Skip to main content
Go to the home page of the European Commission (opens in new window)
English en
CORDIS - EU research results
CORDIS

Enzymatic STEreospecific RNA phosphorothioate modification for next-generation therapeutics

Objective

RNA therapeutics have emerged as a powerful medical modality, from COVID-19 vaccines to treatments for rare genetic diseases and cancer. However, a fundamental bottleneck limits their potential: RNA instability. Most approved RNA drugs rely on phosphorothioate (PS) modifications for stability and delivery, but current chemical synthesis produces stereochemical mixtures reducing efficacy, increasing toxicity, and complicating regulatory approval. Moreover, chemical PS synthesis is restricted to short RNAs, completely excluding therapeutic mRNAs from site-specific backbone optimization despite evidence that strategic PS placement can dramatically enhance translation efficiency. This represents a fundamental technological gap constraining millions of patients with rare genetic diseases and cancer needing personalized immunotherapies from accessing safer and more effective RNA medicines.
Our ERC-funded discovery revealed the first naturally occurring stereospecific RNA PS modifications and the enzyme family responsible for their installation. This breakthrough provides a biological route to manufacturing next-generation RNA therapeutics, simultaneously eliminating stereochemical heterogeneity, enabling cost-effective stereopure synthesis (≥10x cheaper), and uniquely installing site-specific PS modifications in full-length mRNAs.
This PoC will advance our technology from TRL 3 to TRL 4 through three objectives targeting industrial readiness: 1) we will demonstrate scalable enzymatic PS installation to produce stereopure modified RNA, 2) we will engineer programmable substrate specificity to create variants targeting therapeutic ASOs and mRNAs and 3) we will demonstrate commercial feasibility through techno-economic analysis and engagement with RNA therapeutics companies. Our success will address the unmet patient needs while unlocking market opportunities in the $6.5B ASO therapeutics and $11.4B mRNA therapeutics markets currently constrained by manufacturing limitations

Fields of science (EuroSciVoc)

CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.

You need to log in or register to use this function

Programme(s)

Multi-annual funding programmes that define the EU’s priorities for research and innovation.

Topic(s)

Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.

Funding Scheme

Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.

HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

See all projects funded under this funding scheme

Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

(opens in new window) ERC-2026-POC

See all projects funded under this call

Host institution

WEIZMANN INSTITUTE OF SCIENCE
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 150 000,00
Address
HERZL STREET 234
7610001 Rehovot
Israel

See on map

Activity type
Higher or Secondary Education Establishments
Links
Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

No data

Beneficiaries (1)

My booklet 0 0