#USA #Houston #PackGene #AAV Vector #CMRI

PackGene Partners with Children's Medical Research Institute

In a groundbreaking move aimed at accelerating advancements in gene therapy, PackGene Biotech has forged a strategic licensing agreement with the Children's Medical Research Institute (CMRI). This collaboration focuses on the development of barcoded AAV (Adeno-Associated Virus) capsid screening kits. These innovative kits are set to transform the landscape of gene therapy research by enabling rapid, high-throughput screening for optimal AAV capsids suitable for targeted tissue delivery, crucial for therapeutic product development.

Unveiling the Partnership

PackGene, a leader in AAV vector technologies, and CMRI, renowned for its pioneering work in gene therapy, have combined their expertise through this strategic partnership. The kits they introduce will offer researchers the ability to perform in vitro, ex vivo, and in vivo screenings, effectively streamlining the process of identifying the most efficient AAV capsids. These capsids are vital for ensuring that gene therapies reach specific tissues while minimizing potential side effects, making the kits an indispensable tool in the research community.

According to Professor Leszek Lisowski, who heads the Translational Vectorology Research Unit at CMRI, the innovative barcoded AAV capsid kits are designed to facilitate parallel evaluations of numerous AAV variants. This unprecedented capability allows for swift identification of the most functional AAV variants based on their performance both at the DNA level, which proxies for cell entry, and at the RNA level, indicative of transgene expression.

Lisowski explains, "The barcoded AAV kits not only promise a reduction in costs and preclinical model usage but also serve to enhance the overall research efficiency in gene therapy development. With combined efforts from both CMRI and PackGene, researchers around the globe will gain unprecedented access to top-tier research tools, which is essential for speeding up the progress of AAV-based gene therapies."

Driving Innovation in Gene Therapy

PackGene's Chief Strategy Officer, Dr. Xin Swanson, emphasized the objective of making gene therapy more accessible and affordable. She stated, "By merging CMRI's cutting-edge technology with PackGene's extensive technical know-how in AAV vector design and production, we are enabling our clients to confidently advance from discovery to clinical applications. This partnership marks a significant leap forward in the realm of high-throughput AAV capsid screening and will greatly expedite the development of next-generation gene therapies."

The collaboration promises enhanced AAV capsids with improved traits, including increased tissue specificity and reduced off-target toxicity. The insights gained through these kits could be integral to developing safer and more effective gene therapies for a variety of conditions.

The Future of Gene Therapy

This licensing agreement underscores both companies' commitment to pushing the boundaries of what is achievable in gene therapy research. As global demand for effective therapeutic solutions rises, such collaborations are crucial in propelling innovation and making significant advances in medicine more accessible to patients in need. PackGene’s state-of-the-art π-alpha 293 AAV production platform is expected to deliver leading yields, supporting the scalability necessary for burgeoning clinical use, thus tying together their efforts to optimize both research and production capabilities.

With organizations like PackGene and CMRI at the forefront, the future of gene therapy looks promising, with expectations of accelerated breakthroughs that could transform the treatment landscape for various genetic diseases.

For more information about PackGene and its capabilities, visit www.packgene.com. For insights into CMRI's groundbreaking research, check out www.cmri.org.au.