Drug Profile

Research programme: in vivo HSC editing medicines - Editas Medicine

Latest Information Update: 31 Mar 2025

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At a glance

Originator Editas Medicine
Class Antianaemics; Gene therapies; Haematopoietic stem cells therapies
Mechanism of Action Cell replacements; Fetal haemoglobin expression stimulants; Gamma-globin expression modulators; 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 No

05 Mar 2025 Preclinical trials in Beta-thalassaemia in USA (Parenteral) prior to March 2025
05 Mar 2025 Preclinical trials in Sickle cell anaemia in USA (Parenteral) prior to March 2025
05 Mar 2025 Pharmacodynamics data from a preclinical study in beta thalassemia and sickle cell anaemia released by Editas Medicine

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