Despite progress in understanding the functions of novel long non-coding RNAs, their applications as therapeutic tools have been very limited – but that’s about to change thanks to a £9.1million seed round for Transine Therapeutics.
The new company – founded in 2020 – has emerged from stealth to advance its novel class of therapeutic RNAs following the closure of the round, which was co-led by Takeda Ventures, Inc and the Dementia Discovery Fund.
The Babraham Research Campus company has developed a novel class of therapeutic RNAs based on its pioneering SINEUP platform technology.
SINEs – short interspersed nuclear elements – are long, non-coding RNAs containing repetitive sequences, and they upregulate the translation of specific mRNAs with complementary base sequences, hence SINEUP.
SINEUPs offer a novel class of therapeutic RNAs which upregulate protein expression with unprecedented control and specificity. They precisely bind to a selected mRNA and boost expression of almost any protein of choice by enhancing translation. This astonishing and newly-identified mechanism addresses diseases beyond the reach of small molecules, conventional biologics or gene therapies.
Transine’s development is based on the pioneering work of world-renowned geneticists Professors Stefano Gustincich and Piero Carninci, who have identified this functional and naturally-occurring class of long non-coding RNAs.
Prof Gustincich, co-founder of Transine Therapeutics and director of the Central RNA Laboratory at the Italian Institute of Technology in Genoa, 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.”
Speaking of the finance round, Christian Jung, partner at the Dementia Discovery Fund (DDF), said: “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. We look forward to working with Transine to realise the full potential of the company’s transformational technology platform.”
The financing will enable Transine to further develop its proprietary platform towards building a pipeline of novel mRNA-targeted therapeutics initially focused on central nervous system and ophthalmology applications.
“We have known Prof Piero Carninci and Prof Stefano Gustincich and followed their ground-breaking research for many years and are pleased to contribute to the creation of Transine,” noted Rob Woodman, senior partner at Takeda Ventures. “We are delighted to be joined by DDF as the company rapidly grows.”
Prof Carninci, co-founder of Transine Therapeutics and head of the Genomics Research Center at Human Technopole in Milan, Italy, said: “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.”
Published on 11.06.21Article sourced from Cambridge Independent