ARNAY Sciences: Transforming Healthcare with RNA-Based Therapeutics

Sudhir Agrawal, D.Phil, President and Founder

Sudhir Agrawal, D.Phil, President and Founder of ARNAY Sciences, brings over 30 years of expertise in advancing RNA-based therapeutics. His pioneering research has focused on developing innovative drugs that disrupt the flow of genetic information from disease-associated mRNA. This groundbreaking approach aims to prevent the production of harmful proteins linked to conditions such as high cholesterol, hypertension, and neurological diseases, positioning ARNAY Sciences at the forefront of therapeutic innovation.

Disrupting mRNA with Innovative Genetic Band-Aids

While conventional medicines focus on targeting proteins to manage the symptoms of genetic diseases, Agrawal advocates for a more direct approach— targeting genetic information at the mRNA level. This method accelerates drug development by identifying unique mRNA sequences in individuals and rapidly synthesizing their mirror images in the laboratory—often within days. By significantly reducing the time and costs associated with traditional drug development, this strategy broadens the scope for creating highly targeted therapeutic solutions for various genetic disorders.

At ARNAY Sciences, Agrawal applies this innovative approach to transform genetic sequences into injectable drugs that are safe for human use. These informational drugs, known as antisense therapies, chemically modify genetic sequences to reach specific targets and block disease-associated mRNA. Agrawal likens these therapies to “genetic Band-Aids” made entirely of DNA, which are designed to enter the body, locate specific genes, and bind to them, effectively halting the production of harmful proteins.
“Our genetic Band-Aid design assists researchers, entrepreneurs, and organizations in the biotech and pharmaceutical sectors to create hundreds of drugs, with only the genetic sequences varying,” says Agrawal.

Agrawal began pioneering the “genetic Band-Aid” design while working in the laboratory of Paul Zamecnik, a pioneering scientist of this approach, at Worcester Foundation in Shrewsbury, MA. Building on this foundational research, he co-founded Idera Pharmaceuticals with Zamecnik to advance antisense therapies capable of surviving in the human body, targeting specific mRNA, and shutting down its activity. During his time at Idera, Agrawal’s innovations in genetic Band-Aid design became the industry standard, contributing to approved treatments for conditions such as Duchenne muscular dystrophy (DMD), neurological disorders, and elevated lipid levels.

Advanced Antisense Solutions for Genetic Disorders

In the late 1990s, Agrawal co-authored a landmark paper with leading experts, demonstrating how antisense technology could address the genetic defects associated with DMD. This debilitating genetic disorder leads to severe health complications and chronic disability in children, underscoring the urgent need for innovative therapeutic solutions. The use of antisense technology has since led to the approval of several treatments for DMD, but high costs have limited access in regions like India.

To tackle this challenge, Agrawal developed a platform at ARNAY Sciences to make therapies more globally accessible. The platform supports drug developers by correcting genetic defects and facilitating rapid drug creation, significantly reducing development timelines.

In 2017, after retiring from Idera Pharmaceuticals, Agrawal saw an opportunity to enhance the genetic Band-Aid technology while working on his book, Advances in Nucleic Acid Therapeutics. This realization inspired him to establish ARNAY Sciences and pursue advancements in genetic Band-Aid designs.
His latest innovation involves a novel genetic Band-Aid with a transient circular shape, engineered to activate only upon binding to specific disease-associated mRNA. This design significantly improves the specificity and potency of antisense therapies. ARNAY Sciences has filed multiple patent applications to protect these breakthroughs.

Agrawal established a licensing and collaboration agreement between ARNAY Sciences and Alloy Therapeutics to expand access to this cutting-edge technology. The partnership aims to accelerate the development of antisense drugs for the biotech and pharmaceutical industries, significantly reducing delivery time. By focusing on commercial viability and improved patient outcomes, the collaboration positions ARNAY Sciences at the forefront of RNA therapeutics.

Our genetic Band-Aid design assists researchers, entrepreneurs, and organizations in the biotech and pharmaceutical sectors to create hundreds of drugs, with only the genetic sequences varying

Agrawal’s commitment to innovation has contributed to a surge in clinical studies and the approval of several customized medicines for rare genetic disorders. He has published over three hundred research papers, edited four books, and is a coinventor of over 400 patents worldwide.

Under his leadership, ARNAY Sciences is advancing the chemical engineering of DNA and RNA and redefining medical health by developing effective, high-quality antisense drugs. As the company continues to push the boundaries of biotechnology, it enhances the potential for transformative treatments, addressing previously untreatable genetic conditions and improving quality of life.