Drugs / BB-301
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06185673 | Nov 2023 → Nov 2030 expected | oculopharyngeal muscular dystrophy | Benitec Biopharma, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 16
| Date | Issuer | Release |
|---|---|---|
| 2026-04-28 | Benitec Biopharma, Inc. | Results Benitec Biopharma Announces Oral Presentation of Interim Phase 1b/2a Clinical Study Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 at the 2026 American Society of Gene and Cell Therapy Annual Meeting benitec.com ↗
Interim clinical results for patients enrolled into Cohort 1 and Cohort 2 will be discussed in an oral presentation at ASGCT , being held in Boston, MA from May 11 – 15, 2026. |
| 2026-03-10 | Benitec Biopharma, Inc. | Results Benitec Biopharma to Host Industry Forum Breakfast Event at the 2026 Muscular Dystrophy Association Clinical and Scientific Conference on March 10, 2026, Highlighting Continued Progress for BB-301, the Only Clinical-Stage Therapeutic in Development for Oculopharyngeal Muscular Dystrophy (OPMD) benitec.com ↗
The presentation will highlight the key clinical results observed to date for OPMD Patients enrolled into the BB-301 Phase 1b/2a Clinical Treatment Study (NCT06185673) and the potential for BB-301 to transform the treatment landscape for patients with OPMD. |
| 2026-03-09 | Benitec Biopharma, Inc. | Results Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 Treatment at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference benitec.com ↗ |
| 2026-01-11 | Benitec Biopharma, Inc. | Results Benitec Biopharma Provides Positive Long-Term Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial Demonstrating Robust Efficacy and Continued Durability of Response benitec.com ↗
At the 24-month follow-up timepoint, Patient 1 continued to demonstrate robust, disease-modifying outcomes. |
| 2025-11-03 | Benitec Biopharma, Inc. | Results Benitec Biopharma Provides Positive Interim Clinical Study Results for BB-301 Phase 1b/2a Clinical Trial and Receives FDA Fast Track Designation for BB-301 benitec.com ↗
today provides positive interim clinical results for the BB-301 Phase 1b/2a Clinical Trial. |
| 2025-03-19 | Benitec Biopharma, Inc. | Results Benitec Biopharma Reports Positive Interim Clinical Results for Three Subjects Treated with BB-301 in Phase 1b/2a Study to be Presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference benitec.com ↗ |
| 2024-10-12 | Benitec Biopharma, Inc. | Results Benitec Biopharma Reports Positive Data from Two Subjects Treated with Low-Dose BB-301 in Phase 1b/2a Study Presented at 29th Annual Congress of the World Muscle Society benitec.com ↗
Subject 1 and Subject 2 experienced durable, clinically meaningful improvements in swallowing at 9-months and 6-months post-BB-301 treatment, respectively, with Subject 2 achieving a Sydney Swallow Questionnaire Score Representative of Clinically Normal Swallowing |
| 2024-07-15 | Benitec Biopharma, Inc. | Results Benitec Biopharma Reports Continued Durable Improvements in the Radiographic Assessments of Swallowing Efficiency and the Subject-Reported Outcome Instrument at the 180-Day Timepoint for First OPMD Subject Treated with Low-Dose BB-301 in Phase 1b/2a Study benitec.com ↗
Similar to the results observed for the radiographic swallowing study assessments of Total Pharyngeal Residue, the post-dose average Total SSQ Score was lower at the 180-day post-dose timepoint than at any point during the 9-month pre-dose assessment period |
| 2024-04-18 | Benitec Biopharma, Inc. | Results Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b/2a Study benitec.com ↗
BB-301 facilitated improvements across multiple measures of swallowing function in the first Phase 1b/2a clinical study subject as compared to pretreatment assessments conducted during the observational natural history portion of the study |
| 2023-06-26 | Benitec Biopharma, Inc. | Regulatory Benitec Biopharma Receives FDA Clearance of the IND for BB-301 for the Treatment of Oculopharyngeal Muscular Dystrophy benitec.com ↗ |
| 2021-09-08 | Benitec Biopharma, Inc. | Results Benitec Biopharma Provides Positive Updates on the BB-301 Pilot Dosing Study in Large Animals benitec.com ↗
The updated BB-301 analyses outlined today continue to demonstrate robust, dose-dependent target tissue transduction, dose-dependent transgene expression, and biologically significant knock-down of the target protein. |
| 2021-09-08 | Benitec Biopharma, Inc. | Regulatory Benitec Biopharma Provides Positive Regulatory Updates for the BB-301 Development Program; BB-301 Phase 1b/2a Clinical Trial to Begin in 2022 benitec.com ↗ |
| 2021-02-24 | Benitec Biopharma, Inc. | Results Benitec Biopharma Announces Successful Results from the Interim Analysis of the BB-301 Pilot Dosing Study benitec.com ↗
Low-Dose, Intermediate-Dose, and High-Dose BB-301 administration achieved similar levels of inhibition, with an average of 74% inhibition of PABPN1 expression observed across all doses |
| 2018-01-16 | Benitec Biopharma, Inc. | Regulatory Benitec receives U.S. Orphan Drug Designation for BB-301, its ddRNAi therapeutic for the treatment of oculopharyngeal muscular dystrophy benitec.com ↗
Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) today announced that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug Designation to BB-301 for the treatment of oculopharyngeal muscular dystrophy (OPMD). |
| 2017-11-27 | Benitec Biopharma, Inc. | Regulatory Benitec Biopharma provides update on OPMD orphan disease program benitec.com ↗ |
| 2017-01-17 | Benitec Biopharma, Inc. | Regulatory Benitec Receives Orphan Drug Designation in the European Union for BB-301, a ddRNAi Therapeutic in Development for the Treatment of Oculopharyngeal Muscular Dystrophy benitec.com ↗
Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is pleased to announce that the European Commission, based on a favourable recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP), has granted Orphan Drug Designation to BB-301 as an orphan medicinal product for the treatment of patients with oculopharyngeal muscular dystrophy (OPMD). |
All press releases naming this drug 26 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BB-301 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06185673 ↗ |
| Action | Restore | “a single RNA transcript that produces a codon-optimized, wildtype PABPN1 protein” NCT06185673 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “BB-301 is composed of an AAV9 capsid, AAV9PL, which delivers the gene of interest, comprised of a recombinant genome” NCT06185673 ↗ |
| Route | Intramuscular | “fixed number of intramuscular (IM) injections of BB-301” NCT06185673 ↗ |
| Target | PABPN1 | “2 short hairpin (sh)RNAs directed against the disease-causing mutant PABPN1 gene” NCT06185673 ↗ |