Cell and gene therapy is widely used to treat, prevent, or potentially cure diseases, and helps to alleviate the underlying cause of both genetic and acquired diseases. Both cell therapy and gene therapy are overlapping fields of biomedical research and treatment. It can be used in children, adults, and even animals. Cell and gene therapy plays an important role in modern health care, as it helps to provide techniques and concepts that can be used in gene regulation, regenerative capacity, cell–cell interactions, and remodeling.
It is an innovative medical procedure that offers personalized treatments for various illnesses.Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body. Cell and gene therapies (CGTs) are at the forefront of innovation to treat severe diseases and rare diseases. Several therapies are now available in the market, with the Food and Drug Administration (FDA) accelerating approvals and some treatments already on the market.
Europe is one of the largest Cell and Gene Therapy Research Challenges Marketacross the world, with France and the United Kingdom at pole positions.
Regulatory approvals for cell and gene therapies are a significant challenge. Whereas preparing for these approvals has traditionally been a cumbersome process, new regulatory pathways are now available. New designations such as Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy (BT) allow for accelerated timelines and rolling review processes. Regulatory agencies have recognized this need and are creating accelerated pathways for cell and gene therapy products.
Scalability and manufacturability are the two, closely-related challenges, especially if cell and gene therapies are to fulfill their clinical potential. Cell and gene therapy companies will have limited amounts of clinical data at the time of approval of these therapies. This in turn makes it difficult to estimate their positive effects over an extended period of time. It is also difficult to estimate the true value of these treatments in life-changing chronic indications. Hundreds of research studies are under way to test cell/gene therapy as a treatment for genetic conditions.