Trials OK’d for Inherited Eye Disease Therapy with Crispr

Crispr-Cas9 illustration (LBL.gov)

30 Nov. 2018. The company developing a treatment for a rare inherited eye disease using the genome editing technique Crispr received authorization from the Food and Drug Administration to begin clinical trials. Editas Medicine Inc. in Cambridge, Massachusetts says FDA approved its investigational new drug application for the experimental therapy code-named EDIT-101 to treat Leber congenital amaurosis type 10, or LCA10.

Editas Medicine Inc. was founded in 2013 by geneticist Feng Zhang and colleagues from Broad Institute, a medical research center affiliated with Harvard University and MIT, to commercialize research from their labs on genome editing known as Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is based on bacterial defense mechanisms that use ribonucleic acid or RNA to identify and monitor precise locations in DNA. The actual editing is done by enzymes such as Cas9 or Cpf1.

EDIT-101 is the lead program in Editas Medicine’s pipeline. Leber congenital amaurosis is an inherited disorder that results from mutations in at least 18 genes. The condition affects infants, occurring in 2 to 3 in 100,000 newborns, but is still considered one of the more common causes of blindness in children. Leber congenital amaurosis primarily affects the retina. causing severe visual impairment beginning in infancy. LCA10, a variation of the disease, is traced to a mutation in the CEP290 gene, accounting for 20 to 30 percent of individuals with Leber congenital amaurosis.

Editas Medicine is developing EDIT-101 with drug maker Allergan, headquartered in Dublin. As reported by Science & Enterprise, Allergan first received an option to license EDIT-101 in March 2017, but in August of this year, decided to fully license the technology. Under the agreement, Allergan is developing and commercializing EDIT-101 worldwide, while Editas is co-developing the treatment in the U.S., sharing those costs and profits equally.

An investigational new drug application is technically a request to FDA to ship a non-approved drug across state lines in the U.S., which companies need to send experimental drugs to clinical trial sites. To receive authorization for these shipments, companies are required to show the new drug was tested in animals for toxicity and other safety measures. Upon approval, the new drug is, in effect, cleared to begin clinical trials and becomes subject to the agency’s drug regulatory system.

The clinical trial for LCA10, says the company, is likely to enroll 10 to 20 participants in an early- and mid-stage study of EDIT-101’s safety, tolerability, and efficacy. One of the deal’s financial provisions entitles Editas to a $25 million payment when FDA approves an investigational new drug application for EDIT-101. Editas says it now earned that payment from Allergan.

More from Science & Enterprise:

• Appeals Court Backs Broad Institute in Crispr Case
• More Damage Detected from Crispr-Cas9 Edits
• UC-Berkeley Gains U.S. Crispr-Cas9 Patent
• FDA Delays Crispr Trial for Inherited Blood Diseases
• Crispr Deployed to Correct Inherited Vision Disorder

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