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Nanostar Sieving for Oligonucleotide Therapeutics

Project description

Getting the right polymer sequence at the right cost

Millions of molecules are made of repeating units of individual building blocks (monomers). DNA and RNA are important natural polymers made of repeating nucleotides as building blocks. The manufacture of short strands of chemically modified RNA (oligonucleotides) has shown great promise for the development of novel therapies. It is currently achieved using solid-phase synthesis in which the growing oligo is bound to an insoluble substrate and monomers are added one at a time. Solid-phase synthesis is difficult to scale and the resulting purity is low, leading to high prices. The UK start-up Exactmer has developed patented technology enabling polymer synthesis entirely in the liquid phase to overcome these obstacles. The EU-funded NANOLIGO project will bring this technology to market with the potential to significantly speed the development of novel complex polymeric pharmaceuticals and slash manufacturing costs.

Objective

Since the Nobel Prize winning discovery of small interfering RNA its specificity and selectivity have driven the pharmaceutical industry to design 15- to 25-mer oligonucleotide analogues as a new class of drugs. These offer exciting new therapeutic possibilities for example in rare diseases, but also large patient population indications including cardiovascular disease, and viral pandemics such as Covid-19. However, the high cost and limited scalability of manufacturing these complex polymeric molecules using solid phase synthesis is holding back benefits for thousands of patients – and has motivated us to seek an improved technology for oligo synthesis.

ERC Advanced Grant 786398 is funding research investigating a new technology platform for the fabrication of defined monomer sequence polymers - Nanostar Sieving. Our preliminary research into oligo synthesis using Nanostar Sieving has found that it is potentially both feasible and game-changing. NANOLIGO seeks proof-of-concept that Nanostar Sieving can unlock the tremendous potential of oligos through dramatically reducing production cost and increasing production capacity. We will work with RNA oligos – key targets for novel APIs – which are currently produced by solid phase synthesis, and show that these can be made entirely in the liquid phase, with unprecedentedly high purity, efficient chemistry and lower solvent consumption. Crucially, membrane separations are readily scaled up, making this process a strong candidate for kg-scale oligo production.

The PI and members of his research team have founded a start-up, Excatmer, to ensure the results of the ERC Grant have impact. We will carry out the PoC project at Exactmer, so that if we are successful the technology can be more rapidly developed and scaled up, leading to significant economic benefits. We are driven by a desire to make oligonucleotide therapies affordable and so available to patients as rapidly as possible.

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Programme(s)

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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.

ERC-POC-LS - ERC Proof of Concept Lump Sum Pilot

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Call for proposal

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

(opens in new window) ERC-2020-PoC

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Host institution

EXACTMER LIMITED
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
WINDSOR HOUSE CORNWALL ROAD
HG1 2PW HARROGATE
United Kingdom

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Yorkshire and the Humber North Yorkshire North Yorkshire CC
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
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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)

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