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New oligonucleotide analogues for therapeutic applications

Reference	BB/S018794/1
Principal Investigator / Supervisor	Professor Tom Brown
Co-Investigators / Co-Supervisors
Institution	University of Oxford
Department	Oxford Chemistry
Funding type	Research
Value (£)	463,172
Status	Completed
Type	Research Grant
Start date	03/06/2019
End date	19/11/2021
Duration	30 months

Abstract

unavailable

Summary

Therapeutic oligonucleotides (Th-ONs) are rapidly becoming highly important agents for hard to treat diseases. They target mRNA thereby changing protein expression. With four Th-ONs now in the clinic; Eteplirsen, Nusinersen, Mipomersen and Patisiran, the market is worth billions of dollars. Moreover, with ~150 Th-ONs currently in clinical trials for cancer and other diseases the field is set for rapid commercial growth. Chemical modifications are essential to make ThONs bind tightly to their RNA targets, to resist enzymatic degradation in vivo and improve uptake into cells. Further improvements in these areas are urgently needed to improve efficacy, reduce toxicity and lower costs. In a BBSRC-funded project we have designed an entirely new class of ThONs (LNA-triazole and LNA-amide) to address these problems and we now plan to evaluate them in biological assays. When this data has been obtained we will file additional patents and set up a new company to exploit the technology.

Committee	Not funded via Committee
Research Topics	Technology and Methods Development
Research Priority	X – Research Priority information not available
Research Initiative	Follow-On Fund Super (SuperFOF) [2012-2015]
Funding Scheme	X – not Funded via a specific Funding Scheme

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