Imagine treating diseases by delivering new genetic instructions to the body guiding cells to fight cancer, correct faulty genes, or stop viral infections. This idea forms the foundation of RNA-based medicines, including the technology used in COVID-19 mRNA vaccines. However, nucleic acid therapeutics are highly fragile and can quickly degrade in the body if delivered directly.
Dr G Somu Naidu, Ramanujan Fellow and Faculty at the Centre for Interdisciplinary Research (CIDR), has been awarded a project by ANRF titled “Synthesis of Next-Generation Ionizable Lipids for Delivering Nucleic Acid Therapeutics.”The project, with a total outlay of ₹1.19 crore for five years, aims to develop advanced lipid nanoparticles that can safely protect, transport, and deliver genetic material to target cells, improving the effectiveness of next-generation gene-based therapies.
Brief Discussion of the Project
Nucleic acid therapeutics, including mRNA, siRNA, pDNA, and gene-editing systems, hold immense promise for the treatment of cancer, viral infections, and genetic disorders. The success of COVID-19 mRNA vaccines has demonstrated the clinical potential of these drugs. However, their broader therapeutic application remains limited by the lack of safe, efficient, and tissue-selective delivery systems. Currently, most clinical studies rely on lipid nanoparticle (LNP) technology, yet existing LNP platforms depend on a small number of ionizable lipids. Many of these lipids exhibit suboptimal endosomal escape, limited tissue specificity, and potential safety concerns.
This project aims to design and synthesise next-generation ionizable lipids to enhance delivery efficiency and safety. By strategically engineering novel headgroups, biodegradable linkers, and branched lipid tails, we will establish structure–activity relationships governing transfection efficiency, immune activation, and biodistribution. Lead formulations will be evaluated in disease-relevant models to assess tissue targeting, therapeutic efficacy, and safety profiles. The goal is to develop clinically feasible delivery systems that can accelerate the development of next-generation nucleic acid therapeutics in India and globally.
Practical Implementation and Social Implications of the Research
The practical implementation of this research lies in the development of clinically translatable lipid nanoparticle (LNP) platforms for the safe and efficient delivery of nucleic acid therapeutics. The newly designed ionisable lipids and optimised formulations can be integrated into scalable manufacturing processes compatible with current pharmaceutical infrastructure. These delivery systems can be applied to RNA therapeutics & vaccines, and gene-editing technologies, enabling rapid therapeutic development for cancer, rare genetic disorders, and infectious diseases. The platform-based nature of LNP technology also allows rapid adaptation to emerging health threats, including future pandemics.
From a societal perspective, improved delivery technologies can enhance the safety, affordability, and accessibility of genetic medicines, particularly in low- and middle-income countries. By enabling local innovation and manufacturing capacity, this research can strengthen healthcare self-reliance in India and contribute to global health equity. Ultimately, advancing safe and effective RNA delivery systems has the potential to transform treatment standards, reduce disease burden, and improve quality of life across diverse patient populations.
Funding agency and amount sanctioned: Anusandhan National Research Foundation (ANRF), Budget: 1.19 Cr
Your collaborations: Dr Seok-Beom Yong, Senior Scientist, Korea Research Institute of Bioscience and Biotechnology, South Korea.
Your future research plans.
Design and synthesis of novel delivery systems for safe and efficient delivery of nucleic acid therapeutics and small molecules, targeting the prevention and treatment of cancer, genetic disorders, and infectious diseases.Design and synthesis of potent small-molecule anticancer agents.
