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A First-In-Human, Open Label, Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of a Single Dose of AXV-101 in Patients With Bardet-Biedl Syndrome 1 (BBS1) Bi-Allelic Mutations and Retinal Degeneration

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Trial Profile

A First-In-Human, Open Label, Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of a Single Dose of AXV-101 in Patients With Bardet-Biedl Syndrome 1 (BBS1) Bi-Allelic Mutations and Retinal Degeneration

Status: Not yet recruiting
Phase of Trial: Phase I

Latest Information Update: 17 Feb 2026

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At a glance

  • Drugs AXV-101 (Primary)
  • Indications Bardet-Biedl Syndrome; Retinal degeneration
  • Focus Adverse reactions; First in man
  • Acronyms AXIS
  • Sponsors Axovia Therapeutics

Most Recent Events

  • 06 Jan 2026 According to Axovia Therapeutics media release, the company has received $1.1 million funding A Race Against Blindness ,to support this study .the study is expected expected to begin in early 2026.This latest funding brings A Race Against Blindnesss total commitment for AXV-101 to $5.1 million.
  • 04 Sep 2025 According to Axovia Therapeutics media release,it has received $1.0 million grant from A Race Against Blindness, to support the clinical development of AXV-101, The new funding will contribute to the execution of the first-in-human open-label dose-escalation trial for AXV-101, which is expected to commence shortly in the United Kingdom, following the recent Clinical Trial Application (CTA) clearance.
  • 28 Apr 2025 According to an Axovia Therapeutics media release, company expects to initiate this trial in mid-2025.

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