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Benchling Launches Support for the Discovery and Development of RNA Therapeutics

Life sciences cloud leader’s unified platform now supports development of RNA-based treatments for previously ‘undruggable’ diseases

SAN FRANCISCO, Calif., June 22, 2021 – Benchling, the leading life sciences cloud, today announced the launch of a digital solution for scientists to design, analyze, and develop complex RNA therapeutics in a unified R&D platform for the first time.

RNA therapeutics have the potential to transform treatment for diseases that have been notoriously difficult to target with antibodies or small molecules. The ingenuity of this modality means researchers can create more effective treatments for common diseases like lung cancer, chronic kidney disease, and cardiovascular disease, and also potentially address rare and ultra-rare conditions in unprecedented ways. For example, of the 21 late-stage RNA therapeutics, 18 have “orphan status,” which are drugs that receive government assistance as incentive to treat or prevent critical diseases affecting fewer than 200,000 patients in the U.S.

The RNA therapeutics category is one of the most exciting in biotechnology, with ten RNA-targeting drugs approved by the FDA in the past five years and more than 1,300 drugs in the pipeline. With the successful development of the Pfizer-BioNTech and Moderna Covid-19 vaccines, RNA technology has also garnered mainstream global attention. These mRNA vaccines alone are expected to generate $45.2 billion in sales this year. Even prior to the pandemic, RNA therapeutics attracted significant attention — according to an April 2020 report in Nature Reviews Drug Discovery, the market capitalization of public oligonucleotide companies nearly doubled from 2015 to 2020.

“Our customers are using both biology and chemistry to push the envelope of what’s possible and they deserve software that is as innovative as they are,” said Saji Wickramasekara, CEO and co-founder of Benchling. “We developed our RNA therapeutics solution in partnership with researchers focused on RNA in order to deliver on the specific needs of this hybrid modality. Benchling is committed to expanding our support for all therapeutic modalities, including antibodies, cell and gene therapies, and other emerging drug classes.”

RNA therapeutics are driving a massive shift in biopharma from the traditional “one-drug, one-target” approach to multi-dimensional and iterative targeting. However, as these approaches have gained traction scientifically and clinically, the tools to engineer them have been frustratingly limited. There has not been a single software solution to date that handles both the biological and chemical nature of RNA therapeutics and captures the full workflow of designing, analyzing, and testing complex biomolecules. As a result, scientists have struggled to standardize how chemically modified biomolecules are modeled and connect structure to experimental impact on stability, efficacy, and potency.

Biopharmaceutical R&D teams can use Benchling to collaborate across biology and chemistry workflows to support their collective efforts on this hybrid modality. These teams can work faster and improve their overall success rate by unifying traditionally siloed tasks and phases in one cloud-based solution. For the first time, scientists can design chemically modified oligonucleotides with ease, centralize experimental context and results, standardize on syntax, and collaborate with teammates more effectively on a single platform.

Scientists using Benchling can now:

  • Create complex oligonucleotides with ease and contextualized experimental insights:Benchling saves researchers from having to repeatedly create their RNA from the atomic-level up. The visualization capabilities with Benchling’s HELM editor are built for oligo-level editing with the ability to zoom in and out on specific areas for modification. Experimental data is contextualized within Benchling’s solution, making high-quality data actionable throughout the oligo design process.

  • Standardize data and simplify collaboration to accelerate the entire RNA therapeutic lifecycle:Benchling's monomer library helps researchers quickly search and customize the most common base monomers. The embedded HELM notation support standardizes complex biomolecule representation so multidisciplinary teams can all access, understand, and contribute to the molecule's ongoing development. The robust, integrated platform easily registers or creates oligos with uniqueness checks to save time and prevent redundant work.

“Our scientists have been eager to find an informatics tool that could help us design and display our modified oligonucleotides easily,” said Alexandre Debacker, Research Scientist at MiNA Therapeutics.  “Benchling offers an extensive and easy-to-use tool that supports our work with modified RNA across our multidisciplinary teams."