相关临床试验
2
0 进行中
药物批准
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成立时间
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已完成
1
50.0%
招募中
1
50.0%
暂无批准数据
- Benitec Biopharma reported that Patient 1 in their BB-301 Phase 1b/2a trial demonstrated deepening improvements in swallowing function at 24 months post-treatment, with a 60% reduction in post-swallow residue and 78% improvement in dysphagic symptom burden. - All four patients in Cohort 1 who completed the 12-month follow-up period met the pre-specified criteria for response to BB-301, demonstrating durable therapeutic effects of the gene therapy. - BB-301 utilizes a novel "Silence and Replace" mechanism targeting mutant PABPN1, the causative gene for oculopharyngeal muscular dystrophy, and has received Orphan Drug and Fast Track designations from the FDA. - The company plans to engage with the FDA in mid-2026 to confirm pivotal study design for BB-301, marking a significant step toward potential approval for this rare neuromuscular disorder.
- The FDA granted Fast Track Designation to Benitec Biopharma's BB-301 gene therapy for treating Oculopharyngeal Muscular Dystrophy (OPMD) on November 3, 2025. - This regulatory milestone could expedite the development and review process of the potentially groundbreaking gene therapy for this rare muscular disorder. - Benitec appointed Dr. Sharon Mates to its Board of Directors to bolster strategic direction, particularly in advancing the BB-301 program. - The company utilizes its proprietary 'Silence and Replace' DNA-directed RNA interference platform to silence disease-causing genes while delivering replacement genes.
- Benitec Biopharma's independent Data Safety Monitoring Board recommended continuation of the Phase 1b/2a clinical trial for BB-301 after completing safety review of all six subjects in Cohort 1. - The sixth and final subject of Cohort 1 was safely treated with the low dose of BB-301 in April 2025, demonstrating a benign safety profile with direct intramuscular delivery. - Following the positive safety recommendation, enrollment of Cohort 2 is expected to begin in Q4 2025, advancing the novel gene therapy for oculopharyngeal muscular dystrophy. - BB-301 utilizes Benitec's proprietary "Silence and Replace" platform, combining RNA interference with gene therapy to simultaneously silence mutant genes and deliver functional replacement proteins.
- Benitec Biopharma's BB-301 gene therapy demonstrates durable improvements in swallowing function in OPMD patients, according to interim clinical study data. - Five patients have been safely treated with the low dose of BB-301 in an ongoing Phase 1b/2a clinical trial, with a sixth patient expected to be dosed soon. - The company plans to initiate a higher dose cohort later this year, building on the positive safety and efficacy signals observed in the low-dose group. - An interim study update will be presented at the Muscular Dystrophy Association Clinical & Scientific Conference on March 19, 2025.
- Benitec BioPharma reports a 35% reduction in total score on the Sydney Swallow Questionnaire in the first patient treated with their gene therapy for oculopharyngeal muscular dystrophy (OPMD). - Therapeutic RNA editing has been clinically demonstrated in humans for the first time, according to the company's press release. - A phase 1 clinical trial initially cleared for lupus nephritis has expanded to include more eligible indications following IND amendments.
- Benitec Biopharma's BB-301 gene therapy demonstrated clinically meaningful improvements in swallowing for OPMD patients in a Phase 1b/2a trial. - The first patient showed a 35% reduction in the Sydney Swallow Questionnaire (SSQ) score after 270 days of treatment. - The second patient achieved an 89% improvement in SSQ Total Score, indicating a clinically normal swallowing profile after 180 days. - BB-301 leverages a 'Silence and Replace' mechanism targeting the mutant PABPN1 gene, with no significant adverse events reported.
