Precision Medicine
Govt Reiterates Gene Therapy Framework as India Reports Early Clinical Progress
India reports early clinical progress in gene therapy as government reinforces regulatory framework for advanced treatments
India is advancing its efforts in gene therapy and gene editing, with early clinical progress and an existing regulatory framework supporting the evolution of treatment for genetic disorders, according to a recent update presented in Parliament.
In a written reply, the government reiterated that a structured policy ecosystem is already in place to guide the development of advanced therapies, including CRISPR-based gene editing, gene augmentation and cell-based treatments. These technologies aim to address diseases at their root cause, indicating the potential to move beyond long-term disease management.
A key highlight of the update is progress in gene therapy for Hemophilia A. Early-phase clinical research conducted in India has demonstrated sustained production of Factor VIII, indicating the potential to reduce bleeding episodes and dependence on lifelong treatment. While still in initial stages, such outcomes point to the growing clinical feasibility of gene-based interventions in the country.
The government emphasised that these advancements are being pursued under an established regulatory framework. The National Guidelines for Gene Therapy (2019), issued by the Indian Council of Medical Research in collaboration with the Department of Biotechnology, along with oversight from the Central Drugs Standard Control Organisation, are designed to ensure patient safety, ethical compliance and scientific rigour in clinical development.
The update also reflects a broader focus on advanced and emerging therapies, including cell and gene therapies and advanced biologics. These efforts are supported by policy initiatives aimed at strengthening domestic research capabilities.
Experts note that despite encouraging progress, challenges related to affordability, scalability and specialised infrastructure remain. However, the alignment of policy support with ongoing scientific advancement indicates that India is positioning itself to transition toward more targeted and innovation-driven healthcare solutions.
As regulatory pathways evolve alongside clinical research, gene therapy and gene editing are expected to play an increasingly important role in shaping future treatment approaches for a range of diseases.
In a written reply, the government reiterated that a structured policy ecosystem is already in place to guide the development of advanced therapies, including CRISPR-based gene editing, gene augmentation and cell-based treatments. These technologies aim to address diseases at their root cause, indicating the potential to move beyond long-term disease management.
A key highlight of the update is progress in gene therapy for Hemophilia A. Early-phase clinical research conducted in India has demonstrated sustained production of Factor VIII, indicating the potential to reduce bleeding episodes and dependence on lifelong treatment. While still in initial stages, such outcomes point to the growing clinical feasibility of gene-based interventions in the country.
The government emphasised that these advancements are being pursued under an established regulatory framework. The National Guidelines for Gene Therapy (2019), issued by the Indian Council of Medical Research in collaboration with the Department of Biotechnology, along with oversight from the Central Drugs Standard Control Organisation, are designed to ensure patient safety, ethical compliance and scientific rigour in clinical development.
The update also reflects a broader focus on advanced and emerging therapies, including cell and gene therapies and advanced biologics. These efforts are supported by policy initiatives aimed at strengthening domestic research capabilities.
Experts note that despite encouraging progress, challenges related to affordability, scalability and specialised infrastructure remain. However, the alignment of policy support with ongoing scientific advancement indicates that India is positioning itself to transition toward more targeted and innovation-driven healthcare solutions.
As regulatory pathways evolve alongside clinical research, gene therapy and gene editing are expected to play an increasingly important role in shaping future treatment approaches for a range of diseases.
Advanced Therapies IndiaBiotechnology IndiaCRISPR Gene Editing
