New Opportunities with New Technology 

Advances in molecular technology have meant that previously inaccessible drug targets are now becoming reachable using a new generation of therapeutics including drug conjugates, oligonucleotides, and new gene silencing modalities. These modalities often require drug delivery technologies, which can pass biological barriers to deliver the molecular cargo. The delivery technologies also may allow poorly soluble or unstable therapeutic moieties, or otherwise toxic compounds, to be used with reduced side effects. 

The mRNA COVID-19 vaccines from Moderna and Pfizer/BioNTech, have begun to transform commercial views of nanoparticle technology. There is potential for bold biotechs to rapidly develop investable propositions. 

Improving Success 

How do you take a novel drug delivery technology, or a novel drug modality, from initial pre-clinical research through to a clinically ready drug candidate? Expert advice and practical support is needed for technology characterisation  from the early pre-clinical research stage, advancing to industrial validation, through to clinical trials and regulatory approval. 

There are five main processes in the characterisation of a complex medicine: Circulation; Accumulation; Penetration; Internalisation; and Release of the active pharmaceutical ingredient. 

Each process is a potential barrier to success of the carrier and cargo technologies, and needs to be tracked from the systemic level to the subcellular level. Relevant technologies to deploy include positron emission tomography imaging of biodistribution, high throughput mass spectrometry, tissue IHC, spatial transcriptomics, confocal and super-resolution imaging, mass spectrometry, and tissue-imaging using confocal and super-resolution microscopy. These diverse methodologies allow the key processes to be monitored and the carrier and cargo technology to be modified if necessary to achieve success.

Thse are expensive, specialist technologiers, so the technical barriers for a new SME include obtaining access to imaging facilities for pre-clinical investigations; and the cost and technical difficulty of development of new bioanalytical methods and validation assays.  Also, improved access to scalable manufacturing is needed - through a better understanding of product composition, physiochemical properties, stability, delivery and performance, these technologies will become more readily  industrialised. Beyond these technical aspects, challenges for the SME include an understanding of the requirements of an investor or large pharma partner;  access to key sectoral players for collaborative partnerships; and access to drug development know-how. 

Ways Forward

Future success would involve transforming national productivity with a multi-faceted approach. This would include greater support for technology innovation, business growth and skills development, along with facile access to complex characterisation facilities, business support and accelerators. 

A national laboratory network would provide a single-entry point for companies to receive support from pre-clinical assessment, process development, scalability and manufacturing. Funders could offer targeted funding to support drug development, clinical trials design and regulatory issues. Expert drug discovery advisors can help provide confidence to investors through clear strategic development plans. Finally, ambitious national partnerships and collaborations would bring experiences and skills together to help push forward product development. 

I have been involved in >20 complex medicines projects, from large scale companies through to early-stage academic collaborators. So, Peter Simpson Consulting Ltd can help SMEs navigate this complex experimental, strategic and funding landscape - to identify partners for efficacy, biodistribution, safety and scaleup; to consider key questions to achieve interest from Pharma or investors; and to have confidence that their technology is viable through a lean, clear strategic drug development focus. 

Contact us if you’d like to discuss further!