Editas Medicine’s EDIT-101 Clinical Trial Update Brings Mixed News on LCA10 (CEP290) Research

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Data from Editas Medicine’s clinical trial on gene-editing therapy showed meaningful outcomes and improvements to treat a form of Leber congenital amaurosis (LCA). The improvements, though, were seen in a limited patient population, prompting the biotech to pause the enrollment of new patients and look for a partner to continue research into the CRISPR-based treatment.

Editas, a clinical-stage genome editing company, announced results last month from its Phase 1/2 BRILLIANCE trial of EDIT-101, a CRISPR/Cas9 gene editing medicine under development to treat blindness caused by LCA10, a CEP290-related retinal degenerative disorder. Through gene editing, the trial is designed to repair a mutation affecting about 1,500 people living with LCA10 in the country.

Editas demonstrated proof of the trial’s design concept in this phase of the study by safely administering the new treatment to humans. The gene-editing therapy is delivered to photoreceptors by a subretinal injection and works like a pair of molecular scissors to cut out the mutation. The treatment contrasts to that of gene augmentation therapy, wherein a patient’s retina receives copies of a new gene to replace the defective copies.

In its research update, Editas reported that three of 14 study patients showed clinically meaningful improvements in best-corrected visual acuity (BCVA).

Gilmore O’Neill, President and CEO of the Cambridge, Mass., biotech business, detailed the clinical data in a company statement and via webcast, which can be viewed here.

“The results from the BRILLIANCE trial provide a proof of concept and important learnings for our inherited retinal disease programs. We’ve demonstrated that we can safely deliver a CRISPR-based gene-editing therapeutic to the retina and have clinically meaningful outcomes,” O’Neill said.

“While we will not progress EDIT-101 on our own and have made the decision to pause enrollment, we have the patient community top of mind and are looking for a collaboration partner to advance this program.”


Trial details

The trial update includes safety and efficacy data from all 14 patients treated to date in the study comprised of 12 adults and two pediatric patients. EDIT-101 was tolerated with no serious ocular adverse events or dose-limiting toxicities observed. Most adverse events were mild and expected for subretinal delivery.

Along with showing improvement in BCVA, three of the 14 people demonstrated consistent improvements in two of the following three additional endpoints: Full-field sensitivity test (FST), visual function navigation, which means navigating a mobility course to assess mobility and functional vision in people with an inherited retinal disease such as LCA10, and visual function quality of life, information derived from the patient or caregiver while participating in the study.

Of those three people, two of them each had two identical versions of the genetic marker for the CEP290 IVS26 mutation, meaning they were homozygous for that mutation.

Because LCA10 patients homozygous for CEP290 IVS26 represent an estimated 300 people in the United States, and no other baseline characteristics were identified in the study’s dataset, the Editas statement said, “[T]he company will not progress this program independently and will seek to identify a collaboration partner to continue the development of EDIT-101.

“As a result, Editas Medicine is pausing further enrollment in the BRILLIANCE trial and will continue long-term follow-up of all patients who have been treated to date.”


Stay informed, stay connected

Hope in Focus keeps the LCA community informed of various trials focused on finding cures for any one of the more than 27 forms of LCA, including people living with LCA10 and associated research into developing therapies to correct mutations in the CEP290 gene. LCA10 is the most common form of the rare disease, affecting 20 to 30 percent of all LCA patients. LCA affects about 1 in 33,000 people worldwide.

Hope in Focus Co-Founder and Board Chair Laura Manfre asks anyone interested in learning more about the Editas trial to please send us an email at info@hopeinfocus.org, and we would be happy to facilitate a conversation.

“Science, business, and people are all key to getting a treatment from the lab to the people who need it. The announcement from Editas Medicine is a win for science, most certainly. Being able to go into the back of the eye and safely edit a genetic mutation is nothing short of a historic milestone,” Manfre said.

“The announcement isn’t entirely what we hoped for, however, as Editas has paused its LCA10 trial. More needs to be learned on the science side of things, there are business and regulatory issues to address, and as always, access to the patient community is critical.

“This journey was never going to be a straight line, and setbacks, although disappointing, are just proof we’re still moving. Stay informed, stay connected, and please make sure you’re registered and up-to-date in My Retina Tracker®.”

It will be three years this July when Editas began its CRISPER/Cas9-based trial of EDIT-101, administered through a subretinal injection to reach and deliver the gene-editing machinery directly to photoreceptor cells.

Researchers designed the BRILLANCE Phase 1/2 clinical trial of EDIT-101 to assess the safety, tolerability, and efficacy of the potential treatment. Patients received a single dose of EDIT-101 under the retina in one eye. They are monitored every three months for a year after dosing and less frequently for two more years. More details about the trial can be found at here under the reference NCT#03872479.




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