Intent to Cure: The Need for a Rare Disease Platform in India and Across the Global South

The democratization of genomic technologies presents substantial opportunities for addressing rare genetic diseases, particularly in collaborations between the Global South and North. In this Perspective, we describe the current progress in gene therapy, including CRISPR, in India and see an upward trajectory of innovation. We propose the establishment of a rare disease platform in India and across the Global South designed to bridge scientific, clinical, and economic gaps, transforming untapped genetic diversity into shared opportunities for therapeutic innovation and health care equity. This platform would encompass a comprehensive data infrastructure capturing clinical, genomic, and biosample data, complemented by an artificial intelligence-powered analytics layer to enhance patient engagement and clinical trial matching, ultimately enabling cost-effective research and development (R&D) of novel therapies.