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How stem cell and gene therapies are revolutionising healthcare

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Tanaz Buhariwalla, Asia Director, IDA Ireland explains how stem cell and gene therapies are changing the healthcare game

In the realm of medical breakthroughs, there is a lot of buzz around stem cell therapy and gene therapy. These innovations offer hope for conditions once thought untreatable. Think of genetic disorders or inherited diseases – they have long been seen as incurable, but now, there is a glimmer of hope. The 21st century has seen incredible strides in medicine, with cell and gene therapy (CGT) emerging as groundbreaking innovation in healthcare. This advanced treatment involves introducing healthy genes into the body to restore tissue function and tackle diseases in a more effective way. Unlike traditional treatments that simply relieve symptoms, Stem Cell and Gene therapies can tackle the root causes of diseases. According to research from the World Health Organization (WHO), CGT shows promise, with diverse genes and vectors used in trials, leading to successful outcomes.

You might have heard of success stories individually: stem cell therapy in bone marrow transplants and gene therapy in CAR-T therapy for lymphoma (blood cancers), which are examples of CGT. Projections indicate that the CGT market will skyrocket from £6.1 billion in 2024 to a monumental £14.1 billion by 2027, highlighting a remarkable compound annual growth rate of 18.6 per cent.

Further, regulatory approvals, successful clinical trials, and substantial funding have solidified the status of CGT. Beyond oncology, CGT demonstrates promise in addressing degenerative disorders, inflammatory conditions, and even burn injuries. In fact, more research has been focussed in this area of medtech.

This proliferating field has attracted the attention of start-ups and large companies alike, signalling robust growth ahead. Amongst, other countries, Ireland with its strong manufacturing history and research base in pharmaceuticals and biotechnology, plays a crucial role in supporting biotech companies from India as they venture into this exciting field. Ireland, presents a promising scope for stem cell and gene therapy, with advancements in gene transfer methods and gene editing technologies. Early-phase trials are moving rapidly toward advanced clinical phases. Likewise, India, is at pace too for CGT, for example, researchers at IIT-Bombay and oncologists at Tata Memorial Centre recently developed India’s first CAR T-Cell therapy, a significant milestone. Ireland and India have the chance to lead in personalized medicines and CGT treatments globally.

Ireland has emerged as a global hub for advanced therapies, driven by biotech investments and government-funded research institutes. Simultaneously, India’s CMO/CRO sector and dynamic startup ecosystem, supported by favorable regulations, position it as a significant contender in the global market. Ireland’s clinical trial network is expanding, with nearly 291 sites, attracting key players.

In leading nations, partnerships between industry and researchers are driving gene therapy advancements for hereditary diseases. For instance, in Ireland, Allergan and the National Institute for Bioprocessing Research and Training (NIBRT) are collaborating on Adeno-Associated Virus (AAV) gene therapies. AAV-based treatments deliver functional genes to patients, showing promise for conditions like Haemophilia and macular degeneration, with increasing approvals. Ireland’s ecosystem strengthens as NIBRT expands its Dublin facility, enhancing research and training capabilities for advanced therapeutics, including cell and gene therapies, mRNA and DNA-based treatments, and novel vaccines.

In Asian nations, notably China, Japan, and Korea, have been actively advancing gene medicines. In India, the Indian Council of Medical Research (ICMR) focusses on genetic diseases affecting the brain, muscles, eyes, heart, and blood, including Sickle Cell Disease and Haemophilia. Additionally, it prioritizes cancer, diabetes, and lung diseases. To bridge gaps, ICMR seeks proposals to fund gene therapy research.

Stem cell and gene therapy represents a new frontier in medicine, offering transformative treatments for a range of diseases and conditions Both Ireland and India have made strides in creating supportive ecosystems that encourage collaboration, investment, and knowledge sharing. Together, these two countries can achieve synergy in navigating the complex manufacturing landscape. With global demand for manufacturing capacity expected to outstrip supply, there are ample opportunities for both nations to invest in additional capacity to meet the growing needs of this sector.

With their unique strengths and strategic approaches, Ireland and India are well positioned to make significant contributions to global health and well-being. The future of medicine is here, and it is being shaped by the pioneering work in stem cell and gene therapy in these two countries.

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