Nanoparticle platform enables enhanced delivery of gene therapies

Scientists have developed polypeptide-based materials that can act as effective vectors for delivering gene therapies. According to the researchers, from the Royal College of Surgeons in Ireland (RCSI), this first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

The team say that a major challenge for gene therapies is preparing them in a way that can deliver the genetic information into the host cells. They developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies. The researchers say that, crucially, these polypeptides are more flexible and easier to handle than the lipids that are used to deliver and maintain the stability of the mRNA technology of COVID-19 vaccines. To demonstrate the potential of this material, the researchers used it to deliver a gene therapy that regenerated bone.

In pre-clinical work, the team loaded the material with DNA molecules that promote bones and blood vessels to regrow. They placed these nanomedicines in a scaffold that could be implanted into a defect site and deliver the genetic cargo into infiltrating host cells. The gene-loaded scaffold accelerated bone tissue regeneration, with a six-fold increase in new bone formation 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,” said Professor Sally-Ann Cryan, the study’s senior author. 

“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. 

The work is published in Biomaterials Science.