Interim Phase 1b/2a Data on Gene Therapy BB-301 for OPMD-Related Dysphagia: Jerel A. Banks, MD, PhD

This content was originally published by our sister publication, CGTLive®.

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"We've seen benign safety and clinically meaningful improvements in swallowing function. The 1 and 2 year data at the low dose give us strong evidence that the drug is improving swallowing in a durable and safe way, and the high dose data, while early, further supports our enthusiasm for the disease-modifying effect of the drug to improve the swallowing for patients with OPMD.”

Benitec Biopharma is currently investigating BB-301, an experimental adeno-associated virus (AAV)–based “silence and replace” gene therapy, as a potential treatment for dysphagia associated with oculopharyngeal muscular dystrophy (OPMD). The therapy is currently being studied in a phase 1b/2a clinical trial (NCT06185673). Interim findings from this study were recently shared at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference, which took place March 8 to 11 in Orlando, Florida.

During the meeting, NeurologyLive®’s sister site CGTLive® spoke with Jerel A. Banks, MD, PhD, CEO and chairman of Benitec Biopharma, to discuss the emerging data and their clinical relevance. He noted that the first-in-human trial is assessing outcomes through a combination of patient-reported measures and objective imaging techniques. Participants complete the Sydney Swallow Questionnaire to capture symptom burden, while X-ray–based swallowing assessments are used to examine functional measures such as throat closure and the ability to clear food or liquid after swallowing.

According to Banks, the first 7 treated patients have shown encouraging results. Six patients received a lower dose of the therapy and demonstrated a favorable safety profile. Notably, the first patient treated in the study has now reached the 2-year follow-up mark, with data suggesting durable and even deepening improvements in swallowing function over time. Furthermore, the first patient treated at the higher dose demonstrated particularly strong responses in objective swallowing metrics.

Banks emphasized that given the progressive nature of OPMD-related swallowing impairment, functional improvements could carry meaningful clinical impact by reducing aspiration risk. He also discussed future plans for advancing the program.

Click here for more MDA 2026 coverage.