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A Phase 1b/2a, Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

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Trial Profile

A Phase 1b/2a, Open-label, Dose Escalation Study to Evaluate the Safety and Clinical Activity of Intramuscular Doses of BB-301 Administered to Subjects With Oculopharyngeal Muscular Dystrophy With Dysphagia

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 20 May 2025

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At a glance

  • Drugs BB 301 (Primary)
  • Indications Oculopharyngeal muscular dystrophy
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Benitec Biopharma

Most Recent Events

  • 14 May 2025 According to a Benitec Biopharma media release, The sixth and final Subject of Cohort 1 was safely treated with the low dose of BB-301 in April 2025. We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year. Additional clinical study updates for Subjects enrolled in Cohort 1 are planned for the fourth calendar quarter of this year.
  • 19 Mar 2025 According to a Benitec Biopharma media release, an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, taking place in Dallas, Texas, on March 19, 2025 at 1:15 pm Central Time .
  • 19 Mar 2025 Interim results presented in the Benitec Biopharma Media Release.

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