June 19, 2024

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Race for gene-editing therapies heats up

Race for gene-editing therapies heats up


Race for gene-editing therapies heats up.

On June 9, two gene-editing therapies announced the positive results of clinical trials. The gene-editing therapy race is heating up.



Race for gene-editing therapies heats up




Editas Medicine

Editas Medicine announced early data from the RUBY and EdiTHAL trials demonstrating the potential of its gene-editing therapy EDIT-301 in sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).


EDIT-301 is an experimental cell therapy drug consisting of patient-derived CD34+ hematopoietic stem and progenitor cells that are expressed at the gamma globulin gene (HBG1 and HBG2) promoters via a highly specific and highly efficient proprietary engineered AsCas12a nuclease editing at the promoter that is the site of naturally occurring fetal hemoglobin (HbF)-induced mutations.


Preliminary safety and efficacy data were positive for the first four patients with sickle cell disease treated with EDIT-301 in the RUBY trial and the first transfusion-dependent beta-thalassemia patient treated in the EdiTHAL trial.



Vertex Pharmaceuticals and CRISPR Therapeutics

Vertex Pharmaceuticals and CRISPR Therapeutics also posted positive data for their sickle cell disease and transfusion-dependent beta thalassemia drug exagamglogene autotemcel ( exa-cel ), cementing their positions as frontrunners in the gene editing space. The two partners announced that the FDA has accepted the Biologics Marketing Application ( BLA) for exa-cel in these indications .

Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-editing cell therapy, which has been granted Regenerative Medicine Advanced Therapy (RMAT) for sickle cell disease and transfusion-dependent β-thalassemia by the US FDA, Fast Track, Orphan drug and rare pediatric disease designations. exa-cel will receive a ruling from the FDA by December 8, 2023. The target action date for transfusion-dependent beta thalassemia is March 30, 2024.

Two pivotal trials of exagamglogene autotemcel (exa-cel) in patients with transfusion-dependent beta thalassemia or severe sickle cell disease met primary and key secondary endpoints at pre-assignment interim analyses.

Joining these companies in the sickle cell disease race is Bluebird Bio, which filed a BLA for its gene therapy candidate lovotibeglogene autotemcel (lovo-cel) in April 2023, following the FDA’s December 2021 Some clinical trials were suspended due to safety concerns. The hold is set to be lifted in December 2022.

In recent months, though, a number of other players have dropped out of the sickle cell disease race, including Graphite Bio and Novartis and its partner Intellia. Sanofi also withdrew from the sickle cell disease project cooperation with Sangamo in January 2022.


(source:internet, reference only)

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