Drug Profile

Research programme: siRNA therapeutics - Generation Bio

Alternative Names: ctLNP-siRNA program

Latest Information Update: 17 Jun 2025

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At a glance

Originator Generation Bio
Class Small interfering RNA
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.

No
New Molecular Entity Yes

Highest Development Phases

Research Autoimmune disorders

Most Recent Events

15 May 2025 Generation Bio plans to submit first IND for Autoimmune disorders in the second half of 2026 (Generation Bio pipeline, may 2025)
07 May 2025 Early research in Autoimmune disorders in USA (Parenteral) before May 2025

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