Research programme: small molecules therapeutics - Fulcrum Therapeutics
Latest Information Update: 23 Oct 2025
At a glance
- Originator Fulcrum Therapeutics
- Class Small molecules
- Mechanism of Action Gene expression modulators
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Orphan Drug Status
No
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Discontinued Blood dyscrasias; Fragile X syndrome; Musculoskeletal disorders; Neurological disorders
Most Recent Events
- 23 Oct 2025 Discontinued - Preclinical for Blood dyscrasias in USA (unspecified route)
- 23 Oct 2025 Discontinued - Preclinical for Musculoskeletal disorders in USA (unspecified route)
- 23 Oct 2025 Discontinued - Preclinical for Neurological disorders in USA (unspecified route)