Amsterdam, The Netherlands, June 4, 2024 / Biotech Newswire
Annogen, a company with expertise in bespoke promoter design and gene expression engineering, today announced a research collaboration with Orchard Therapeutics, a global gene therapy leader, for the identification of immune cell-specific human promoters for use in certain pre-clinical hematopoietic stem cell (HSC) gene therapy programs.
Using its proprietary Survey of Regulatory Elements (SuRE™) technology, Annogen exhaustively interrogates the entire human regulatory genome in vitro and in vivo to identify DNA elements that are active in different cell types and conditions. Subsequently, combinations of promoters and enhancers are screened to identify the best hybrid promoter to drive gene expression in a cell- or disease-specific manner. The SuRE™ technology is unique among other promoter identification technologies in that it does not deliver (AI-based) predictions that need to be verified by the customer but instead, the SuRE™ technology delivers the power of millions of wet-lab measurements in relevant cells and in vivo models, providing validated elements that support more effective and safer therapies.
“Building on the safe and effective human promoters we have for our existing programs, we are excited to further establish a leadership position in the field by supporting research for additional CNS-focused cellular targets,” said Frank Thomas, president and chief operating officer of Orchard Therapeutics. “This work is critical as we apply our clinically-validated HSC gene therapy platform to more prevalent diseases. We look forward to utilizing Annogen’s SuRE technology to potentially advance certain pre-clinical programs.”
“Annogen has gained substantial experience identifying and developing promoters for in vivo gene therapy,” Joris van Arensbergen CEO of Annogen adds. “We are very pleased to work with a company in this field that has managed to bring autologous cell therapy through to commercialization in the U.S. and Europe. We are proud to be part of their program and to contribute to the safety and efficacy of certain pre-clinical therapies.”
About Orchard
Orchard Therapeutics, a Kyowa Kirin company, is focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Its team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.
