Drug Profile

LETI 101

Alternative Names: LETI-101

Latest Information Update: 10 Apr 2025

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At a glance

Originator ElevateBio
Class Gene therapies
Mechanism of Action Huntingtin protein expression inhibitors

Orphan Drug Status

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

No
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Preclinical Huntington's disease

Most Recent Events

25 Feb 2025 Preclinical trials in Huntington's disease in USA (Intrastriatal), prior to February 2025
24 Feb 2025 LETI 101 is available for licensing as of 24 Feb 2025. http://www.elevate.bio
24 Feb 2025 ElevateBio received positive regulatory feedback from the UK’s MHRA regarding the CMC and development pathway

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