Editas, Allergan open first trial of in vivo CRISPR therapy

One of the four sites of the trial, in a rare form of inherited blindness, is currently listed as open for enrollment, and the companies plan to dose the first patient soon.


A clinical trial of a CRISPR/Cas9-based gene therapy in an inherited form of blindness is expected to dose its first patient in the second half of this year.

Cambridge, Massachusetts-based Editas Medicine and Dublin-based Allergan said Thursday that they had opened their Phase I/II study of AGN-151587 in Leber congenital amaurosis 10, also known as LCA10, for patient enrollment. The companies said it would be the first study of an in vivo medicine based on CRISPR-based genome editing, with which the editing process takes place inside the human body.

Last month, Chicago-based AbbVie said it would acquire Allergan – best known for manufacturing drugs like Botox (onabotulinumtoxinA) – for $63 billion.

LCA10 is caused by mutations in the CEP290 gene and is one of several subtypes of LCA. LCA generally has a prevalence of about one or two per 100,000 births and is a monogenic disease, with at least 27 genes implicated, according to the National Organization for Rare Disorders.

Allergan is the main sponsor of the study, described as a single ascending-dose study that will enroll 18 patients aged 3 to 17. The trial has four locations: the Bascom Palmer Eye Institute in Miami; the Massachusetts Eye and Ear Infirmary in Boston; the University of Michigan’s W.K. Kellogg Eye Center, in Ann Arbor; and Oregon Health and Science University’s Casey Eye Institute, in Portland. Of those, only the Boston site is open for recruitment so far.

“Now that enrollment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients,” Editas CEO Charles Albright said in a statement. “The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”

In February, Vertex Pharmaceuticals and CRISPR Therapeutics became the first company to dose a patient in a clinical trial of a gene therapy based on CRISPR/Cas9 technology, namely CTX001, in transfusion-dependent beta-thalassemia, a blood disorder. Unlike AGN-151587, CTX001 is an ex vivo therapy whose editing of patients’ hematopoietic stem cells takes place outside the body.

The first gene therapy to win Food and Drug Administration approval, Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), was approved for another inherited form of blindness, biallelic RPE65 gene mutation-associated retinal dystrophy. However, Luxturna is an adeno-associated viral vector-based therapy that uses a modified cold virus to insert a corrected gene into the cells of the retina, as opposed to editing the genome.

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