ASGCT Symposium Catalyzes Discussions to Expand Reach of Gene Therapy in India

As part of its partnered educational events to address the challenges and opportunities in developing gene therapies in middle-income countries, ASGCT hosted Clinical Gene Therapy: ASGCT Indo-UK Symposium on October 9, a free virtual program for researchers and other stakeholders in India.

Planned by ASGCT’s Global Outreach Committee and held in collaboration with patient advocacy groups, professional associations, and academic institutions in India, the goal of the event was to discuss how to facilitate gene therapy clinical research as a step toward expanding patient access to these therapies in the country for the future.

India developed regulatory guidelines for gene therapy clinical trials in 2019, according to Geeta Jotwani, PhD, deputy director general at the Indian Council of Medical Research. Jotwani noted that these guidelines provide a framework to enable preclinical research guidance, manufacturing, and quality assurance for the initiation of gene therapy clinical trials while providing needed regulatory oversight. Some remaining regulatory needs include the development of disease-specific guidelines and streamlining the role of multiple regulatory authorities to facilitate the trials.

Indigenous gene therapy clinical trials are planned in the near future, but a CAR T-cell therapy trial in India has dosed five patients, according to Vikram Mathews, MD, professor in the department of hematology at Christian Medical College, Vellore. Mathews outlined specific requirements for site readiness for clinical trials and for a decentralized system for product manufacturing. He also provided an overview of the status of CAR-T cell therapies in the country. Persis Amrolia, PhD, talked about his journey thus far in CAR T cells for childhood ALL and ongoing work to address some of the issues encountered.

Jayandharan G. Rao, PhD, from IIT-Kanpur, stated that indigenous vector manufacturing is needed to promote local initiatives. Rao gave the example of pre-clinical studies carried out successfully in India where several indigenous technologies were developed for potential treatment of Leber congenital amaurosis, hemophilia and hepatocellular carcinoma. Federico Mingozzi, PhD, chief scientific officer at Spark Therapeutics, observed that biotechnology companies with early phase trials need to be able to follow subjects closely in almost real time, so they often need to have dosing sites close to them or where they have a close connection with the trial site. Early phase development may therefore be more likely to be indigenous, while there could be more opportunity for collaboration with multinational companies in later-phase trials.

Rangarajan, who has experience conducting several large trails in the UK, outlined her vision to bring affordable gene therapies for diseases such as hemophilia to India. She advocated for the involvement and education of local clinicians and encouraged them to participate in such studies. The government of India views emerging therapies as a high priority, according to Vinod Paul, MD, PhD, member (health) of the National Institute for Transforming India (NITI) Aayog, a key governmental entity. Paul emphasized there is excitement to work with industry, encouraged global collaboration among stakeholders, and emphasized the need for organizations such as ASGCT to partner with Indian research fraternity to upscale such preclinical initiatives.

The event’s co-organizers, Rangarajan and Rao, concluded the event with the hope that this symposium catalyzes discussions and a framework for engagement with preclinical, clinical, and industry stakeholders with handholding from international partners to expand the reach of gene and cell therapy in India. India may be joining the party a bit late but given that it is the “pharmacy” of the world with its capabilities in generic, biosimilars, and vaccine manufacturing, it can certainly leapfrog and stand shoulder to shoulder with the international fraternity.

The full event is available on demand on ASGCT’s YouTube channel.

Dr. Rangarajan is an associate professor of clinical hematology and an honorary consultant at  Southampton General Hospital, a director at KJ Somaiya Hospital, and a member of ASGCT's Global Outreach Committee. Dr. Rao  is a professor at the Indian Institute of Technology and a member of ASGCT's Global Outreach Committee and International Committee.