An upcoming human study will use CRISPR genome editing technique to treat an eye disorder that causes blindness. The study will be conducted in the US later this year, and for now, 18 people, ages three and above will be part of it. This study is the first CRISPR therapy to be performed in the US.
The trial is being supervised by a collaboration of pharmaceutical companies, Allergen and Editas Medicine, co-developers of the treatment. Both the companies signed a strategic research and development alliance in 2017 to cooperate on several genome editing optical programs.
According to Associated Press reports, the treatment requires to be performed only once and is planned to cut or alter a person’s DNA at a particular spot. Leber congenital amaurosis patients lack a gene needed to convert light into brain signals. During a short surgery, injections are used for the treatment.
This is a breakthrough which drives the likelihood of a string of new treatments for other genetic related diseases. “We are very proud of our continued commitment to developing innovative treatments for unmet needs in eye care,” Dr. David Nicholson, chief research and development officer at Allergan, said.
“Beginning patient enrolment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine for people with [Leber congenital amaurosis]10.”
Many CRISPR based treatments have started trials in the US, but this trial is unprecedented in the US as it uses CRISPR to edit DNA inside the human body. CRISPR has not only generated a buzz of excitement among researchers but has also invited a lot of criticism. In 2018 Chinese researcher Dr. He Jiankui used CRISPR to edit the embryonic genes of twin girls in an effort to protect them from HIV. His work was severely criticized by many in the scientific circle since any modifications to the genetic code in embryos would be passed on to the subject’s offspring, permanently altering the human genome.
However, this trial administered by Allergan and Editas Medicine does not involve any changes to genetic material that will be handed on to future generations.
Previously a non-CRISPR gene therapy for LCA called Luxturna was used, which includes vaccinating a reformed virus that transports the corrected gene into the retina’s cells. Both the US Food and Drug Administration and the European Commission has approved the treatment. Treatment for both eyes costs as much as a whopping US$850,000.