Preclinical Pipeline

[Genetic Counselor]

Preclinical Pipeline — Indigenous Gene Therapy Programs for DMD in India

[Genetic Counselor]

Not yet in human trials, but worth tracking since these could move to CTRI-registered studies:

Intas Pharmaceuticals (Centre for Cell & Gene Therapy) – preclinical AAV and lentiviral vector development targeting DMD, alongside Hemophilia A/B, SMA, β-thalassemia, and Sickle Cell Disease.
Reliance Life Sciences – preclinical autologous/allogeneic cell therapy and viral vector platforms (primarily ocular and hematopoietic disorders currently, but part of the same domestic gene-therapy manufacturing base that could extend to DMD).
Micro CRISPR Private Limited – gene-editing research platforms for rare monogenic conditions and diagnostics.

Domestic CAR-T success (NexCAR19, developed by ImmunoACT/IIT Bombay, CDSCO-approved) is frequently cited as proof that India’s regulatory and manufacturing ecosystem can support advanced cell/gene therapies, this is part of why analysts expect DMD-directed gene therapy trials to follow.

References:

Recent developments in gene therapy research in India, https://www.ias.ac.in/article/fulltext/jbsc/049/0037

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