The Indian Institute of Technology Jodhpur has established a Duchenne Muscular Dystrophy (DMD) Research Centre in collaboration with Dystrophy Annihilation Research Trust (DART) Bangalore and the All India Institute of Medical Sciences (AIIMS) Jodhpur. Many challenges are faced concerning DMD, which is a rare and incurable genetic disease. DMD is the most common and fatal type of muscular dystrophy, marked by gradual muscle deterioration. The condition is predominantly seen in boys. In India, over five lakh boys are suffering from DMD. The centre at IIT Jodhpur aims to develop affordable therapeutics for DMD and enhance the efficacy of Antisense Oligonucleotide (AON)-based therapeutics.
The AON-based therapeutics’ idea 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 personalised medicine. The current therapeutic options available to treat DMD are minimal and highly expensive (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 main focus areas of the DMD Research Centre at IIT Jodhpur include:
• To design an affordable and effective therapeutic for DMD patients by adopting a bioengineered concept under the “Make in India” (AtmaNirbhar) initiative.
• To conduct 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. It has also initiated the expansion of this 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.
