Pharma giant GSK is buying into Rgenta Therapeutics’ mission to develop a pipeline of oral RNA-targeting small molecules. The new partners aim to develop new splice modulators for multiple disease areas, including oncology, with Rgenta able to receive up to nearly $500 million biobucks per target.
The British Big Pharma is paying Rgenta up to $46 million in cash upfront and pre-option milestone payments, according to a Dec. 4 release. The Massachusetts-based biotech can also earn royalties on potential therapeutics that arise from the pact, plus a future equity investment.
More specifically, Rgenta will use its RNA-targeting discovery platform to develop new splice modulators against multiple targets set by GSK. Should GSK exercise its options, the pharma will then be responsible for bringing drug candidates forward. The Big Pharma also has the option to expand the deal to include additional targets.
Besides oncology, details about what indications the companies would be pursuing were not revealed, though Rgenta says its approach could be used across neurological disorders, cardiovascular and metabolic conditions, rare diseases and immunology, among others.
“We are excited about Rgenta’s differentiated approach to discover and develop oral small molecule splice modulators for high-value targets,” GSK’s Senior Vice President of Research Technologies Christopher Austin, M.D., said in the release. “We are dedicated to partnering with leading companies to complement our existing expertise in RNA-targeting medicines and look forward to advancing this promising modality to more patients with difficult-to-treat diseases."
Rgenta launched in 2020 with seed money from Boehringer Ingelheim Venture Fund, among others, and later raised a $52 million series A in 2022. The biotech is set to present preclinical data for its lead program, RGT-61159, at the American Society of Hematology annual meeting in San Diego on Dec. 9. RGT-61159 is currently in a phase 1a/b clinical trial in patients with advanced relapsed or refractory adenoid cystic carcinoma or colorectal cancer.
Before being translated into proteins, mRNA molecules are processed by the cell through various mechanisms. In order to circumvent the undruggable nature of some proteins, Rgenta is working to develop molecules that target the cell’s RNA processing machinery to prevent disease-causing proteins from being made in the first place.