Sponsor: Dystrophy Annihilation Research Trust (DART India), Bengaluru — India’s first dedicated DMD research lab, founded in 2012.
Overall Lead: Dr. Surinder Kher
Scientific Contact: Dr. Arun Shastry
CTRI ID: CTRI/2021/04/032498

What it is: An indigenously developed 2′-O-Methyl Antisense Oligonucleotide (ASO) designed for exon skipping — targeting Exon 45, 51, or 53 depending on the patient’s specific deletion mutation. The ASO binds splice donor/acceptor sites on the pre-mRNA, causing the spliceosome to skip the mutated exon. This restores the reading frame, allowing production of a shortened but partially functional dystrophin protein (converting a severe frameshift phenotype into a milder in-frame one).

Trial design: 48-week randomized, double-blind, placebo-controlled trial, followed by a 24-week open-label extension.
Eligibility: Male, ambulant, ages 5–25, with a deletion amenable to Exon 45/51/53 skipping (genetic confirmation required).
Primary endpoints: 6-Minute Walk Test (6MWT) distance and North Star Ambulatory Assessment (NSAA) score.
Sites: Reported to span 9 hospitals across 8 cities including Bengaluru, Delhi, Hyderabad, Pune, Jaipur, and Bhopal.

Update: DART India signed a Memorandum of Understanding with GenAssist Ltd to accelerate clinical development of this DMD therapy — a sign the program is moving toward expanded/accelerated trials. Members should watch for updated CTRI status.

How to inquire: DART India, Bengaluru — Ph: +91-80-2341-2725 / +91-98801-12725.
Also offers counseling, medical advisory, and rehab support independent of trial enrollment.

References:

CTRI record, https://ctri.nic.in/Clinicaltrials/showallp.php?mid1=53212&EncHid=&userName=Duchenne+Muscular+Dystrophy
DART India profile, https://www.karnataka.com/ngos/dart-dystrophy-annihilation-research-trust/
GenAssist–DART MoU announcement, https://www.genassisttx.com/newsroom/P02601070