RARE Daily

Transine $12.8 Million Seed Funding to Advance Novel Class of Therapeutic RNAs

June 2, 2021

U.K.-based Transine Therapeutics emerged from stealth and Rraised $12.8 million (£9.1 million) in seed funding to advance its proprietary platform towards building a pipeline of novel mRNA-targeted therapeutics initially focused on the central nervous system and ophthalmology applications.

Photo: Stefano Gustincich, co-founder of Transine Therapeutics

Takeda Ventures and the Dementia Discovery Fund co-led the financing.

Transine is based on the work of academic geneticists Stefano Gustincich and Piero Carninci who identified a new functional and naturally occurring class of long non-coding RNAs that they named SINEUPs.

“Long non-coding RNAs are emerging as key cellular regulators which could be exploited as new therapeutic approaches for multiple indications,” said Stefano Gustincich, co-founder of Transine Therapeutics and associate director for Technologies for Life Science, and director of the Central RNA Laboratory at the Italian Institute of Technology. “Transine SINEUPs belong to this family and can be engineered to target almost any protein with exquisite specificity.”

SINEUPs bind in a highly specific manner to their target mRNA to elevate the level of a protein by enhancing protein translation. Transine says the attributes of synthetic SINEUPs—universality, specificity and tunability, together with safety controls—afford this platform broad applicability with the potential to address diseases that have been beyond the reach of small molecules, conventional biologics, or gene therapies.

The platform enables the action of SINEUPs on translation to be controlled so the resulting increase in protein expression is maintained within a physiological range. Their size is optimized 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.

“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,” said Piero Carninci, co-founder of Transine and head of the Genomics Research Center, Functional Genomics Programme at Human Technopole, and deputy center director of the Riken Center for Integrative Medical Sciences.

“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,” said Christian Jung, partner at the Dementia Discovery Fund.

In connection with the financing, Rob Woodman, senior Partner at Takeda Ventures, and Christian Jung, partner at the DDF have joined Transine’s Board.

Author: Rare Daily Staff

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