Gene therapy: Human trials to begin in Karnataka for treatment of genetic disorders

Gene therapy work was started 10 years ago under the guidance of founder and former chairman Dr K Bhujang Shetty.
Image used for representational purposes only
Image used for representational purposes only

BENGALURU: Gene therapy human trials will be initiated in India for the first time by Narayana Nethralaya for treating genetic disorders -- keratoconus, corneal dystrophy and stargardt retinal disease. It is expected to start by December-end.

In a country like India where the prevalence of genetic disorders is one in 2,000 to one in 10,000 people, there is a need to study gene therapy for multiple diseases to not only provide a cure to currently considered incurable diseases but also reduce chances of genetic transmission from a parent to a baby, said Dr Rohit Shetty, chairman, Narayana Nethralaya.

On the sidelines of a press conference organised in Bengaluru on Wednesday, Dr Shetty explained that initial research was done to identify defective genes for multiple diseases for close to 5,000-6,000 people over a 10-year period. Twelve diseases with a higher prevalence, including corneal dystrophy, night blindness, stargardt retinal disease, keratoconus, dry eye disease, muscular dystrophy and sickle cell anaemia, have been identified to be worked upon on a priority basis. 

They have also identified close to 700-1,000 people suffering from these disorders to perform gene therapy. Further, they aim to cover 60-plus genetic disorders like Parkinson’s disease or Alzheimer’s, which remain incurable due to lack of research.

Doctors further explained that there are 70 lakh “rare and neglected” disease patients in India which require gene therapy. Currently, available gene therapies cost $1-3.4 million per patient, and are only done in developed countries. Until now, no trials were done in India. Indigenously developed vectors and production processes can reduce the cost to 1/20th - 1/50th of international costs.

Gene therapy work was started 10 years ago under the guidance of founder and former chairman Dr K Bhujang Shetty. So far, approximately Rs 150 crore has been pumped in by the organisation towards starting gene therapy procedures, with multiple laboratories set up to provide research-based patient outcomes.

What is gene therapy?
It is a technique that uses a gene to prevent or cure a disease or medical disorder. It works by adding new copies of genes that are broken, or by replacing a defective or missing gene in a patient’s cell with a healthy version of that particular gene. Both inherited and acquired disorders can be treated with gene therapy.

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