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Benitec Biopharma Inc. (NASDAQ: BNTC) is a clinical-stage biotechnology company pioneering the use of its proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform to develop novel genetic medicines. Headquartered in Hayward, California, Benitec is focused on creating treatments for chronic and life-threatening human diseases through gene-silencing technology that targets and 'turns off' specific disease-related genes, offering sustained treatments, and even potential cures, with a single dose.
Benitec's ddRNAi technology combines RNA interference with gene therapy to deliver long-lasting silencing of disease-causing genes. This approach has significant advantages over traditional RNA interference methods, being easier to deliver, safer, more targeted, and more efficient. This transformative technology is protected by over 40 global patents and can potentially silence genes related to thousands of human diseases.
The company is advancing a robust pipeline of in-house and partnered drug development programs targeting conditions such as Oculopharyngeal Muscular Dystrophy (OPMD) and Hepatitis B Virus (HBV). One of its leading projects, BB-301, is designed to slow or halt the progression of OPMD by silencing the faulty mutant PABPN1 gene and replacing it with a functional version. The BB-301 program has shown promising preliminary results, with significant improvements in clinical trial endpoints.
Benitec has recently announced several operational updates, including the initiation of the first clinical trial using the 'Silence and Replace' platform and the successful closing of a $40 million PIPE financing to support the development and commercialization of BB-301. The company's financial condition shows a focused investment in research and development with notable participation from prominent investors.
For the latest updates on Benitec Biopharma's performance, projects, and developments, visit their official website at www.benitec.com.
Benitec Biopharma (NASDAQ: BNTC), a clinical-stage biotechnology company focused on gene therapy and developing genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference platform, has announced their participation in two upcoming investor conferences in December 2024.
The company will participate in the Piper Sandler Annual Healthcare Conference on December 4, 2024, at 12:00 PM EST in New York, featuring a fireside discussion and one-on-one meetings. Additionally, they will attend the Oppenheimer Movers in Rare Disease Summit on December 12, 2024, from 9:50-10:40 AM EST in New York, participating in a panel discussion and one-on-one meetings.
Benitec Biopharma (NASDAQ: BNTC) has released its Q1 2025 financial results and operational updates. The company reported positive interim clinical study data for BB-301 in treating Oculopharyngeal Muscular Dystrophy (OPMD). The first subject showed 35-40% improvement in dysphagic symptoms at 270 days, while the second subject achieved a clinically normal swallowing profile at 180 days. A third subject was treated in October 2024, with a fourth expected in December.
Financial results show no revenue for Q1 2025, with total expenses of $5.8 million. The company reported a net loss of $5.1 million ($0.48 per share) and held $67.8 million in cash as of September 30, 2024.
Benitec Biopharma (NASDAQ: BNTC), a clinical-stage biotechnology company focused on gene therapy and developing genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform, has announced their participation in the Guggenheim Securities Healthcare Conference. CEO Jerel A. Banks, M.D., Ph.D., will present at the conference on November 11, 2024, at 2:30 PM in Boston. The presentation will be conducted in a fireside chat format with opportunities for one-on-one meetings, and will be available via live webcast.
Benitec Biopharma Inc. (NASDAQ: BNTC) has announced updated investor webcast information for their BB-301 Phase 1b/2a Clinical Study update. The webcast is scheduled for October 14th at 8:30 am EDT, replacing the previously announced conference call information from October 12th.
The live webcast will feature a presentation of interim clinical study data, including insights from management and Dr. Emily Plowman, Professor at The Ohio State University College of Medicine. Investors can access the webcast through a provided link, and a replay along with presentation slides will be available on the company's website afterward.
Benitec Biopharma is a clinical-stage biotechnology company focused on developing novel genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.
Benitec Biopharma (NASDAQ: BNTC) reported positive interim data from its Phase 1b/2a study of BB-301 for Oculopharyngeal Muscular Dystrophy (OPMD). Two subjects treated with the low-dose BB-301 showed durable, clinically meaningful improvements in swallowing at 9 and 6 months post-treatment, respectively.
