Drug Profile

Research programme: TDP-43 targeting small molecule therapeutics - Transition Bio/Voyager

Latest Information Update: 28 Nov 2025

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At a glance

Orphan Drug Status

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

No
New Molecular Entity Yes

10 Nov 2025 Transition Bio and Voyager enters into a collaboration and license option agreement to develop small molecules targeting TDP-43 for Amyotrophic lateral sclerosis and Frontotemporal dementia
10 Nov 2025 Early research in Amyotrophic lateral sclerosis in USA (unspecified route)
10 Nov 2025 Early research in Frontotemporal dementia in USA (unspecified route)

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