Guess the Therapy Answer May 1, 2026
Which Benitec Biopharma gene therapy is currently in clinical trials for the treatment of OPMD-Related Dysphagia?
Answer: BB-301.
See below for further reading on BB-301:
April 19, 2026 — Benitec Biopharma’s BB-301, an investigational adeno-associated virus (AAV) vector-based silence and replace gene therapy, is currently being assessed for the treatment of oculopharyngeal muscular dystrophy (OPMD)-related dysphagia in a phase 1b/2a clinical trial (NCT06185673). Jerel A. Banks, MD, PhD, the chief executive officer and chairman of the board of Benitec, recently presented updated data from this study at the 2026 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 8-11 in Orlando, Florida.
CGTLive® interviewed Banks on the floor of the conference to learn more about the findings. Banks gave some context regarding the study design, went over the key data points he presented and their big picture implications, and discussed future plans for the program.
"We've seen benign safety and clinically meaningful improvements in swallowing function,” Banks told CGTLive. “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.”
March 25, 2025 — Benitec announced that 1,443,000 shares of its common stock will be priced at $13.00 per share, and that along with prefunded warrants to purchase common stock in lieu thereof and a concurrent registered direct offering of 900,000 shares with Suvretta Capital a total of approximately $30 million in gross proceeds are expected to find their way to the company. The funding will help support development of the company's therapeutics, such as BB-301, an investigational AAV vector-based silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy-related dysphagia.