Key findings include:
- No Significant Adverse Events reported
- Subject 1 experienced a 35% reduction in Sydney Swallow Questionnaire (SSQ) Total Score and significant improvements in videofluoroscopic swallowing study (VFSS) assessments
- Subject 2 achieved an 89% reduction in SSQ Total Score, reaching a clinically normal swallowing profile
- Strong correlation between SSQ scores and VFSS Total Pharyngeal Residue results
These results represent the first reported successful improvements in swallow function using a novel gene therapy for OPMD. Benitec plans to treat a third subject with the low dose and enroll additional subjects at higher doses in 2025.
Benitec Biopharma Inc. (NASDAQ: BNTC) has released its full year 2024 financial results and operational update. Key highlights include:
- Positive 90-day and 180-day interim clinical trial data for the first OPMD subject dosed with low-dose BB-301
- Second subject dosed in February 2024, third expected in Q4 2024
- Closed $40.0 million private placement financing in April, extending cash runway through 2025
- Total revenues were $0 for FY2024, compared to $75,000 in FY2023
- Total expenses increased to $22.5 million in FY2024 from $19.2 million in FY2023
- Net loss of $22.4 million, or $5.51 per share, compared to $19.6 million, or $14.12 per share in FY2023
- Cash and cash equivalents of $50.9 million as of June 30, 2024
Benitec Biopharma Inc. (NASDAQ: BNTC) announced that a Late Breaking Abstract on BB-301 has been accepted for oral presentation at the 29th Annual Congress of the World Muscle Society in Prague, Czech Republic, from October 8-12, 2024. The presentation, scheduled for October 12, 2024, will focus on interim clinical data from a Phase 1b/2a study evaluating the safety and clinical activity of BB-301, an AAV9-based gene therapy for Oculopharyngeal Muscular Dystrophy (OPMD) with dysphagia. Professor Milan R. Amin, M.D., from the New York University Grossman School of Medicine, will present the findings. This presentation represents a significant milestone in Benitec's development of novel genetic medicines using their proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.
Benitec Biopharma (NASDAQ: BNTC) announced that CEO Jerel A. Banks, M.D., Ph.D., will participate in the Oculopharyngeal Muscular Dystrophy (OPMD) Awareness Day Webinar on September 23, 2024, at 12 PM EST. The event, organized by the OPMD Association, aims to raise awareness about OPMD, a rare, genetic, adult-onset, progressive myopathy.
Dr. Banks will provide an update on the BB-301 Phase 1b/2a clinical development program. The webinar will offer patients, families, and caregivers clinical insights, patient perspectives, and informational resources to support understanding and care for OPMD. Benitec Biopharma is a clinical-stage biotechnology company developing novel genetic medicines based on its proprietary 'Silence and Replace' DNA-directed RNA interference (ddRNAi) platform.
Benitec Biopharma (NASDAQ: BNTC) reports continued improvements in swallowing efficiency for the first OPMD subject treated with low-dose BB-301 in their Phase 1b/2a study. At the 180-day post-dose assessment, Total Pharyngeal Residue (TPR) remained meaningfully reduced compared to pre-dose values. For three of four food types, TPR values were lower than at any point during the 9-month pre-dose observation period.
The Sydney Swallow Questionnaire (SSQ) Total Score showed continued reductions in dysphagic symptoms, indicating improved swallowing function. Both TPR and SSQ scores are considered central markers for evaluating clinical improvement in OPMD patients. The company plans to dose the third subject in Q3 2024 and report additional interim results in Q1 2025.
Benitec Biopharma, a clinical-stage gene therapy-focused biotechnology company, has appointed Kishen Mehta to its board of directors, effective June 26, 2024. This follows a $40 million PIPE financing led by Suvretta Capital, where Mehta is a portfolio manager. CEO Jerel A. Banks emphasized Mehta's expertise in investing, corporate development, and strategy, particularly as the company advances its Phase 1b/2a study for BB-301, a treatment for Oculopharyngeal Muscular Dystrophy (OPMD). Mehta brings over 15 years of financial industry experience, including roles at Biohaven Pharmaceuticals, Surveyor Capital, and Adage Capital, with a focus on healthcare investments. He expressed enthusiasm for contributing to Benitec's mission to develop effective treatments for OPMD patients.
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