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Editas (EDIT) Focuses on Developing Gene Drug for Eye Disease

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Editas Medicine, Inc. (EDIT - Free Report) has made rapid progress in the development of its lead pipeline candidate, EDIT-101, which employs CRISPR gene editing, to treat Leber congenital amaurosis type 10 (LCA10) — a rare genetic illness that causes blindness.

Last month, the company announced initial data from the phase I/II BRILLIANCE study evaluating EDIT-101 for the treatment of blindness due to LCA10.

Preliminary results from the study were based on safety and efficacy data from the first two cohorts – the adult low-dose cohort and the adult mid-dose cohort.

In June 2021, Editas started enrollment in the first of two planned pediatric cohorts in the phase I/II BRILLIANCE study evaluating EDIT-101 for treating LCA10. Treatment in the adult high-dose cohort is currently ongoing while treatment in the pediatric mid-dose cohort has just begun.

This apart, Editas has commenced the phase I/II RUBY study to assess the safety and efficacy of another pipeline candidate, EDIT-301, for treating sickle cell disease. The company plans to dose the first patient in the RUBY study by the end of 2021.

Editas also plans to submit an investigational new drug filing to the FDA to begin clinical studies on EDIT-301 for the treatment of beta-thalassemia by 2021-end.

We note that Editas has no approved product in its portfolio at the moment. Therefore, successful development of EDIT-101, along with other pipeline candidates, remains in key focus for the company.

Stiff competition remains a headwind as several other companies like CRISPR Therapeutics (CRSP - Free Report) , Intellia Therapeutics (NTLA - Free Report) and Beam Therapeutics (BEAM - Free Report) are also engaged in developing candidates to address different indications using CRISPR/Cas9 gene-editing technology. Moreover, Editas’ pipeline candidates are in early-stage development and years away from commercialization. Hence, any regulatory setbacks for the pipeline candidates will be a major setback for the company.