Co-development programmes, anonymised

The challenge

The partner had a potent siRNA against an oncology target, but its standard lipid nanoparticle accumulated mainly in the liver and gave no measurable knockdown in tumour tissue.

Our approach

• Screened 48 ionisable lipid and PEG-lipid combinations against tumour and hepatocyte cell panels.
• Introduced an acid-labile surface layer so the carrier reverses charge only below pH 6.8.
• Co-labelled the formulation with quantum dots to measure tumour and liver distribution in the same animal.

Outcomes

4.1×

higher tumour-to-liver ratio than the starting formulation

62%

target mRNA knockdown in xenograft tumour tissue

14 months

from feasibility study to candidate selection

Stage: Feasibility → co-development
Model: Co-development with milestone payments

The challenge

The institute's saRNA candidate lost potency within days at 2–8 °C, so every dose had to be shipped and stored frozen — impractical for its target regions.

Our approach

• Mapped degradation pathways with capillary electrophoresis and particle-size tracking over 12 weeks.
• Re-balanced helper lipid and cholesterol content to reduce lipid hydrolysis at the particle surface.
• Developed a sucrose–trehalose lyophilisation cycle and confirmed potency after reconstitution.

Outcomes

6 months

at 2–8 °C with RNA integrity above the release specification

< 10 nm

change in particle size after lyophilisation and reconstitution

1 process

transferred unchanged from bench to 10 L continuous-flow scale

Stage: Formulation → GMP technology transfer
Model: Fee-for-service development with a supply agreement

The challenge

The partner's antisense oligonucleotide needed intrathecal dosing, but the only exposure measure was terminal tissue analysis, which slowed every dose-ranging decision.

Our approach

• Designed a hybridisation-based quantum-dot assay for the oligonucleotide and its main metabolite.
• Validated it in cerebrospinal fluid against LC-MS reference measurements.
• Packaged the method for transfer to the partner's contract research organisation.

Outcomes

50 pM

lower limit of quantification in cerebrospinal fluid

r = 0.97

agreement with the LC-MS reference method

9 weeks

saved per dose-ranging cycle

Stage: Assay development → method transfer
Model: Fixed-scope assay development with licence