Novel platform may lead to more improved gene therapies

Researchers from RCSI University of Medicine and Health Sciences in Ireland have developed polypeptide-based materials that serve as effective vectors in delivering gene therapies.

Professor Sally-Ann Cryan, the study’s senior author and Professor of Drug Delivery, RCSI. Image Credit: RCSI

The team has developed the first-of-its-kind platform, which allows vectors to adapt depending on the specific gene therapy cargo. The research was conducted by members of the RCSI University of Medicine and Health Sciences and was financially supported by Science Foundation Ireland. The study has been published in the Biomaterials Science journal.

The main challenge posed by gene therapy is the delivery of genetic information to the host cells. In Covid-19 vaccines that use mRNA technology, the genetic information is delivered in a lipid nanoparticle into host cells to maintain its stability. The success of COVID vaccines has also established the use of nanoparticles for developing many advanced therapies.

The researchers created a new platform that generates bespoke star-shaped polypeptide nanoparticles. These nanoparticles effectively deliver a wide range of therapies, including gene therapies.

Most significantly, these polypeptides, when compared to lipids, are more flexible and much easier to handle. The researchers demonstrated the potential of this material by using it to deliver a gene therapy that regenerated bones.

In a preclinical study, the material was loaded with DNA molecules that promote the regeneration of bones and blood vessels. The nanomedicines were placed in a scaffold that could be implanted into the site of defect and deliver the genetic cargo into the infiltrating host cells. The gene-loaded scaffold accelerated the regeneration of bone tissue, with a six-fold increase in new bone formation when compared to a scaffold alone.

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, Study Senior Author and Professor of Drug Delivery, RCSI

According to Professor Andreas Heise, “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.”

Professor Andreas Heise is also the project collaborator and a Professor of Polymer Chemistry at RCSI.

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.”

Professor Andreas Heise, project collaborator, and Professor of Polymer Chemistry, RCSI