Editas (EDIT) Focuses On Developing Eye Candidate EDIT-101

 | Jul 05, 2019 08:57AM ET

On Jul 3, we initiated a research report on Editas Medicine, Inc. (NASDAQ:EDIT) .

The company’s lead pipeline candidate, EDIT-101, which uses CRISPR gene editing, is being developed for treating Leber congenital amaurosis type 10 (LCA10), a rare genetic illness that causes blindness.

Editas is developing EDIT-101 in partnership with Allergan (NYSE:AGN) . The company has a strategic alliance and option agreement with Allergan under which, the latter reserves rights to in-license up to five of Editas’ genome editing ocular programs. Last July, Allergan exercised its option to develop and commercialize EDIT-101 globally while Editas exercised its right to a profit-sharing understanding with Allergan under which, the company will co-develop and equally divide the profits and losses from EDIT-101 in the United States.

Both companies plan to begin patient dosing in the phase I/II dose escalation study on EDIT-101 during the second half of 2019.

The company is also pursuing the development of CRISPR candidates for eye diseases other than LCA10, which include Usher Syndrome type 2A (USH2A) and the recurrent ocular Herpes Simplex Virus type 1 (HSV-1).

Last month, Editas commenced investigational new drug (IND) enabling activities for EDIT-301, an experimental CRISPR medicine, designed to treat sickle cell disease and beta-thalassemia by editing the beta-globin locus.

By the end of 2022, Editas targets to have three medicines in early-stage clinical development and at least two in or ready for late-stage studies.

Shares of Editas have gained 8% so far this year, underperforming the Zacks Investment Research

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