IIT Jodhpur to develop affordable therapeutics for children with Duchenne Muscular Dystrophy

The Indian Institute of Technology Jodhpur has established a Duchenne Muscular Dystrophy (DMD) Research Centre to aid in the development of affordable therapeutics and enhance the efficacy of Antisense Oligonucleotide (AON)-based therapeutics.

Duchenne Muscular Dystrophy or DMD is a rare and incurable genetic disease that has many challenges. It is the most common and fatal type of muscular dystrophy, marked by gradual muscle deterioration. Its onset is in early childhood, usually between ages 2 and 3, and predominantly seen in boys, with over five lakh boys in India suffering from it. But in rare cases, it can also affect girls.

A genetic disorder characterised by progressive muscle degeneration and weakness is caused due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies. The other three diseases that belong to this group are Becker Muscular dystrophy – a mild form of DMD, an intermediate clinical presentation, and DMD-associated dilated cardiomyopathy (heart disease) with little or no clinical skeletal, or voluntary, muscle disease.

The Centre at IIT Jodhpur has been established in collaboration with Dystrophy Annihilation Research Trust (DART) Bangalore and the All-India Institute of Medical Sciences (AIIMS) Jodhpur.

The idea behind AON-based therapeutics is to hide or mask specific exons (a segment of a DNA or RNA molecule containing information coding for a protein) in a gene sequence. In DMD patients, one or more exons can be masked with specific molecules called AON or molecular patches. Due to these challenges, DMD patients need personalized medicine. The current therapeutic options available to treat DMD are minimal and highly expensive costing over Rs. 2-3 crores per child a year. Drugs are mostly imported from abroad, accelerating dosing costs and putting them out of reach for most families.

The DMD Research Centre at IIT Jodhpur will have two main focus areas. These include designing an affordable and effective therapeutic for DMD patients by adopting a bioengineered concept under the ‘Make in India’ (AtmaNirbhar) initiative and conducting social outreach programmes to generate awareness about this condition and extend support for children with DMD and their parents.

Towards this mission, the DMD Research Centre at IIT Jodhpur has hosted two, day-long outreach programmes besides expanding the activity to every district in Rajasthan.

The current team involved in this project are Prof Surajit Ghosh - Dean, Research and Development, Professor of Bioscience & Bioengineering at IIT Jodhpur, Dr Sudipta Bhattacharyya - Assistant Professor, Bioscience & Bioengineering at IIT Jodhpur, Dr Dibyendu Sasmal - Assistant Professor, Department of Chemistry at IIT Jodhpur, Dr Nirmal Rana - Assistant Professor, Department of Chemistry at IIT Jodhpur, Dr Arun Shastry - Chief Scientific Officer at DART Bangalore, Professor Kuldeep Singh - Dean Academics at AIIMS Jodhpur and Dr Varuna Vyas - Clinician and Expert Paediatrician, AIIMS Jodhpur.

Talking about the DMD Research Centre at IIT Jodhpur, Prof. Surajit Ghosh, Dean Research and Development, Professor of Bioscience & Bioengineering, IIT Jodhpur, said, “The primary goal of our team is to develop two therapeutic leads for clinical trials on high priority."

The DMD Research Centre at IIT Jodhpur is working in collaboration with DART Bangalore, a parent-founded NGO conducting research and awareness programmes in DMD as well as AIIMS Jodhpur. The institute is primarily involved in designing affordable therapeutics through bioengineering strategy, synthesis of those designed therapeutics, and their in-vitro efficacy. DART has been developing AON-based reasonable therapeutics in collaboration with IIT Jodhpur and screening an organised library of DMD therapeutic leads. AIIMS Jodhpur is the primary and significant clinical collaborator in this project and is associated with this centre as a clinical group.