It is pertinent to develop therapeutics to address unmet needs of Rare Genetic Diseases, in the Indian context. Point of care production of therapeutics to reduce the cost and/or innovation in R&D, for a quick transition from lab to clinically treat RGD patients is the need of the hour. Towards this, multiple approaches are being developed:

  • Protein based therapeutics for enzyme deficiency based disorders (Lysosomal Storage Disorders)
  • Stem cell therapies focused on inborn errors of metabolism and haemoglobinopathies
  • RNA based therapeutics optimized for stability with effective delivery systems for deficiencies
  • Small molecule based biotherapeutics for motor neuron disease

Vertical Lead: Anirudha Lakshminarasimhan

Investigators: Vasanth Thamodaran, Mansi Malik