In a significant move for gene therapy research, Annogen has announced a collaboration with Orchard Therapeutics to identify immune cell-specific human promoters for use in certain pre-clinical hematopoietic stem cell (HSC) gene therapy programmes. This partnership aims to advance the development of more targeted and potentially safer gene therapies for central nervous system (CNS) disorders.

Innovative technology at the heart of the collaboration

Annogen’s proprietary Survey of Regulatory Elements (SuRE™) technology forms the cornerstone of this research collaboration. This cutting-edge approach allows for a comprehensive examination of the entire human regulatory genome, both in vitro and in vivo, to identify DNA elements active in specific cell types and conditions. The SuRE technology stands out in the field of promoter identification due to its ability to provide validated elements based on millions of wet-lab measurements, rather than relying on AI-based predictions that require subsequent verification.

The process involves screening combinations of promoters and enhancers to identify optimal hybrid promoters capable of driving gene expression in a cell- or disease-specific manner. This approach has the potential to significantly enhance the efficacy and safety profiles of gene therapies by allowing for more precise targeting of therapeutic genes to specific cell types within the CNS.

Expanding the horizons of HSC gene therapy

Orchard Therapeutics, a global leader in gene therapy with a track record of bringing autologous cell therapies to market in both the United States and Europe, sees this collaboration as an opportunity to further solidify its position in the field. The company aims to leverage Annogen’s expertise to potentially advance certain pre-clinical programmes, particularly those focused on more prevalent CNS disorders.

Frank Thomas, president and chief operating officer of Orchard Therapeutics, commented on the collaboration: “Building on the safe and effective human promoters we have for our existing programmes, we are excited to further establish a leadership position in the field by supporting research for additional CNS-focused cellular targets. 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.”

Implications for future gene therapies

The partnership between Annogen and Orchard Therapeutics represents a significant step forward in the development of more targeted gene therapies. By focusing on CNS-specific promoters, the collaboration aims to enhance the precision of gene expression in therapeutic applications, potentially leading to improved efficacy and reduced off-target effects.

Joris van Arensbergen, CEO of Annogen, expressed enthusiasm about the collaboration, saying: “Annogen has gained substantial experience identifying and developing promoters for in vivo gene therapy. We are very pleased to work with a company in this field that has managed to bring autologous cell therapy through to commercialisation in the U.S. and Europe. We are proud to be part of their programme and to contribute to the safety and efficacy of certain pre-clinical therapies.”

The specific terms of the collaboration have not been disclosed.