DNA - directed RNA interference (ddRNAi)
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Citizens Lifts Benitec Biopharma Inc. (BNTC) Price Target on Topline Results for Oculopharyngeal Muscular Dystrophy Treatment
Yahoo Finance· 2025-11-18 11:16
Benitec Biopharma Inc. (NASDAQ:BNTC) is one of the best breakout stocks to invest in. On November 4, an analyst at Citizens JMP raised the stock’s price target to $22 from $20 while reiterating an Outperform rating. The price target hike follows topline clinical data results that showed a 100% response rate for BB-301 in Phase 1b/2a of the treatment for Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic muscle-wasting disease. Citizens Lifts Benitec Biopharma Inc. (BNTC) Price Target on Topline Res ...
Benitec Biopharma Announces Appointment of Sharon Mates Ph.D. to its Board of Directors
Globenewswire· 2025-11-03 12:00
Core Insights - Benitec Biopharma Inc. has appointed Dr. Sharon Mates to its Board of Directors, effective November 2, 2025, bringing extensive leadership experience in biotechnology [1][2] - Dr. Mates previously served as Chairman, CEO, and Co-founder of Intra-Cellular Therapies, which was acquired by Johnson & Johnson for approximately $14.6 billion in 2025 [2] - The company is focused on advancing its BB-301 program for Oculopharyngeal Muscular Dystrophy (OPMD) and expanding its proprietary "Silence and Replace" DNA-directed RNA interference platform [2][4] Company Overview - Benitec Biopharma is a clinical-stage biotechnology company headquartered in Hayward, California, specializing in novel genetic medicines [4] - The "Silence and Replace" platform combines RNA interference with gene therapy to silence disease-causing genes while delivering functional replacement genes [4] - The company is developing therapeutics for chronic and life-threatening conditions, including OPMD [4] Product Details - BB-301 is a novel gene therapy utilizing a modified AAV9 capsid to co-express a functional version of PABPN1 while silencing the mutant version [3] - The therapy aims to address a serious unmet medical need in OPMD by halting the expression of faulty proteins and providing a functional replacement [3]
Benitec Biopharma Releases Third Quarter 2025 Financial Results
Globenewswire· 2025-05-14 11:00
Core Viewpoint - Benitec Biopharma Inc. reported its financial results for the third fiscal quarter ended March 31, 2025, highlighting significant increases in expenses and net loss, while also providing updates on its clinical development of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD) [1][3][4]. Financial Highlights - Total expenses for Q3 2025 were $10.2 million, up from $4.1 million in Q3 2024, marking a 148.8% increase [3]. - Research and development expenses were $6.0 million compared to $2.6 million in the same quarter of the previous year, reflecting a 130.8% increase, primarily due to ongoing clinical development of BB-301 [3]. - General and administrative expenses rose to $4.2 million from $1.6 million year-over-year, an increase of 162.5% [3]. - The loss from operations for Q3 2025 was $10.2 million, compared to a loss of $4.1 million in Q3 2024, indicating a 148.8% increase in operational losses [4]. - Net loss attributable to shareholders for Q3 2025 was $9.4 million, or $0.24 per share, compared to a net loss of $4.3 million, or $0.23 per share, in Q3 2024 [4][7]. - As of March 31, 2025, the company had $103.6 million in cash and cash equivalents, a significant increase from $50.9 million as of June 30, 2024 [4][5]. Clinical Development Updates - The sixth and final subject of Cohort 1 was treated with a low dose of BB-301 in April 2025, with plans to enroll additional subjects at a higher dose later in the year [2]. - BB-301 is designed to treat dysphagia in OPMD patients by promoting co-expression of a functional version of the PABPN1 protein while silencing the expression of the faulty mutant version [8]. Company Overview - Benitec Biopharma Inc. is a clinical-stage biotechnology company focused on developing novel genetic medicines using its proprietary "Silence and Replace" DNA-directed RNA interference platform [9]. - The company aims to address chronic and life-threatening conditions, including OPMD, through innovative therapeutic approaches that combine RNA interference with gene therapy [9].