Human beings have always strived for improvement, with the quest for betterment deeply woven into recorded history. A significant part of this journey has involved attempts to enhance the human body. This was alluded to in the 1930s by Aldous Huxley in the dystopian novel Brave New World, where human beings are engineered to fulfill certain roles in an attempt to create a stable and happy society.Cut to the present day, where early forms of cell and gene therapies are succeeding in this quest in a small way. Cell therapies are medical treatments which involve introducing healthy cells into a patient’s body to treat or prevent disease. These cells are often genetically modified to make them more effective. Remarkably, the first such therapy was approved for marketing as recently as 2017 in the United States for certain types of leukemia.

It has been encouraging to see India’s first approved cell therapy become available to patients in 2023. A recent clinical study by scientists at the Center for Stem Cell Research, Tamil Nadu using gene therapy with lentiviral-transduced autologous cells for haemophilia-A showed promising results by restoring factor VIII production in the patients. Similarly, an indigenous therapy for sickle-cell anaemia with CRISPR-Cas is progressing in India. And that is just the tip of the iceberg. There is a lot more research in the pipeline for us to witness in coming years. These include:

●Studies focused on different disease areas, ranging from blood cancer to the more difficult solid cancers, and from anaemia to vision correction, auto-immune disease, and Parkinson’s disease

●Research on different cell types like T-cells, natural killer (NK) cells, macrophages, and retinal pigment cells

In 2023, the global gene therapy market size was estimated at $5.54 billion. It is expected to grow with a compound annual growth rate of 18.88% from 2024 to 2030. Various factors attribute to the market growth including the areas of advanced therapies, gene delivery technologies, progressive competition, as well as key players focusing on commercialisation of their therapies.It is also gratifying to see that many Indian efforts are balanced by a focus on making these therapies affordable and accessible to the majority of the population. Progress in this area has been propelled by advancements in gene-editing technologies, which have become increasingly democratised. The foundational work in this area was awarded a Nobel Prize in 2020, and there have been multiple iterations of this technology ever since.India is emerging as a leader in this space, conducting path-breaking work on creating a new gene editor with better functionality. Such efforts will help develop more compelling and economically meaningful therapies for the Indian population by providing opportunities for the use of locally manufactured products for gene editing.

Furthermore, it is heartening to see Indian academic institutions partner with the corporate world on these initiatives. The first marketed therapy was incubated in IIT Mumbai in collaboration with Tata Memorial Centre and has set the stage for other such efforts across academia and the corporate world. This trend has been responsible for the growth of the biotech industry across many countries and India needs to develop this combination to invest in it for the future.

The Indian pharma industry has already built bio-manufacturing infrastructure primarily focused on chemical entities, and this is now starting to be adapted for biological entities. Companies in this space are focused on making this infrastructure globally competitive so it can serve Indian and global biotech players. This represents a new opportunity for substantial investment by the government and corporate India with many more academia-industry partnerships on the horizon.

In 2019, the Indian Council of Medical Research collaborated with the Department of Biotechnology to publish the National Guidelines for GTP Development and Clinical Trials, paving the path for cell and gene therapy in India. Today, scientists in India are working on proof-of-concept studies on reversing macular degeneration and restoring eyesight, cancer vaccines which carry treatment codes through cells, treatments of sickle-cell anaemia using in-vivo technologies, using NK cells and macrophages for immune disorders, and much more. The future holds immense promise with our research community focused on creating comprehensive, targeted therapies that are both effective and affordable.

Lalit Pai, CEO, and Renjitha Gopurappilly, Chief Scientist, NKure Therapeutics Pvt Ltd, Bangalore.

Human beings have always strived for improvement, with the quest for betterment deeply woven into recorded history. A significant part of this journey has involved attempts to enhance the human body. This was alluded to in the 1930s by Aldous Huxley in the dystopian novel Brave New World, where human beings are engineered to fulfill certain roles in an attempt to create a stable and happy society.Cut to the present day, where early forms of cell and gene therapies are succeeding in this quest in a small way. Cell therapies are medical treatments which involve introducing healthy cells into a patient’s body to treat or prevent disease. These cells are often genetically modified to make them more effective. Remarkably, the first such therapy was approved for marketing as recently as 2017 in the United States for certain types of leukemia.

It has been encouraging to see India’s first approved cell therapy become available to patients in 2023. A recent clinical study by scientists at the Center for Stem Cell Research, Tamil Nadu using gene therapy with lentiviral-transduced autologous cells for haemophilia-A showed promising results by restoring factor VIII production in the patients. Similarly, an indigenous therapy for sickle-cell anaemia with CRISPR-Cas is progressing in India. And that is just the tip of the iceberg. There is a lot more research in the pipeline for us to witness in coming years. These include:

●Studies focused on different disease areas, ranging from blood cancer to the more difficult solid cancers, and from anaemia to vision correction, auto-immune disease, and Parkinson’s disease

●Research on different cell types like T-cells, natural killer (NK) cells, macrophages, and retinal pigment cells

In 2023, the global gene therapy market size was estimated at $5.54 billion. It is expected to grow with a compound annual growth rate of 18.88% from 2024 to 2030. Various factors attribute to the market growth including the areas of advanced therapies, gene delivery technologies, progressive competition, as well as key players focusing on commercialisation of their therapies.It is also gratifying to see that many Indian efforts are balanced by a focus on making these therapies affordable and accessible to the majority of the population. Progress in this area has been propelled by advancements in gene-editing technologies, which have become increasingly democratised. The foundational work in this area was awarded a Nobel Prize in 2020, and there have been multiple iterations of this technology ever since.India is emerging as a leader in this space, conducting path-breaking work on creating a new gene editor with better functionality. Such efforts will help develop more compelling and economically meaningful therapies for the Indian population by providing opportunities for the use of locally manufactured products for gene editing.

Furthermore, it is heartening to see Indian academic institutions partner with the corporate world on these initiatives. The first marketed therapy was incubated in IIT Mumbai in collaboration with Tata Memorial Centre and has set the stage for other such efforts across academia and the corporate world. This trend has been responsible for the growth of the biotech industry across many countries and India needs to develop this combination to invest in it for the future.

The Indian pharma industry has already built bio-manufacturing infrastructure primarily focused on chemical entities, and this is now starting to be adapted for biological entities. Companies in this space are focused on making this infrastructure globally competitive so it can serve Indian and global biotech players. This represents a new opportunity for substantial investment by the government and corporate India with many more academia-industry partnerships on the horizon.

In 2019, the Indian Council of Medical Research collaborated with the Department of Biotechnology to publish the National Guidelines for GTP Development and Clinical Trials, paving the path for cell and gene therapy in India. Today, scientists in India are working on proof-of-concept studies on reversing macular degeneration and restoring eyesight, cancer vaccines which carry treatment codes through cells, treatments of sickle-cell anaemia using in-vivo technologies, using NK cells and macrophages for immune disorders, and much more. The future holds immense promise with our research community focused on creating comprehensive, targeted therapies that are both effective and affordable.

Lalit Pai, CEO, and Renjitha Gopurappilly, Chief Scientist, NKure Therapeutics Pvt Ltd, Bangalore.