Drug Profile

TTX 381

Alternative Names: RGX-381; TTX-381

Latest Information Update: 09 Sep 2025

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At a glance

Originator REGENXBIO
Class Gene therapies; Neuroprotectants
Mechanism of Action Gene transference; Tripeptidyl-peptidase 1 replacements

Orphan Drug Status

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

Yes - Neuronal ceroid lipofuscinosis
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Phase I/II Neuronal ceroid lipofuscinosis

Most Recent Events

24 Jul 2025 TTX 381 receives Innovation Passport designation from U.K. MHRA for Neuronal ceroid lipofuscinosis
03 Jun 2025 TTX 381 receives Regenerative Medicine Advanced Therapy (RMAT) status for Neuronal ceroid lipofuscinosis in USA
03 Jun 2025 Tern therapeutics continues engagement with the FDA to accelerate the development of TTX 381

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