Mass General Brigham’s top gene therapy expert delivered a stark message: the future of medicine is genetic, but bridging laboratory success to patient treatment remains a critical challenge, in a seminar series event organised by Dept of Animal Biology, School of Life Sciences, University of Hyderabad and Federation of Asian Biotech Associations (FABA), last Tuesday
“We’re standing at a crossroads,” said Dr. Roger Hajjar, Director of MGB Gene and Cell Therapy Institute, addressing a packed gathering. “The scientific breakthroughs are here, but turning them into accessible treatments requires rethinking our entire approach.”
Since 2018, gene therapy has seen unprecedented success, particularly in oncology and rare diseases. The recent FDA approval of Elevidys for Duchenne muscular dystrophy and new treatments for sickle cell disease underscore this progress. Dr. Hajjar illustrated this potential through a compelling case study of SERCa deficiency, where gene therapy dramatically improved patient outcomes.
His own groundbreaking work in cardiovascular gene therapy targets heart failure, a growing crisis in aging populations. Using modified adeno-associated virus vectors, his team has successfully targeted abnormal calcium signalling in cardiac cells, with early human trials showing promise.
However, Dr. Hajjar warned of what he calls the “Valley of Death” – the gap between promising research and clinical application. “Prohibitive manufacturing costs, complex regulations, and funding challenges kill most breakthrough therapies before they reach patients,” he explained. His solution, the “Rise” model, proposes resource sharing and industry collaboration to bridge this gap.
The emergence of circular RNA as a therapeutic tool opened new possibilities. Its unique properties – prolonged expression and immunoquiescent nature – make it valuable for both rare genetic diseases and cancer treatment.
A panel discussion led by Dr. Uday Saxena examined India’s role in this evolving landscape. While the country leads in cell therapy, gene therapy development faces hurdles including patent issues and development complexities. The FDA’s recent acceptance of surrogate endpoints in clinical trials could accelerate therapy approvals, potentially expanding treatment to common diseases like type one diabetes within a decade.
“India has the expertise and infrastructure,” noted Dr. Saxena. “What we need now is focused investment and regulatory support to compete globally.”
The message was clear: gene therapy’s promise is within reach, but realizing it requires unprecedented collaboration between academia, industry, and regulators. As Dr. Hajjar concluded, “The science is ready. Now we need the systems to deliver it to patients.”