Editas & Allergan Dose First Patient in Phase I/II Historic Gene Editing Study—Accomplished via In Vivo Treatment

Mar 6, 2020 | Allergan, CRISPR, Editas, Gene Editing, Leber congenital amaurosis 10

Editas & Allergan Dose First Patient in Phase III Historic Gene Editing Study—Accomplished via In Vivo Treatment

Editas Medicine, Inc. (EDIT) and Allegan plc (AGN) announced they have dosed the first patient in a Phase I/II study called BRILLIANCE to evaluate their CRISPR-based candidate, AGN-151587 (EDIT-101), in patients with Leber congenital amaurosis 10 (LCA10), an inherited form of blindness. A historical milestone as for the first time a patient’s genes are actually modified within the body—in vivo treatment. Until now , gene editing operated on a ex vivo basis—targeted cells are taken from the patient, modified and thereafter returned. This pathbreaking study assesses EDIT-101 on 18 LCA patients.

The Disease

LCA10 (Leber congenital amaurosis 10) is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy. The visual impairment tends to be stable, although it may worsen very slowly over time. Leber congenital amaurosis occurs in 2 to 3 per 100,000 newborns. It is one of the most common causes of blindness in children. Currently, there are no approved therapies for treating LCA10. The sponsors/collaborators believe that as there is significant unmet need, the two can reap large profits from this drug if successfully commercialized.

The Study

The study, sponsored by Allergan and collaborator Editas, evaluates the safety, tolerability and efficacy of a single escalating doses of AGN-151587 (EDIT-101) administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene (“LCA10-IVS26”).

Research Sites

This study is being conducted at the following research sites: Bascom Palmer Eye Institute, Miami, FL; Massachusetts Eye and Ear Infirmary, Boston, MA; W.K. Kellogg Eye Center, University of Michigan, Ann Arbor, MI and Casey Eye Institute—OSHU, Portland, OR.

Enrollment this study, known as BRILLIANCE, was initiated in July 2019. Representing a historical moment, the study is the first to evaluate a CRISPR genome editing medicine in vivo in LCA10 patients. The five-cohort study evaluates three dose levels of the drug EDIT-101—the lead candidate in Editas’ pipeline. 

What is EDIT-101?

This CRISPR-based candidate was developed by Editas as part of a strategic alliance and option agreement with Allergan. This agreement grants Allergan an option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including this drug. As Allergan exercised its exclusive rights to EDIT-101 in 2018, both of them will share profit/loss equally from any sales in the U.S. should it be successfully commercialized.


Another venture is developing an experimental product treating LCA10, currently conducting clinical trials—ProQR Therapeutics, N.V. (PRQR). They 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 EDIT-101, reports Yahoo Finance.

Editas Medicine

Editas Medicine is a leading genome editing company focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (Cas12a) genome editing systems into a robust pipeline of medicines for people living with serious diseases around the world. Headquartered in Cambridge, MA, they have a significant presence in Boulder, CO.


Although the venture has huge promises and a number of strategic alliances, it burns through considerable finances.  With losses of about $138 million and its market capitalization of $1.38 billion ($25.15 per share)m it needs large amounts of capital—however, it does posses $457 million cash in the bank.


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