Drug Profile

AAV2 U1a hELP1 - Tikun Therapeutics

Alternative Names: AAV2-U1a-hELP1; rAAV2-U1a-hELP1

Latest Information Update: 05 Mar 2025

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At a glance

Originator Tikun Therapeutics
Class Eye disorder therapies; 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 - Optic nerve disorders
New Molecular Entity Yes

Highest Development Phases

Preclinical Optic nerve disorders

Most Recent Events

22 Jan 2025 Preclinical trials in Optic nerve disorders in USA (Intravitreous)
22 Jan 2025 AAV2 U1a hELP1 - Tikun Therapeutics receives Orphan Drug status for Optic nerve disorders in USA
22 Jan 2025 AAV2-U1a-hELP1 receives Rare Pediatric Disease Designation for Optic nerve disorders in the US

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