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Editas (EDIT) Focuses on Developing Gene-Editing Eye Drug

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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.

The company is evaluating EDIT-101 in the phase I/II BRILLIANCE study. Initial data from the same were announced in September 2021. Early observations from subjects treated in the mid-dose cohort of the study demonstrated clinical evidence of gene editing led by visual improvements.

Editas plans to complete dosing in the adult high-dose and pediatric mid-dose cohorts in the first half of 2022.

Shares of Editas have plunged 72.2% in the past year compared with the industry’s decrease of 35.1%.

Zacks Investment ResearchImage Source: Zacks Investment Research

Editas’ other pipeline candidates are also progressing well. The company is evaluating the safety and efficacy of EDIT-301 for treating sickle cell disease (“SCD”). It is currently enrolling participants in the phase I/II RUBY study and expects to begin dosing in the first half of 2022.

In December 2021, the FDA cleared the investigational new drug application for EDIT-301 to treat transfusion-dependent beta thalassemia (“TDT”). The company plans to begin clinical studies on EDIT-301 in TDT patients later in the ongoing year.

Editas currently has no approved product in its portfolio. Therefore, successful development of EDIT-101, along with other pipeline candidates, remains in key focus for the company. Moreover, Editas’ pipeline candidates are in early-stage development and years away from commercialization. Hence, any regulatory setback for the pipeline candidates will be a significant impediment for the company.

Meanwhile, stiff competition remains a headwind as several other companies, including CRISPR Therapeutics (CRSP - Free Report) and Intellia Therapeutics (NTLA - Free Report) , are engaged in developing candidates to address different indications using CRISPR/Cas9 gene-editing technology.

CRISPR Therapeutics’ lead candidate, CTX001, a CRISPR gene-edited therapy, is being developed for treating SCD and TDT. CRISPR Therapeutics expects to file regulatory submission for CTX001 in both indications by 2022-end.

Intellia Therapeutics is developing NTLA-2001, its lead program in vivo genome editing candidate, as a curative treatment for transthyretin amyloidosis. NTLA-2001 was the first candidate in Intellia Therapeutics’ pipeline to enter clinical development.

Zacks Rank & Stock to Consider

Editas currently carries a Zacks Rank #3 (Hold). A top-ranked stock in the biotech sector is AnaptysBio, Inc. (ANAB - Free Report) , which has a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

AnaptysBio’s loss per share estimates have narrowed 0.3% for 2022, over the past 60 days. The stock has rallied 25.3% in the past year.

AnaptysBio’s earnings have surpassed estimates in three of the trailing four quarters and missed the same on the other occasion.