RNA therapeutics and LNP delivery characterisation

Unlocking the promise of RNA therapeutics

Delivering an RNA therapeutic payload to its target cell is only half the challenge. For lipid nanoparticles, conjugates, and viral delivery systems alike, what happens after internalisation, whether the payload escapes the endosome and reaches the cytosol, determines whether the therapeutic works.

The pharmaceutical industry holds decades of established know-how for small molecules. Characterising RNA therapeutic and LNP delivery requires a new standard of specialised expertise.

Our integrated evaluation platform provides the detailed, multi-stage characterisation necessary to advance RNA therapeutics, lipid nanoparticles, and other complex novel modalities through the drug discovery pipeline. By accelerating your hit identification process, we reduce early-stage development costs while partnering with you to build a robust Target Product Profile roadmap through to IND application.

Our development framework is purposefully aligned with the expectations of regulators, big pharma, and leading investors, ensuring we deliver the data packages you need to meet regulatory standards, derisk your asset, and successfully secure future funding.

Intracellular Delivery Targeted and Extrahepatic Delivery

How we help: We evaluate and optimise RNA payload and vehicle formulation for target engagement — achieving therapeutic expression, depletion, or modification — while maximising efficacy and TPP alignment.

High-throughput formulation screening with target-defined endpoints
Potency (EC50) determination in immortalised and primary cell models
Evaluation of cellular uptake, endosomal rupture, and target expression
Mechanistic determination of active and passive targeting processes

How we help: We optimise targeting strategy, payload release, and clearance profile to ensure sufficient and specific cellular exposure in vivo, maximising efficacy at a sparing dose.

Dynamic in vivo imaging of the payload or particle to understand biodistribution
Ex vivo evaluation of multiorgan cell-type-specific tropism and target expression
Orthogonal and complex cell models
Utilisation of orthotopic and syngeneic disease models

TPP Definition

Target Identification

Target Validation

Hit Identification

Hit to Lead

Lead Optimisation

Candidate in vivo

TPP Definition

Consultancy

Define clear clinical goals with insight into regulatory, safety and efficacy considerations
Build robust, IP-ready plans with timelines, budgets and milestones
Identify and address key risks to candidate nomination and regulatory progression
Flexible collaboration with MDC’s labs and CRO network to rapidly deliver

Target Identification

Consultancy

Define clear clinical goals with insight into regulatory, safety and efficacy considerations
Build robust, IP-ready plans with timelines, budgets and milestones
Identify and address key risks to candidate nomination and regulatory progression
Flexible collaboration with MDC’s labs and CRO network to rapidly deliver

Target Validation

Services

High-throughput formulation screening with target-defined endpoint
Potency (EC50) determination in immortalised and primary cell models
Evaluation of cellular uptake, endosomal rupture, and target expression
Mechanistic determination of active and passive targeting processes

Hit Identification

Services

High-throughput formulation screening with target-defined endpoint
Potency (EC50) determination in immortalised and primary cell models
Evaluation of cellular uptake, endosomal rupture, and target expression
Mechanistic determination of active and passive targeting processes
Dynamic in vivo imaging of the payload or particle to understand biodistribution
Utilisation of orthotopic and syngeneic disease models
Ex vivo evaluation of multiorgan cell type-specific tropism and target expression
Orthogonal and complex cell models

Hit to Lead

Services

High-throughput formulation screening with target-defined endpoint
Potency (EC50) determination in immortalised and primary cell models
Evaluation of cellular uptake, endosomal rupture, and target expression
Mechanistic determination of active and passive targeting processes
Dynamic in vivo imaging of the payload or particle to understand biodistribution
Utilisation of orthotopic and syngeneic disease models
Ex vivo evaluation of multiorgan cell type-specific tropism and target expression
Orthogonal and complex cell models

Lead Optimisation

Services

Dynamic in vivo imaging of the payload or particle to understand biodistribution
Utilisation of orthotopic and syngeneic disease models
Ex vivo evaluation of multiorgan cell type-specific tropism and target expression
Orthogonal and complex cell models

Candidate in vivo

Services

Dynamic in vivo imaging of the payload or particle to understand biodistribution
Utilisation of orthotopic and syngeneic disease models
Ex vivo evaluation of multiorgan cell type-specific tropism and target expression
Orthogonal and complex cell models

Sarah Brockbank

Strategy Leader

Sarah Brockbank leads the strategy for Complex Medicines at Medicines Discovery Catapult. In this role she draws on internal expertise and the external environment, through membership of national programmes and a portfolio of client projects, to develop and implement a core strategy for complex medicines that is responsive to the needs of this emerging area.

Sarah has extensive experience in the pharmaceutical industry. She is a molecular biologist with a focus mainly in target identification and validation and has worked across a broad range of disease areas. Sarah was a Drug Discovery Project Leader at AstraZeneca, and she also has extensive experience in the leadership and management of large public-private consortia and collaborations.

Dr Phil Auckland

Lead Scientist

A chromosome biologist by training, Phil has spent 12 years utilising fluorescence microscopy to visualise molecular processes at the single-cell level.

Phil completed his PhD and early postdoctoral studies at Warwick University, where he developed imaging methods to understand the force-generating mechanisms that move chromosomes during cell division. He then moved to Rice University as a Fellow to work on cell-fate decision making during early human development.

In 2021, he established an independent research group at the Randall Centre for Cell and Molecular Biophysics at King’s College London. He joined Medicines Discovery Catapult to lead the Advanced Microscopy team in 2023.

In his current role, Phil develops bespoke microscopy assays for the mechanistic characterisation of disease phenotypes and novel medicines. He also leads the establishment of novel imaging technologies to ensure that the MDC is at the forefront of microscopy-based drug discovery.