Dosing Begins in First-in-Human Trial of CRISPR Therapy

Rare Daily Staff

Allergan and Editas Medicine said that investigators at Oregon Health & Science University Casey Eye Institute treated the first patient in a clinical study of an experimental gene editing therapy for a rare genetic form of blindness.

The BRILLIANCE clinical trial is a phase 1/2 study to evaluate AGN-151587 for the treatment of patients diagnosed with LCA10 and is the world’s first human study of an in vivo CRISPR genome editing medicine. The trial will assess the safety, tolerability, and efficacy of AGN-151587 in approximately 18 patients with LCA10.

Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is caused by mutations in the CEP290 gene and is the cause of disease in approximately 20 to 30 percent of all LCA patients.

AGN-151587, is a CRISPR-based experimental medicine under development for the treatment of Leber congenital amaurosis 10.  It is administered via a subretinal injection to deliver the gene editing machinery directly to photoreceptor cells

“This dosing is a truly historic event – for science, for medicine, and most importantly for people living with this eye disease,” said Cynthia Collins, president and CEO, Editas Medicine. “The first patient dosed in the BRILLIANCE clinical trial marks a significant milestone toward delivering on the promise and potential of CRISPR medicines to durably treat devastating diseases such as LCA10.”

In March 2017, Editas Medicine and Allergan entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicine’s genome editing programs for ocular diseases, including AGN-151587. Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicine’s option to co-develop and share equally in the profits and losses of two optioned products in the United States. 

Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicine’s CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a). 

In August 2018, Allergan exercised its option to develop and commercialize AGN-151587 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from AGN-151587 in the United States.

Photo: Cynthia Collins, president and CEO, Editas Medicine