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ABLE suggests key changes in ICMR gene therapy guidelines

The ICMR, which drafted the guidelines with inputs from the Directorate General of Health Services (DGHS) and the Department of Biotechnology (DBT) had announced the guidelines on July 29,2019 and sought public comments by August 1, 2019. ABLE has submitted detailed inputs to modify some of the key terminologies, added definitions and suggested changes to make the guidelines more comprehensive and support orderly development of the gene therapy sector.

“The guidelines for gene therapy product development have been well drafted and our congratulations to the team for bringing out these guidelines to fruition,” said Dr Kiran Mazumdar-Shaw, Chairman of ABLE and Biocon Group, in the ABLE’s submission to the ICMR. The Regenerative Medicines Group of ABLE compiled the inputs from biotech industry members.

ICMR has incorporated inputs received from ABLE and other experts and is expected to announce the final guidelines very soon.

Even though gene therapy has emerged as a potential treatment option for disorders that involve malfunctioning of a single gene as well as rare genetic diseases, there is barely any policy prescription in India to guide the research agenda and suggest regulatory requirements to scientists and companies developing such therapies.

“It provides for basic guidance for research involving human participants, including clinical trials, pertaining to the broad area of gene therapy covering all the technologies and processes. Disease specific guidelines will subsequently be developed as part of standard operating procedures for different conditions,” the guidelines says.

“It’s a guideline to help researchers and regulators as gene therapy is an emerging technology. It will facilitate the clearance of such therapies by the Drugs Controller General of India. The draft would be finalised after receiving public comments,” Renu Swarup, secretary, Department of Biotechnology, told Deccan Herald recently.

India has a large burden of monogenic diseases like haemophilia, thalassemia, sickle-cell anaemia, muscular dystrophy, retinitis pigmentosa, primary immunodeficiency in children, lysosomal storage disorders such as Pompe disease, Gaucher’s disease, haemangioma and cystic fibrosis.

So far, tertiary care hospitals in India have reported 450 rare diseases, but the epidemiological data remains inadequate. For instance, in India, haemophilia A is reported in 11,586 patients although the prevalence is estimated to be around 50,000 patients. Approximately 6-8 per cent of India’s population or about 70 to 80 million people are affected by various genetic disorders.

Some of ABLE’s inputs include, a clear definition of the requirements of GMP facility for gene therapy products, appropriate mention of use of new technologies such as CRISPR-Cas 9, guide RNA etc, inclusion of stem cell based modification of tissues into various organs as gene therapy products.

Some of other ABLE’s suggestions include that primary tissue collection and infectious disease evaluation must be done only in labs with appropriate accreditation ( eg NABL), additional pathogen testing can be done on imported cell lines,. Another suggestion is that a “ potency assay is not required to initiate early phase clinical trials, but it is recommended to have a well qualified assay to determine the dose to be used.”

ABLE hopes most of its well meaning inputs will be incorporated by ICMR so that the guidelines become really robust and serve the immediate needs of critical product development using gene therapies.

While welcoming the comprehensive Draft Guidelines for Gene Therapy Product Development and Clinical Trials announced by the Indian Council of Medical Research (ICMR), the Association of Biotechnology Led Enterprises(ABLE) has suggested some key changes to be incorporated in the final guidelines.

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