AAPS PharmSci 360: Advancements and Challenges in Large RNA Therapeutics

Pharmaceutical Technology® sat with Bala Addepalli, PhD, director, R&D New Modalities Portfolio, Waters Corporation, at AAPS PharmSci 360, which is taking place Oct. 20–23, 2024 in Salt Lake City, UT, to discuss the challenges seen in large RNA therapeutics as well as their challenges.

Addepalli pointed out that there is much interest by industry in developing RNA-based drugs for treating genetic disorders, especially after the success of messenger RNA (mRNA), which was used to develop COVID-19 vaccines. He also noted that pharmaceutical vendors and companies are developing mRNA vaccines for other viral diseases such as influenza or as a combination of COVID and influenza vaccines. Furthermore, mRNA vaccines are also part of protein replacement therapies for patients who have deficiency in enzymes or proteins.

Meanwhile, challenges in the large RNA therapeutic space include inadequate analytical tools, Addepalli said. “The analytical tools that are currently available are inadequate, in a sense; they would provide only incomplete or partial information, so the purity and identity of the molecule is not fully revealed. And it also necessitates development of complex procedures in order to get such information,” he stated. And, he further explained, when specialized procedures and complex techniques are employed, the cost of development and manufacturing goes up, “and that leads to accessibility issues,” Addepalli added.

Click above for the full interview.

Attendees can visit Waters Corp. at Booth #2438 at AAPS PharmSci 360.

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About the speaker

Bala Addepalli, PhD, Director, R&D New Modalities Portfolio, Waters Corporation

Bala Addepalli, Bala_Addepalli@waters.com, is currently working as director of the Evaluations and Application Science Team under New Modalities (Cell and Gene Therapy product analysis) portfolio of Waters Corporation. His interests include development of simpler but efficient approaches for cell and gene therapy products such as messenger RNA (mRNA), lipid nanoparticle (LNP), and adeno-associated virus (AAV) analysis through liquid chromatography coupled with light scattering and mass spectrometry-based detection techniques.Prior to joining Waters, Addepalli developed novel enzymes and analytical methods for characterization of cellular RNA and modified oligonucleotides through mass spectrometry at University of Cincinnati. Before that he worked on RNA processing events including mRNA polyadenylation at University of Kentucky, and viral RNA characterization through mRNA translation for his graduate studies at Indian Agricultural Research Institute, New Delhi, India. Bala has authoredover 60 publications in peer-reviewed scientific journals and has delivered oral presentations at scientific meetings.