The event will feature two key opinion leaders who will discuss the clinical symptoms and the natural history of oculopharyngeal muscular dystrophy (OPMD), a rare genetic muscle disorder. The key opinion leaders will also review the clinical and radiographic methods employed to evaluate disease progression and discuss the current treatment landscape for patients diagnosed with OPMD.
Bernard Brais , MDCM, PhD - Professor,Department of Neurology and Neurosurgery ,Montreal Neurological Institute ,McGill University Health Centre Emily Plowman , PhD, CCC-SLP, FASHA - Professor,Department of Otolaryngology - Head and Neck Surgery,The Ohio State University College of Medicine
The event will focus on the Company’s clinical development program for its investigational gene therapy agent BB-301, which is currently being evaluated in a Phase 1b/2a clinical trial for the treatment of OPMD-derived dysphagia. Key areas of focus will include discussions of clinical, radiographic, and subject-reported assessments of disease progression, clinical study design, and the specific primary and secondary endpoints that will be used to quantify subject improvement over the course of the BB-301 clinical development program.
A live question and answer session will follow the formal presentations.
About
About
About BB-301
BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting 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 are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein.
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Forward-Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec's plans to develop and commercialize its product candidates, the timing of the initiation and completion of pre-clinical and clinical trials, the timing of patient enrolment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec's product candidates, potential future out-licenses and collaborations, the intellectual property position and the ability to procure additional sources of financing, and other forward-looking statements.
These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company's ability to protect and enforce its patents and other intellectual property rights; the Company's dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company's ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of the COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus and similar events, which may adversely impact the Company's business and pre-clinical and future clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company's reports filed with the
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ikoffler@lifesciadvisors.com
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