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Patient Receives First Crispr Genetic Eye Treatment

Editing DNA

(LaCasadeGoethe, Pixabay)

4 May 2020. An individual with a rare genetic eye disease received the first dose of an edited gene in a clinical trial of an experimental treatment for the disorder. The patient at Oregon Health and Science University in Portland is being treated for Leber congenital amaurosis 10, or LCA10, with a therapy based on the gene-editing technique known as Crispr, the first individual to receive an edited gene treatment delivered into the body, according to its developers.

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.

The treatment being tested is code-named AGN-151587, the lead program of Editas Medicine in Cambridge, Massachusetts, a spin-off enterprise from the labs of geneticist Feng Zhang and colleagues from Broad Institute, a medical research center affiliated with Harvard University and MIT. The therapy was originally called EDIT-101 at Editas, but then renamed when licensed to drug maker Allergan, officially headquartered in Dublin, Ireland.

AGN-151587 uses the gene-editing technique called 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. In this case,the treatment delivers an edited CEP290 gene, without the disease-causing mutation, aiming to express proteins that restore normal retinal functions and vision.

The early- and mid-stage clinical trial is recruiting 18 individuals, both children and adults, at four sites in the U.S., divided into five groups testing AGN-151587 at various dosages injected into the retina. The researchers are looking primarily for safety and tolerability issues, such as adverse effects, within one year after treatment. But the study team is also gathering evidence of changes in vision in a series of both clinical assessments and functioning tests, such as navigating an obstacle course.

In a company statement, Editas Medicine CEO Cynthia Collins calls the first person receiving treatment in the trial “a significant milestone toward delivering on the promise and potential of Crispr medicines to durably treat devastating diseases such as LCA10.” Collins adds, “This dosing is a truly historic event: for science, for medicine, and most importantly for people living with this eye disease.”

The first recipient of AGN-151587 is a patient at the Casey Eye Institute at Oregon Health and Science University, one of the four trial sites. In November 2018, Science & Enterprise reported on Editas Medicine receiving approval for an investigational new drug application, technically a request to FDA to ship a non-approved drug across state lines in the U.S., but in effect permission to begin clinical trials of an experimental therapy, in this case AGN-151587.

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