CMC Vellore to conduct 1st clinical trial in haemophilia gene editing

Gene editing is a very powerful technology as over 50 million people live in India today with Mendelian rare diseases and many of these are in some sense theoretically amenable to genome editing which would cure them.

Serial entrepreneur Vijay Chandru, who is on the research council of the Christian Medical College (CMC) Vellore’s Centre For Stem Cell Research, said that the first clinical trial in gene editing in hemophilia is expected to take place in the country by 2019.

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“I am working with CMC Vellore group to bring about the first trial in hemophilia which will probably happen in 2019,” he said while participating in a panel discussion at the ‘Conclave on Managing Disruptive Technologies’ organized by the Institute of Management Technology (IMT) and Cerebra Skills Academy Pvt Ltd

While a disruption in the area of gene editing is already taking place in the world, the major challenge lies the absence of regulatory mechanisms as well as the fact that it is very expensive in nature.

He pointed out that it would cost anywhere between $2-4 million to get a gene edited, which means it won’t be accessible to many in its current form.

Recently, Medical Dialogues has reported that a Chinese researcher, He Jiankui has claimed to develop the world’s first genetically modified human — twin girls whose DNA has been altered with a powerful new tool capable of rewriting the very blueprint of life that would not contract HIV. But it has triggered controversies also.

While Chandru rooted for a proper regulatory framework for gene editing, he said it would be beneficial in many areas such as plants that would be able to generate more oxygen or microbes that would clean up the environment.

Genome editing or gene editing is a technology that gives scientists the ability to change an organism's DNA which allows genetic material to be added, removed, or altered at particular locations in the genome.