Adeno-associated virus, illustration
Credit: Kateryna Kon/Science Photo Library/Getty Images

PerkinElmer’s SIRION Biotech business announced today that it will work with biomedical research center of excellence, the Centre for Genomic Regulation (CRG), to jointly develop new adeno-associated virus (AAV) vectors for type 1 and type 2 diabetes gene therapy in the pancreas.

The collaboration will combine SIRION’s viral vector-based gene delivery technologies and expertise in AAV development and production with CRG’s expertise in genetic regulatory mechanisms. According to SIRION and CRG, the goal of the codevelopment work is aims to increase the precision, safety, and efficacy of future AAV-based gene therapies for diabetes by creating new AAV vectors that target specific pancreatic cell types and contain payloads that express therapeutic genes under control of cell-specific regulatory elements.

“Our hope is that our joint efforts will not only facilitate better gene therapy options for type 1 and type 2 diabetes but also bring the life science industry closer to creating more successful and specialized gene therapies for other diseases such as neuronal disorders,” said Dr. Christian Thirion, founder and managing director of SIRION in a press release.

Munich, Germany-based SIRION was acquired by PerkinElmer in late 2021, as the company looked to expand its footprint in the development of viral vectors used to deliver cell and gene therapies. Prahlad Singh, president and CEO of PerkinElmer, said at the time of acquisition: “Seventy percent of gene therapy trials today leverage viral vector approaches and we expect this to remain strong going forward given the demand for targeted, high-payload delivery in treating diseases like cancer. By combining SIRION’s innovative ‘payload’ transport technology with our existing Horizon genetic material editing tools and phenotypic research solutions, we will be able to support organizations’ cell and gene therapy workflows as they look to streamline and accelerate their efforts.”

SIRION’s key technologies include transduction enhancers; what it calls BAC a technology to build viral vectors from scratch using bacterial artificial chromosomes; and RNAiONE a gene silencing technology for target validation and drug discovery aimed at the pharma and biotech sector.

The CRG is a non-profit biomedical research center based in Barcelona, Spain established in 2000 funded by the Catalan Government. The CRG, part of a life science researcher umbrella organization The Barcelona Institute of Science and Technology (BIST) which also includes the Institute of Chemical Research of Catalonia (ICIQ), the Catalan Institute of Nanoscience and Nanotechnology (ICN2), the Institute of Photonic Sciences (ICFO), the High Energy Physics Institute (IFAE), the Institute for Research in Biomedicine (IRB Barcelona), and the Institute for Bioengineering of Catalonia (IBEC).

BIST’s goals are:

  • To contribute towards improving conditions for carrying out high-quality research at the research centers and to increase its international visibility.
  • To offer coordinated postgraduate programs of excellence with international impact.
  • To build a critical mass to multiply the capacity of the research centers in different fields, such as knowledge transfer, scientific and technological platforms and talent attraction.
  • To cooperate with scientific institutions, companies, individuals and public or private entities to fulfill these aims.

The CRG counts to more than 400 interdisciplinary scientists focused on understanding the complexity of life, from the genome to the cell and the entire organism and has recently created a Medical Genomics Programme. For SIRION, collaborating with the CRG allows it to continue to expand its viral vector technology licensing portfolio.

“In this joint project we will leverage our development platform for regulatory elements and harness our research results on gene networks from recent years,” said Prof. Jorge Ferrer, and expert in regulatory genomics and diabetes and project leader. “Teaming with SIRION and translating our findings into real products and applications underscores the importance of having state-of-the art technologies and capabilities that can support others in their own endeavors.”

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