Scientists have developed polypeptide materials that act as efficient vectors for the delivery of gene therapy. The unique platform allows the vectors to be tailored to the specific gene therapy shipment.
The work, led by researchers at the RCSI University of Medicine and Health Sciences and funded by the Science Foundation Ireland, is published in Biomaterials Sciences.
A major challenge for gene therapies is to prepare them in such a way as to transmit genetic information to host cells. For Covid-19 vaccines that use mRNA technology, genetic information is delivered in a lipid nanoparticle to maintain its stability and deliver it to cells. The success of COVID vaccines has established that nanoparticles are the key to the development of many advanced therapies.
Researchers have developed a platform that produces bespoke star-shaped polypeptide nanoparticles, which effectively deliver a range of therapies, including gene therapies. Importantly, these polypeptides are more flexible and easier to handle than lipids. To demonstrate the potential of this material, the researchers used it to deliver gene therapy that regenerates bone.
In preclinical work, researchers loaded the material with DNA molecules that promote regrowth of bones and blood vessels. They placed these nanomedicines in a scaffold that could be implanted in a defective site and deliver the genetic cargo into the infiltrating host cells. The gene-laden scaffold accelerated the regeneration of bone tissue, with a six-fold increase in new bone formation compared to scaffolding alone.
“With the success of COVID-19 vaccines, the potential of gene therapies becomes evident, and advanced nanoparticle delivery systems are essential to enable their clinical use. We have shown that these nanoparticles have real game-changing potential in the delivery of gene therapy, ”said Professor Sally-Ann Cryan, lead author of the study and Professor of Drug Delivery, RCSI.
“Although more testing is needed before these therapies can be used in the clinic, our platform allows us to design our polypeptides to meet a variety of delivery scenarios and provide tailored solutions to the challenges of gene delivery. Added Professor Andreas Heise, Project Collaborator and Professor of Polymer Chemistry, RCSI.
“We are developing this patented technology for commercialization, with the support of an Enterprise Ireland Commercialization Fund Award, and seeking expressions of interest from industry partners and investors.
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