Benitec Biopharma to Host Virtual R&D Day to Discuss BB-301 Clinical Program in Oculopharyngeal Muscular Dystrophy on April 18, 2024

HAYWARD, Calif. — Benitec Biopharma Inc. (NASDAQ: BNTC), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (ddRNAi) platform, today announced it will host a virtual R&D Day, on Thursday, April 18, 2024 from 9:00 AM to 11:00 AM ET. To register, click here.

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 Bernard Brais, MDCM, PhD

Bernard Brais, MDCM, PhD, Professor, Department of Neurology and Neurosurgery at Montreal Neurological Institute (MNI), McGill University Health Centre (MGH, MNH) is Director of the Rare Neurological Diseases Group. He completed his MDCM, neurology residency and PhD at McGill. He is also trained as a historian of neurosciences and genetics. His research largely focuses on the genetic basis of neurogenetic disorders with founder effects in Quebec, with an increasing focus on disorders with ataxic manifestations such as Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay (ARSACS). Since 2007, he has headed a team of researchers on ARSACS. Dr. Brais has played important roles in identifying causal genes for Oculopharyngeal muscular dystrophy (OPMD), Hereditary Sensory and Autonomic Neuropathy type II (HSANII), Limb Girdle Muscular Dystrophy with Quadriceps atrophy (LGMD2L), Pol III-related leukodystrophies, and ZAK congenital myopathy.

About Emily Plowman, PhD, CCC-SLP, FASHA

Emily Plowman, PhD, CCC-SLP, FASHA, Professor, Department of Otolaryngology – Head and Neck Surgery, The Ohio State University College of Medicine is Director of the Aerodigestive Research Core across its two sites at the Ohio State University and University of Florida and Director of the Wexner Medical Center Dysphagia Program. She is an internationally recognized expert in the field of dysphagia who has held uninterrupted funding from the National Institutes of Health (NIH) since commencing her academic career in 2009. Her current research at OSU and UF are supported by the National Institute of Aging, National Institute of Nursing Research, National Institute of Cancer, Department of Defense, and the ALS Association. Dr. Plowman has authored over 85 peer-reviewed scientific manuscripts, given over 600 lectures worldwide, and obtained over 30 external research grants. In addition to her own research, Dr. Plowman is passionate about mentoring the future generation of clinician scientists and her mentorship efforts were recently recognized by the National Institutes of Health with the NINDS Story Landis Award for Outstanding Mentorship by a Neuroscientist (2022) and the University of Florida Doctoral Mentor of the Year award (2021). She was inducted into the American Speech and Hearing Association as a Fellow in 2022 and was elected to be the incoming President of the international Dysphagia Research Society for 2026.

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.

About Benitec Biopharma, Inc.

Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD).


Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387
[email protected]