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

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On Jan 3, we issued a research report on Editas Medicine, Inc. (EDIT - Free Report) .

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. The disease has a significant unmet need as no therapies are presently approved to cure the same.

Editas is developing EDIT-101 in partnership with Allergan plc (AGN - Free Report) . The company has a strategic alliance and option agreement with Allergan under which, the latter reserves rights to in-license up to five genome editing ocular programs of the former including EDIT-101. Editas is entitled to receive potential milestone payments and royalties per the deal.

Editas and Allergan plan to begin patient dosing in the phase I/II dose escalation study called Brilliance on EDIT-101 for LCA10 in early 2020. The Brilliance study opened for patient enrolment in July 2019.

Shares of Editas have rallied 16.3% in the past year against the industry’s decrease of 1.9%.

Meanwhile, Editas is pursuing the development of CRISPR candidates for eye diseases other than LCA10 including Usher Syndrome type 2A (USH2A) and the recurrent ocular Herpes Simplex Virus type 1 (HSV-1). Outcomes from pre-clinical studies on these diseases have been positive, thereby providing the basis for developing a potential medicine.

The company initiated 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.

This apart, Editas has collaborations with big pharma players for its CRISPR technology, which provide research support and funds to fulfill its pipeline development. The company has a collaboration and licensing pact with Juno Therapeutics, a Celgene entity {now part of Bristol-Myers (BMY - Free Report) }, to use the latter’s gene-editing approaches including CRISPR-Cas9 for developing the engineered T cell medicines to treat cancer.

Editas also has a research and cross licensing agreement with BlueRock Therapeutics, LP, to combine the respective genome editing and cell-therapy technologies for discovering and developing novel engineered cell medicines. In October 2019, Editas signed a collaboration contract with AskBio, a leader in AAV gene therapy to develop in vivo CRISPR medicines for the treatment of neurological diseases.

Such collaboration deals provide regular funds to Editas for executing its pipeline activities.

We note that Editas currently has no approved product in its portfolio. As a result, the company is heavily dependent on its partners for collaboration revenues. Moreover, apart from EDIT-101, all of Editas’ pipeline candidates are still in preclinical or research stage of development and will take several years prior to commercialization. Stiff competition is another major concern as other companies, such as ProQR Therapeutics N.V. (PRQR - Free Report) are conducting clinical programs for treating LCA10 with respective product candidates.

Hence, any regulatory setback in Editas’ pipeline development might affect the stock.

Editas Medicine, Inc. Price and Consensus

Zacks Rank

Editas currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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