New platform aims to develop novel therapies

New Delhi: Researchers are working on a new platform that aims to develop several treatments, including gene and mRNA therapies, for three rare diseases which are estimated to affect about one million people in India.

The Myo-Mission anchored by Ashoka University, Sonipat in partnership with 18 institutions and 38 investigators across India, will enhance capabilities for carrying out future clinical trials for Duchene Muscular Dystrophy, GNE Myopathy and Limb-Girdle Muscular Dystrophy (LGMD). All the three diseases either lead to muscular weakness or muscle wasting, and can cause permanent disability.

"In India, estimates suggest that the three diseases combined affect about one million people. The burden of these debilitating, often incurable diseases is made greater by the rarity and lack of diagnosis and treatment options in India," said Alok Bhattacharya, Professor of Biology at Ashoka University.

"Our goal is to develop patient cohorts, create a comprehensive database, and conduct research on disease progression to pave the way for clinical trials," Bhattacharya said. In its Phase 1, the mission plans to have five clinical centres in Mumbai, Bengaluru, Chennai, Delhi and Anand in Gujarat that have been managing patients of these diseases.

The mission researchers plan on different aspects such as developing systems or technology platforms for therapeutic molecules for treatment, as well as developing animal and cellular models for testing purposes. The clinical groups associated with the mission will also monitor the progression of the disease for the Indian patients, the researchers said. Such models for diseases such as GNE myopathy and LGMD have not been done for Indian patients and are required for carrying out clinical studies and drug trials, according to a statement.