Gene therapy technology secures funding with a promise to lower costs and transform rare disease management

Pharma News: Researchers from the Centenary Institute and the Sydney Local Health District (SLHD) have secured a substantial $500,000 grant from CUREator, Australia’s renowned national biotech incubator. The funding aims to propel the development of cutting-edge technology that promises to revolutionise the safe and effective delivery of gene therapy for rare diseases.

This ambitious project will be spearheaded by a team of scientists and experts in a newly established startup called AAVec Bio. Their primary focus will be advancing adeno-associated virus (AAV) technology, a remarkable bioengineered virus that acts as a carrier to transport healthy genes to targeted cells. Harnessing the power of this technology holds immense potential for curing a wide range of genetic diseases that have long plagued humanity.

Under the leadership of Professor John Rasko AO, renowned for his contributions to the field, the research team aims to refine AAV technology to ensure more efficient delivery of therapeutic genes to the precise cells within the human body. By doing so, they hope to overcome several current limitations of AAV-based gene therapy, such as the requirement for high AAV doses to achieve therapeutic effects, dose-related toxicities, and the high costs associated with manufacturing.

“The success of gene therapy hinges on the precise delivery of therapeutic agents to the targeted cells within the body,” explained Professor Rasko. “Our innovative platform has the potential to significantly enhance human gene therapies, leading to more effective treatments for individuals with unmet medical needs. Not only would this result in significantly reduced dosage requirements, but it would also minimize toxicities and side effects, ultimately improving patient outcomes.”

Another groundbreaking aspect of this research lies in its potential to significantly reduce the manufacturing costs of gene-based drugs. The researchers anticipate that their innovative AAV approach could slash production expenses by a staggering five- to ten-fold, thanks to the reduced amount of therapeutic material needed.

“This cost-saving breakthrough has the power to transform the gene therapy market, making these life-changing treatments more accessible and affordable for patients,” added Professor Rasko.

In their quest to validate the utility of their technology, the researchers have chosen to focus their initial efforts on Pompe disease. This rare genetic disorder, affecting approximately 1 in 40,000 individuals worldwide, results in progressive muscle weakness and heart damage due to the body’s inability to break down glycogen, which accumulates in various tissues.

Sanofi’s Nexviazyme (avalglucosidase alfa), an IV monotherapy that was TGA registered in November 2021, is now funded through the Life Saving Drugs Program (LSDP) for Pompe disease.

Dr Chuck Bailey, senior scientist at the Centenary Institute and AAVec Bio, shed light on this promising direction. He said “By employing gene therapy to replace the defective gene responsible for Pompe disease, we have the potential to achieve a long-lasting cure, while simultaneously demonstrating the proof-of-concept for our AAV technology and the effectiveness of our groundbreaking approach.”

Excitement surrounding this breakthrough technology is palpable, as it has the potential to transform the lives of millions of Australians suffering from a myriad of rare diseases. The researchers hope that their novel AAV technology will pave the way for improved gene therapies, granting renewed hope to patients facing medical conditions that were once considered insurmountable obstacles. With this innovative solution, a new era in medicine is dawning, promising accessible treatments for all those in need.

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