Biosimilar gene therapy holds promise for the treatment of a range of rare diseases, including Progeria, Spinal muscular atrophy (SMA), and Duchenne muscular dystrophy (DMD), as well as cancers and other chronic diseases. 

This was the message delivered by Prof. Surajit Sinha of the Indian Association for the Cultivation of Science (IACS) at a lecture titled "RNA Targeted Therapy: A Biosimilar Approach" organized by the GITAM School of Pharmacy at GITAM Deemed to be University.

In his lecture, Prof. Sinha explained that genetic disorders are caused by defects in genes, and cancer is one of the most well-known examples.

He pointed out that several diseases caused by DNA defects are notorious for the lack of available treatments or drugs due to insufficient scientific research data.

Prof. Sinha highlighted the possibilities of developing DNA-based materials for the treatment of gene-related diseases using a biosimilar approach. He shared insights into his research work on manufacturing these drugs in India.

Additionally, he discussed the potential of MO-based therapy for cancer treatment by inhibiting the "NANOG" gene, which plays a crucial role in the hallmarks of cancer. This approach represents a promising avenue for cancer therapy development.

Prof. Sinha emphasized the importance of chemical modification in improving Watson-Crick base pairing affinity, metabolic stability, and cellular uptake of RNA, which are essential factors in the discovery and development of RNA-based therapeutics.

The lecture was presided over by Prof. Jagattaran Das, Dean of the GITAM School of Pharmacy, and coordinated by Prof. S. Raja, Principal of GITAM Deemed to be University. Faculty members, students, and research scholars from the School of Pharmacy attended the informative event.

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