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: 11 Dec 2025

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At a glance

28 Apr 2025 According to an Axovia Therapeutics media release, company expects to initiate this trial in mid-2025.
22 Sep 2023 New trial record
19 Sep 2023 According to an Axovia Therapeutics media release, the company plans this study in the next 18-24 months.

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