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News, Research

First in-human US CRISPR trial targets blindness

31/07/2019By Callum Glennen
An upcoming US study will test the effectiveness of a treatment that utilises the CRISPR gene-editing technique to cure patients suffering from Leber congenital amaurosis.

The study is the first in vivo human trial of a CRISPR-based therapy to be performed in the US, and has the potential to prompt a wave of new treatments for many other conditions caused by genetic flaws.

The trial, which will include 18 people across the US and commence later this year, is being overseen by Allergan and Editas Medicine, co-developers of the treatment. The two companies entered into a strategic research and development alliance in 2017 in order to collaborate on several genome-editing ocular programs.

The treatment only needs to be performed once and is designed to cut or ‘edit’ a person’s DNA in a specific spot, the Associated Press reports. People born with Leber congenital amaurosis lack a gene needed to convert light into brain signals. Treatments are administered via an injection during a brief surgery.

David Nicholson
David Nicholson
“We are very proud of our continued commitment to developing innovative treatments for unmet needs in eye care”
David Nicholson, Allergan

“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.”

The trial is the first of its kind in the US to use CRISPR to edit DNA inside the human body. CRISPR-based treatments have commenced trials in the US this year, but the technology has not yet been used directly inside the human body.

CRISPR has generated excitement among researchers due to the ease it offers editing individual genes, but has also been the source of ethical quandaries. In 2018 Chinese researcher Dr He Jiankui used CRISPR to edit the embryonic genes of twin girls in an attempt to protect them from HIV.

His work was scorned my many in scientific community, since any changes to the genetic code in embryos would be passed on to the subject’s offspring, permanently altering the human genome.

The CRISPR trial being undertaken by Allergan and Editas Medicine does not involve any changes to genetic material that will be passed on to future generations.

This trial also follows the success of Luxturna, which has been approved by both the US Food and Drug Administration and the European Commission. Luxturna, which is also used to treat Leber congenital amaurosis, is also a form of gene therapy. Treatment for both eyes costs as much as US$850,000 (AU$1.2 m).


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