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<p>Cell and gene therapy (CGT) represents a paradigm shift in the treatment of diseases once regarded incurable. This is particularly true for pediatric cancers and inherited disorders which often have a monogenic basis. While the global landscape of CGT is rapidly evolving with numerous approved products, India faces unique challenges in adopting high-cost, complex therapies. This perspective offers a comprehensive overview of the current status of pediatric CGT in India, emphasizing the urgent need for affordable and indigenous alternatives. We examined the spectrum of conditions for which CGT shows potential for pediatric patients, analyzed significant research and clinical studies from India, and investigated the pipeline of new products and therapies approaching clinical trials. This analysis provides a roadmap for the future of this transformative field in India in light of emerging regulatory and ethical regulations.</p>

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Democratizing the Cure: Indigenous Innovations and the Future of Pediatric Cell and Gene Therapy in India

  • 期刊论文

  • 发表日期：

    2026年4月13日

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  • Prasad Iyer,
  • Rajat Bhattacharyya,
  • Gaurav Kharya

摘要

Cell and gene therapy (CGT) represents a paradigm shift in the treatment of diseases once regarded incurable. This is particularly true for pediatric cancers and inherited disorders which often have a monogenic basis. While the global landscape of CGT is rapidly evolving with numerous approved products, India faces unique challenges in adopting high-cost, complex therapies. This perspective offers a comprehensive overview of the current status of pediatric CGT in India, emphasizing the urgent need for affordable and indigenous alternatives. We examined the spectrum of conditions for which CGT shows potential for pediatric patients, analyzed significant research and clinical studies from India, and investigated the pipeline of new products and therapies approaching clinical trials. This analysis provides a roadmap for the future of this transformative field in India in light of emerging regulatory and ethical regulations.