. The company will present interim results from its Phase 1b/2a study of BB‑301, a “Silence and Replace” DNA‑directed RNA interference (ddRNAi) therapy for oculopharyngeal muscular dystrophy (OPMD), on May 15, 2026 at 9:15 AM in MCEC Room 257AB.
The presentation will cover 12‑month follow‑up data for the first four Cohort 1 completers, 24‑month follow‑up for the first Cohort 1 patient, and interim data for the first Cohort 2 patient. BB‑301 is the only clinical‑stage therapy for OPMD and has received Orphan Drug Designation from the European Medicines Agency and both Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration.
By sharing extended efficacy and safety data beyond the initial 12‑month results, Benitec aims to provide external validation of BB‑301’s durability and to support its regulatory strategy for a pivotal trial. The presentation also positions the company to engage with the scientific community and regulatory authorities ahead of a mid‑2026 FDA meeting that will shape the pivotal study design.
Dr. Jerel A. Banks, Executive Chairman and CEO, said, “We are strongly encouraged by the 100% response rate and the depth and durability of the responses that have been observed for all patients treated with BB‑301 to date.” He added, “We continue to be encouraged by the benign safety profile and the durability of efficacy demonstrated in our BB‑301 clinical development program.” Earlier statements highlighted the profound impact of BB‑301 on progressive dysphagia, noting that 97 % of OPMD patients experience this life‑threatening complication.
The presentation is expected to reinforce investor confidence in Benitec’s lead program and to underscore the company’s progress toward regulatory approval. The event marks a significant milestone in the development of a therapy for a rare, debilitating disease and is likely to influence the company’s future funding and partnership opportunities.
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