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“Landmark” CRISPR Trial For Genetic Blindness Results Released

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(CTN News) – CRISPR/Cas9 gene editing therapy has shown measurable improvement in 79 percent of patients with a rare form of inherited blindness, according to new results from a Phase I/II clinical trial.

There were 14 participants in the BRILLIANCE trial (12 adults and two children), each of whom received an injection of EDIT-101 into one eye.

There was a form of CRISPR Leber Congenital Amaurosis (LCA) in these individuals caused by mutations in the centrosomal protein 290 (CEP290) gene.

In light of the fact that traditional forms of gene therapy, such as gene augmentation, are not available for treating genetic blindness, this trial represents a landmark in the treatment of genetic blindness,”

Dr Tomas Aleman, the Irene Heinz-Given and John LaPorte Research Professor in Ophthalmology at Penn Medicine with the Scheie Eye Institute and a paediatric ophthalmologist at the Scheie Eye Institute at the Children’s Hospital of Philadelphia (CHOP), who is the principal investigator of the study and co-author of the study.

Learn more about the CRISPR-based gene editing therapy trial

A total of two adults received low-dose therapy, five received mid-dose therapy, and five more received high-dose therapy. Additionally, two children received a mid-dose of EDIT-101.

Over a period of twelve months, participants were monitored every three months to assess the safety of CRISPR-based gene editing medicine. The researchers confirmed that follow-up was less frequent for two additional years.

According to the study, published in The New England Journal of Medicine, there were no serious adverse effects or toxicities related to the treatment or procedure.

How might the future unfold?

“The results of the BRILLIANCE trial provide proof of concept and important learnings for the development of new and innovative medicines to treat inherited retinal diseases.

It has been demonstrated that CRISPR-based gene editing can be safely delivered to the retina and can have clinically meaningful results,” explained Dr Baisong Mei, PhD, Chief Medical Officer of Editas Medicine.

Hopefully, future studies will examine “ideal dosing, whether a treatment effect is more pronounced in certain age groups such as younger patients”, along with refined endpoints to measure the effects of improved cone function on activities of daily living,” according to the researchers.


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Alishba Waris is an independent journalist working for CTN News. She brings a wealth of experience and a keen eye for detail to her reporting. With a knack for uncovering the truth, Waris isn't afraid to ask tough questions and hold those in power accountable. Her writing is clear, concise, and cuts through the noise, delivering the facts readers need to stay informed. Waris's dedication to ethical journalism shines through in her hard-hitting yet fair coverage of important issues.

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