Rare diseases can be treated with tailored gene therapy but India’s regulatory system must evolve

Earlier this month, The New York Times carried a widely forwarded essay about Annette Harlow, a woman in her eighties who, in early 2020, had been told that her lung cancer had stopped responding to treatment. Soon after that, she was enrolled on a Phase 1 trial of a new drug and six years later, she is still alive. As heartening as the story is, the larger point it makes is that it is not science which holds us back today, but the failings of our regulatory system.