Cambridge startup Transine Therapeutics has emerged from stealth with £9.1 million seed funding – co-led by Takeda Ventures, Inc. and the Dementia Discovery Fund (DDF) – to advance a novel class of therapeutic RNA to combat hard to treat diseases.
The funding will enable Transine to further develop its proprietary platform towards building a pipeline of novel mRNA-targeted therapeutics initially focused on the Central Nervous System and Ophthalmology applications.
Transine is based on the pioneering work of world-renowned geneticists Professors Stefano Gustincich and Piero Carninci who identified a new functional and naturally occurring class of long non-coding RNAs, named SINEUPs.
SINEUPs® bind in a highly specific manner to their target mRNA to elevate the level of a protein by enhancing protein translation. The attributes of synthetic SINEUPs®, universality, specificity and tunability, together with safety controls, afford this platform broad applicability with the potential to address diseases which have been beyond the reach of small molecules, conventional biologics or gene therapies.
Transine’s technology massively extends the druggable proteome and represents an entirely unique mechanism of action for currently hard-to-treat diseases.
The modular nature of the technology enables Transine to rapidly design SINEUPs® with high specificity for almost any target for which a mRNA is present.
The action of SINEUPs® on translation is controlled and the resulting increase in protein expression is maintained within a physiological range. Their size is optimised to enable a delivery-agnostic approach of therapeutic SINEUPs® that includes delivery as naked oligonucleotides, or via the use of viral vectors such as Adeno-associated virus (AAV), or non-viral systems.
Prof. Gustincich said: “Long non-coding RNAs are emerging as key cellular regulators which could be exploited as new therapeutic approaches for multiple indications. Transine SINEUPs® belong to this family and can be engineered to target almost any protein with exquisite specificity.”
Prof. Carninci, added: “Transine SINEUPs® target endogenous mRNA and the naturally controlled cellular process of translation to precisely elevate protein expression safely and effectively within the physiological range, significantly limiting any potential side effects from over-expression or off-target effects.”
In connection with the financing, Rob Woodman, senior partner at Takeda Ventures, and Christian Jung – partner at the DDF – have joined Transine’s board.
Woodman said: “We have known Professor Carninci and Professor Gustincich and followed their ground-breaking research for many years and are pleased to contribute to the creation of Transine.”
Jung added: “If the impressive proof-of-concept data with SINEUPs® in a variety of in vitro and in vivo disease models translates into the clinic, Transine has the potential to be a game-changer in the way neurodegenerative and other diseases are being treated.
“SINEUPs® enable entirely novel therapeutic approaches that cannot be pursued with currently available technologies.”