To ensure that CRISPR gene editing technology is not misused as was done by a Chinese scientist who created the gene-edited babies recently that attracted global criticism, the Indian Council of Medical Research (ICMR) has framed national guidelines and regulations regarding the procedures and requirements to be followed for performing Gene Therapy in India.

CRISPR stands for clustered regularly interspaced short palindromic repeats are DNA sequences found within the genome of prokaryotes, and are used in genome editing along with enzymes called CRISPR-associated nucleases (most commonly Cas9).

Gene Therapy refers to the process of introduction, removal or change in content of an individual’s genetic material with the goal of treating the disease and a possibility of achieving long term cure.

Since this nascent field is emerging in India, the Government has proactively have come up with the National Guidelines for Gene Therapy Product Development and Clinical Trials to promote further research and streamline regulatory processes for future clinical trials using gene therapeutic products (GTPs).

As per the New Drugs and Clinical trial Rules (2019) the GTPs falls under ‘new drug’ and shall always be deemed to be ‘new drug’. Thus as per these rules framed jointly by Indian Council of medical Research and Department of Biotechnology (DBT), ‘academic trials’ are not applicable to clinical trials using GTPs.

"India has large burden of genetic disorders and unmet medical needs and gene therapy can prove to be a turning point in treatment of such disorders. However, it also brings along with it unique technical risks and ethical challenges," said an official from the ICMR.

She cited the creation of babies using germline gene editing by a Chinese scientist recently that has  fuelled a debate on ethical concerns regarding applications of gene therapy technologies.

"This also brought to forefront the requirement of stringent guidelines and regulations to prevent misuse and premature commercialization.

"Many countries around the world have developed rules and guidelines to regulate gene therapy trials.

Taking cognizance of situation, it was felt necessary to frame national guidelines and regulations to direct scientists and clinicians including industry regarding the procedures and requirements to be followed for performing gene therapy in India," she added.

It is proposed to establish Gene Therapy Advisory and Evaluation Committee (GTAEC) anchored at ICMR. GTAEC shall be an independent body of experts representing diverse areas of biomedical research, concerned government agencies and other stakeholders.

"This committee will be composed of a core group of scientists and clinicians in the sector, as well as representation of the government agencies (ICMR, DGHS, CDSCO, DBT, DST, MCI). For each disease area in GTP trials, specific clinical consultants with extensive disease specific expertise will be co-opted to aid in the decision-making process," as per the guidelines.

It says that biological material from humans can be procured only from clinics/hospitals that have an evaluation committee.

The EC must ensure that the Standard Operating Procedures (SOPs) are in compliance with the national guidelines. "Investigators should treat the biological material with utmost respect and adequate care to avoid its misuse. The institute needs to define SOPs for development, production; storage and disposal of the GTPs or its components should be as per the Regulations and Guidelines on Bio-safety of Recombinant DNA Research and Bio-containment 2017," says the guidelines.

Until 2017, almost 2600 gene therapy and 6 clinical trials have been conducted worldwide in 38 countries, of which 64.9 per cent were in USA, 23.2 per cent in Europe and approximately 6.5 per cent were in Asia.

Within Asia, China has reported about 84 gene therapy clinical trials, followed by Japan (44 trials) and South Korea (14 trials). The vast majority of gene therapy trials have addressed cancer (66.6 per cent), monogenic diseases (11.5 per cent), cardiovascular diseases (6.2 per cent) and infectious diseases (6.3 per cent)