Drug Profile

Renizgamglogene autogedtemcel - Editas Medicine

Alternative Names: EDIT-301; Ex vivo gene edited cell medicine - Editas Medicine; Reni-cel

Latest Information Update: 12 Dec 2024

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At a glance

Originator Editas Medicine
Class Antianaemics; Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
Mechanism of Action Cell replacements; Gene transference

Orphan Drug Status

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

Yes - Sickle cell anaemia; Beta-thalassaemia
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Discontinued Beta-thalassaemia; Sickle cell anaemia

Most Recent Events

09 Dec 2024 Updated efficacy and adverse events data from the phase I/II RUBY trial in Sickle cell anaemia released by Editas medicine
07 Dec 2024 Efficacy data from a phase I/II/III trial in Sickle cell anaemia presented at the 66^thAmerican Society of Hematology Annual Meeting and Exposition
01 Dec 2024 Discontinued - Phase-I/II for Beta-thalassaemia in USA (IV)

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