Drug Profile

OTL 204

Alternative Names: OTL-204

Latest Information Update: 18 May 2026

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At a glance

Originator University of Padua
Developer Orchard Therapeutics; University of Padua
Class Antidementias; Gene therapies; Haematopoietic stem cells therapies
Mechanism of Action Gene transference; Progranulin protein replacements

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

Highest Development Phases

Preclinical Frontotemporal dementia

Most Recent Events

05 May 2026 OTL 204 is still in Preclinical for Frontotemporal dementia in the United Kingdom (Orchard Therapeutics pipeline, May 2026)
28 Dec 2024 No recent reports of development identified for preclinical development in Frontotemporal dementia in United Kingdom (Parenteral)
24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin

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