Benitec Biopharma Inc. (NASDAQ: BNTC), a leader in gene therapy, has announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for BB-301. This marks a significant development in the treatment of Oculopharyngeal Muscular Dystrophy-related Dysphagia (OPMD).

Benitec’s BB-301: First Potential Treatment for OPMD

Oculopharyngeal Muscular Dystrophy, a life-threatening genetic disorder, affects roughly 15,000 patients across the United States, Canada, Western Europe, and Israel. Patients suffering from OPMD lose the ability to swallow liquids and solids, which often leads to chronic malnutrition and even fatal aspiration pneumonia. Until now, there have been no approved therapeutic agents to treat this disorder. Benitec’s BB-301, a ‘silence and replace’ gene therapy, represents the first possible treatment for OPMD’s often debilitating and potentially fatal symptoms.

IND Clearance: A Milestone for Benitec

Jerel A. Banks, M.D., Ph.D., Executive Chairman and CEO of Benitec, hailed the FDA’s clearance of BB-301’s IND as a significant milestone for both OPMD patients and the company itself. He emphasized the importance of this breakthrough treatment in potentially addressing the severe symptoms of OPMD.

The Phase 1b/2a Clinical Study: Participants and Process

Participants from Benitec’s ongoing Natural History (NH) study will be eligible for the Phase 1b/2a clinical study of BB-301, following six months of baseline data collection. After undergoing a one-day dosing procedure, each study participant will be evaluated on various clinical and radiographic outcome measures, including objective assessments of swallowing safety and efficiency. Currently, 13 subjects are enrolled in the NH study, with the potential for each to transition to the Phase 1b/2a clinical dosing study for BB-301.

BB-301: A Unique Therapeutic Approach

BB-301 employs a novel silence-and-replace strategy, making use of a unique, single bifunctional construct. This construct promotes co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. The two siRNAs silence the expression of the faulty mutant PABPN1 while permitting the expression of a functional version of the protein. Benitec believes that this unique approach positions BB-301 optimally for the treatment of OPMD by halting mutant expression while providing a functional replacement protein.

As Benitec moves forward with the next stage of trials, the healthcare industry is eagerly awaiting the potential benefits this unique therapeutic approach could offer OPMD patients.

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