Drug Profile

AB 1003 - Asklepios BioPharmaceutical

Alternative Names: AB-1003 - Asklepios BioPharmaceutical; LION-101

Latest Information Update: 15 Oct 2025

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At a glance

Originator Asklepios BioPharmaceutical
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Limb girdle muscular dystrophies
New Molecular Entity No

Highest Development Phases

Phase I/II Limb girdle muscular dystrophies

Most Recent Events

10 Oct 2025 Adverse event data from phase I/II trial in Limb girdle muscular dystrophies released by Asklepios BioPharmaceutical
07 Nov 2024 AB 1003 - Asklepios BioPharmaceutical receives Orphan Drug status for Limb girdle muscular dystrophies in USA
07 Nov 2024 AB 1003 receives Rare Pediatric Disease designation from the US FDA for Limb girdle muscular dystrophies

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