Bengaluru-based eye hospital to soon start human clinical trials of indigenous gene therapy for ocular diseases

In an effort to make gene therapy affordable, researchers from Narayana Nethralaya, a Bengaluru-based ophthalmic hospital, are all set to start human clinical trials of indigenous gene therapy for ocular diseases. This is the first such effort in the country, doctors claimed.

Gene therapy is a treatment modality to correct the pathologic effects of defective genes that are responsible for disease development.

Cost factor

“While gene therapy trials have been happening in Western countries for over 30 years, no trials have been done in India so far. Patients in our country are unable to get the benefits of the therapy because of the exorbitant costs. One injection of the therapy can cost ₹7 crore and above. Many Indian patients need gene therapy, and the cost can be reduced only if the technologies are developed indigenously,” said Rohit Shetty, hospital chairman.

Pointing out that the plan is to initially start with gene therapy as a solution for genetic corneal disorders such as keratoconus through indigenously developed adeno-associated virus (AAV) vector production and delivery, Dr. Shetty said, “The process has begun. We are awaiting the Centre’s approval to start the trials and are hopeful of completing the first phase of trials by early next year.”

Focused meeting

Naren Shetty, the hospital vice-chairman, said the announcement was made after a two-day focused meeting that was held in Bengaluru from July 22 to come out with a consensus on indigenous gene therapy trials. The meeting was attended by over 25 prominent clinicians and scientists from top national and international institutes in the country.

“At the meeting, our scientists showcased how their indigenously developed vectors and production process can significantly reduce the cost of gene therapy to 1/20^th-1/50^th of the international costs. The meeting also resulted in a consensus on establishing a national registry for rare ophthalmic diseases and identified additional diseases in India that urgently require gene therapy,” he said. 

Clinical grade vector production

Making a presentation on indigenous gene therapy, Arkasubhra Ghosh, a geneticist at Narayana Nethralaya Foundation’s GROW (Genes Repair and Regeneration in  Ophthalmic Workstation) Research Laboratory, said the process of starting the first human AAV gene therapy trials for corneal dystrophy and the first dual AAV vector trial for  Stargardt retinal disease had begun.

Dr. Ghosh, who is one of the authors of the National Guidelines for Gene Therapy Product Development and Clinical Trials, said, “The drug-making process is a major technological challenge, and we are the first in India with the clinical grade vector production.”

Dr. Rohit Shetty said patients seeking voluntary participation in gene therapy treatment could contact the hospital at  080-6666-0715 or email genetictest@narayananethralaya.com