Rgenta Therapeutics and GSK Forge Strategic Alliance to Advance RNA-Targeted Small Molecule Medicines

Woburn, Mass. – December 4, 2024 – Rgenta Therapeutics, a clinical-stage biotechnology company specializing in RNA-targeted small molecule therapies, has announced a multi-year strategic research alliance with pharmaceutical giant GSK. The partnership will focus on discovering and developing oral RNA-targeting splice modulators for oncology and other disease areas, leveraging Rgenta’s proprietary platform.

Under the agreement, Rgenta will receive up to $46 million in upfront and pre-option milestone payments. The company stands to earn up to nearly $500 million per target in option exercises and milestone payments, along with tiered royalties and future equity investments. GSK retains the option to expand the collaboration to additional targets. Rgenta will lead discovery efforts, while GSK will manage further development and commercialization of any resulting drug candidates.

A Game-Changing Approach to RNA-Targeting Therapies

Dr. Simon Xi, co-founder and CEO of Rgenta, emphasized the alliance’s significance, stating, “This partnership with GSK validates the potential of our small molecule RNA-targeting drug discovery platform. Together, we can accelerate the development of a new class of medicines for patients with difficult-to-treat diseases.”
Christopher Austin, SVP of Research Technologies at GSK, highlighted Rgenta’s innovative approach, saying, “We are excited about Rgenta’s differentiated methodology for developing splice modulators for high-value targets. Partnering with Rgenta complements GSK’s existing expertise in RNA-targeting medicines.”

Targeting Undruggable Diseases

Rgenta’s lead candidate, RGT-61159, is an orally available small molecule currently in Phase 1a/b trials. It targets the MYB transcription factor, an oncogene implicated in adenoid cystic carcinoma (ACC), colorectal cancer (CRC), acute myeloid leukemia (AML), and other solid tumors. Rgenta’s proprietary platform identifies RNA processing events that can be targeted by small molecules to modulate interactions within the spliceosome and RNA regulatory proteins, unlocking new therapeutic possibilities for historically undruggable targets.

Expanding Therapeutic Horizons

While oncology remains a key focus, Rgenta’s platform has the potential to address a wide range of diseases, including neurological disorders, cardiovascular conditions, rare diseases, and immunology. The collaboration signals a significant step forward in harnessing RNA-targeting medicines to address unmet medical needs.

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