Editas Medicine, Inc. ( EDIT Quick Quote EDIT - Free Report) and Allergan plc announced that they have dosed the first patient in a phase I/II study — BRILLIANCE — evaluating their CRISPR-based candidate, AGN-151587 (EDIT-101), in patients with Leber congenital amaurosis 10 (LCA10), an inherited form of blindness.
Enrollment in the BRILLIANCE study was initiated in July 2019. The study is the first to evaluate a CRISPR genome editing medicine in vivo, or inside the body in LCA10 patients. The five-cohort study will evaluate three dose levels of AGN-151587 in pediatric as well as adult patients. AGN-151587 is the lead candidate in Editas’ pipeline.
The CRISPR-based candidate was developed by Editas as part of a strategic alliance and option agreement with Allergan. The agreement grants Allergan option to license up to five of Editas Medicine’s genome editing programs for ocular diseases including AGN-151587. Allergan exercised its option to gain exclusive rights to AGN-151587 in 2018. Please note that Allergan and Editas are co-developing the candidate and will share profit/loss equally from any sales in the United States, following its successful development.
Share of Editas have declined 3.4% in the past year compared with the
industry’s decrease of 9.7%.
Currently, there are no approved therapies for treating LCA10. With a significant unmet need, AGN-151587 can reap huge profits, if developed successfully and eventually approved. However, another company, ProQR Therapeutics N.V. (
PRQR Quick Quote PRQR - Free Report) is also conducting a clinical study for treating LCA10 with its own product candidate. The company initiated a phase II/III study on its candidate last year. Data from the study can be used for filing regulatory application seeking the candidate’s approval. An earlier approval to ProQr’s candidate will likely have an unfavorable impact on AGN-151587’s prospects.
Editas is also 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) fibrosis. It is also designing novel medicines for non-malignant hematologic diseases, such as sickle cell disease and beta-thalassemia.
Meanwhile, Editas has some high-profile collaborations with big pharma entities for its CRISPR technology, which provide research support and sufficient funds to fulfill its pipeline development plans. Apart from Allergan, it has a collaboration and licensing pact with another big pharma company, Celgene — now part of Bristol-Myers (
BMY Quick Quote BMY - Free Report) — to use the latter’s gene-editing approaches including CRISPR-Cas9 for developing the engineered T cell medicines to tackle cancer.
Such collaboration deals provide regular funds to Editas for executing its pipeline activities.
Editas currently carries a Zacks Rank #3 (Hold). You can see
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