Editas grabs orphan drug status for sickle cell disease CRISPR therapy

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement.

The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023.

Editas had previously announced positive safety and efficacy data with two patients enrolled in the RUBY study, stating that the therapy was well-tolerated and that both patients were successfully engrafted and free of vaso-occlusive events during the follow-up.  The first patient had a total haemoglobin level of 16.4 g/dL and 45.4% fetal haemoglobin after five months, per a December 2022 update. Editas has plans to dose 20 patients on this study by the end of this year.

EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin. Editas’ CRISPR therapies represent a different approach to the gene editing technology than the one used by CRISPR Therapeutics, which relies on the CRISPR/Cas9 platform.

Earlier in the year, the company had announced a move towards developing treatments for hemoglobinopathies like sickle cell disease and beta thalassemia and a focus on in vivo discovery. In a January 9 press release, Editas stated it will be laying off roughly 20% of its workforce, alongside the discontinuation of its investments in its therapies for inherited retinal diseases.

Specifically, this meant that the company stopped investments into EDIT-101 and its use in Leber Congenital Amaurosis and EDIT-103 for rhodopsin-associated autosomal retinitis pigmentosa. Editas also discontinued investments in its preclinical natural killer (NK) cell program, which included candidates like EDIT-202 for use in solid tumours. On January 19, Editas announced that it entered into a definitive agreement with Shoreline Biosciences for the licensing of its natural killer cell program. Shoreline acquired EDIT-202 as part of this agreement.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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