Cell engineering redefines treatment protocols of genetic diseases: Dr Siddhartha Mukherjee

Nandita Vijay, BengaluruSaturday, February 4, 2023, 08:00 Hrs  [IST]

Cell engineering enables permanent manipulation of cells to achieve a desired outcome as it has a long-term impact, and in most cases it has the curative potential. Genetic diseases that had been out of reach of traditional medicine can now be tackled with relative ease, said Dr Siddhartha Mukherjee, co-founder, Immuneel Therapeutics.
There is a great desire and need for cell therapies in India. Funding agencies, both public and private, along with the scientific community in India have already taken note of the potential of cell engineering for cell therapies and are investing in this area with CAR-T (Chimeric Antigen Receptor T-Cell) therapy already in clinical trials. However, we need more investments to develop indigenous technologies to reduce cost to make them affordable for patients in the country, he added.
Considerable research and proven cell therapies are available for cancer. There are also several advances in the treatment of infectious diseases and genetic disorders with cell therapy. In fact, cell therapies with engineered cells are in various phases of pre-clinical and clinical development for infectious and genetic diseases. CAR-T therapy for viral infections such HIV, HBV, HCV, and EBV are being explored. Genomic editing to make T-cells resistant to infection from HIV virus are in various phases of research. For genetic disorders, gene-edited stem cells as a curative option for Sickle cell disease (SCD) and transfusion-dependent ß-thalassemia are in clinical trials, Dr Mukherjee told Pharmabiz.
While cell engineering is a new science, whereas cell biology is one of the oldest sciences. Hence, by training cell biologists for molecular techniques it would be possible to educate individuals to quickly increase the pool of human resources, he said.
Yes there are efforts to introduce cell engineering as subject specialization into the gamut of biological sciences. Given the potential of cell therapies, setting a theoretical background early on will not help in training but it will also help in adopting cell therapies as a treatment option by the population when a need arise, noted Dr Mukherjee.
On the likely future outcomes in the next three years of cell research and related therapies, Dr Mukherjee said, “We will be expanding cell therapies to treat many different conditions that are not necessarily life threatening. The 1,000+ clinical trials which are going on worldwide using cell and gene therapies will collect enough data to improve manufacturing challenges, reduce the cost, and streamline the regulatory approval processes.”