Gene Therapy Targeting – What role can Nanoparticles play?

“With the success of the COVID-19 vaccines, the potential of gene therapies is becoming apparent and advanced nanoparticle delivery systems are key to enabling their use clinically. We have shown that these nanoparticles have real potential to be a game changer in the delivery of gene therapies -” Sally-Ann Cryan.

A platform that produces polypeptide-based materials as vectors for specific gene therapy cargos has been developed by researchers from RCSI University of Medicine and Health Sciences, Dublin. Delivering genetic information into host cells presents a major challenge; Covid-19 vaccines that use mRNA technology, for example, deliver the genetic information in a lipid nanoparticle and the success of the COVID vaccines has established nanoparticles as key to the development of many advanced therapies.

Supported by Science Foundation Ireland, the RCSI research team’s platform is designed around bespoke star-shaped polypeptide nanoparticles as vectors which are, crucially, more flexible and easier to handle than lipids. In pre-clinical work, the material loaded with DNA molecules that promote bones and blood vessels to regrow, were placed in a scaffold that could be implanted into a defect site where the genetic cargo infiltrated host cells. The gene-loaded scaffold accelerated bone tissue regeneration, with a six-fold increase in new bone formation compared to a scaffold alone.

Nanoparticle delivery systems are key

“With the success of the COVID-19 vaccines, the potential of gene therapies is becoming apparent and advanced nanoparticle delivery systems are key to enabling their use clinically. We have shown that these nanoparticles have real potential to be a game changer in the delivery of gene therapies,” said Professor Sally-Ann Cryan, the study’s senior author and Professor of Drug Delivery, RCSI.

“While more testing is needed before these therapies can be used clinically, our platform allows us to design our polypeptides to meet a variety of delivery scenarios and provide tailored solutions to gene delivery challenges,” added Professor Andreas Heise, project collaborator and Professor of Polymer Chemistry, RCSI.  

Industry partners sought

“We are developing this patent-protected technology towards commercialisation, with support from an Enterprise Ireland Commercialisation Fund Award and are seeking expressions of interest from industry partners and investors.”

Published in Biomaterials Science.

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