- Partnership reinforces PackGene’s leadership in AAV solutions and rapid vector serotype screening
- Customizable AAV Vector Screening Kit developed by CMRI and produced by PackGene, featuring unique barcoded variants
- Access to proprietary novel capsids from CMRI and PackGene’s π-Icosa platform accelerates gene therapy R&D
PackGene Biotech, a global contract research organization (CRO) and contract development and manufacturing organization (CDMO) leader in adeno-associated viral (AAV) vector technologies, and Children’s Medical Research Institute (CMRI), a world-renowned institution in gene therapy innovation, announced today a strategic licensing agreement and collaboration partnership around AAV Capsid Screening Kits. The kits offer customers the ability to rapidly perform high-throughput in vitro, ex vivo, and in vivo screening to identify optimal AAV Capsids that are suitable for tissue-specific delivery in gene therapy research and therapeutic product development needs. Simultaneously, customers will gain access to proprietary novel AAV capsids developed by CMRI or PackGene with improved utilities such as increased tissue specificity, decreased off-target toxicity, lower immunogenicity, and higher productivity.
The project is led by Professor Leszek Lisowski, Head of the Translational Vectorology Research Unit at CMRI. “CMRI has pioneered the work of establishing barcoded AAV capsid kits, which allow for NGS-based parallel evaluation of large numbers of AAVs for their performance at both DNA (proxy for cell entry) and RNA (proxy for transgene expression) levels to allow fast, precise and high-throughput identification of the most functional AAV variants while reducing cost and use of preclinical models. Coupled with innovative vector engineering research being conducted at both CMRI and PackGene, this license agreement will allow researchers world-wide to have expanded access to best-in-class research tools, further accelerating the development of AAV-based gene therapies. We are very excited about this partnership,” said Prof. Lisowski, “and we look forward to working with PackGene to empower gene therapy research and help gene therapy to reach its therapeutic potential through innovative technologies.”
“At PackGene, our mission is to make gene therapy affordable,” said Dr. Xin Swanson, Chief Strategy Officer of PackGene. “This deal combined CMRI’s innovative technology with PackGene’s deep technical expertise and proven track records in AAV vector design, research material production and cGMP manufacturing. Our customers will be able to de-risk AAV development with confidence from discovery to the clinic. This partnership is a significant advancement in high-throughput AAV capsid screening, which will serve to accelerate the development of next-generation gene therapies so that patients can access life-saving treatments sooner.”
