GSK Partners with Rgenta to Develop Oral Small Molecule Splice Modulators Targeting RNA

December 20244Day，Rgenta Therapeutics Announces Multi-Year, Multi-Target Strategic Research Alliance with GSK to Advance the Discovery and Development of Novel Small Molecule Splice Modulators Targeting RNA for Multiple Disease Areas, Including Oncology.

According to the terms of the agreement, Rgenta will receive up to $46 million in cash as an upfront payment and pre-option exercise milestone payments. Additionally, the company has the potential to earn nearly $500 million through option exercise fees, research, development, regulatory and commercial milestones, tiered royalties, and potential equity investments. GSK also has the right to expand the scope of the alliance to include additional targets. Under the collaboration framework, Rgenta will leverage its proprietary discovery platform to develop novel oral small molecule splicing modulators targeting RNA for GSK-specified targets. GSK will be responsible for the further development and commercialization of any drug candidates emerging from the alliance.

Christopher Austin, Ph.D., Senior Vice President of Research Technologies at GSK, stated: "We are excited about Rgenta's differentiated approach to discovering and developing oral small molecule splicing modulators for high-value targets. We are committed to collaborating with leading companies to complement our existing expertise in RNA-targeted therapeutics, and we look forward to bringing this promising modality to more patients suffering from refractory diseases."

Rgenta Co-founder and CEO Simon Xi, Ph.D., stated, "We are delighted to partner with leading biopharmaceutical company GSK. This alliance further validates the potential of Rgenta's small molecule RNA-targeting drug discovery platform." Rgenta Co-founder, President, and Chief Scientific Officer Travis Wager, Ph.D., added, "The platform has successfully delivered our first clinical-stage asset—RGT-61159, an oral small molecule candidate designed to specifically modulate the splicing of the transcription factor MYB."

AboutRgenta Therapeutics

Rgenta Therapeutics is a clinical-stage biotechnology company dedicated to developing a range of targeted RNA oral small molecule drugs for oncology and neurological disorders. One of Rgenta's key development programs, RGT-61159, is an oral small molecule designed to specifically modulate the splicing of the transcription factor MYB. It is currently in Phase 1a/b studies and has the potential to treat adenoid cystic carcinoma (ACC), colorectal cancer (CRC), other solid tumors, and acute myeloid leukemia (AML). Rgenta’s proprietary technology platform identifies RNA targets that can be selectively engaged by small molecules and designs molecular glues to modulate spliceosomes and regulate interactions between proteins and RNA. With its unique approach and core programs, Rgenta focuses on unlocking the therapeutic potential of traditionally undruggable targets.