Amsterdam, The Netherlands – May 12, 2025
Orchard Therapeutics, a Kyowa Kirin company focused on innovative gene therapies, and Annogen B.V., experts in bespoke promoter design and gene expression engineering, today announced the successful identification of novel human promoters for use in Orchard’s pre-clinical hematopoietic stem cell (HSC) gene therapy programs. These promoters give Orchard a highly innovative toolkit towards effectively fighting the devastation caused by severe genetic diseases.
Building on their initial collaboration announced in June 2024, the companies have leveraged Annogen’s proprietary Survey of Regulatory Elements (SuRE™) platform to generate promoters based on sequences from the human genome, demonstrating highly cell-type specific and constitutive expression in relevant immune cells. This achievement marks a significant step forward in optimizing the targeted delivery and efficacy of Orchard’s next-generation HSC-based treatments for serious genetic diseases.
The collaboration utilized Annogen’s unique SuRE™ platform, which can perform comprehensive in vivo and in vitro interrogation of human endogenous cis-regulatory elements (CREs) as well as (machine learning based) de novo designs for CREs. The successful identification and subsequent in vitro validation of these promoters in primary human cells provide Orchard Therapeutics with a valuable toolkit to precisely control gene expression within their HSC-derived therapies. This targeted approach has the potential to enhance treatment outcomes by ensuring the therapeutic genes are specifically and effectively expressed in the intended cell type, thereby minimizing off-target effects.
“We are delighted with the progress achieved through our collaboration with Annogen,” said Fulvio Mavilio, the chief scientific officer at Orchard Therapeutics. “The successful identification and the preliminary testing of these novel promoters are crucial for advancing our pre-clinical HSC gene therapy programs. This milestone underscores our commitment to pushing the boundaries of gene therapy and developing more precise and effective treatments for patients with severe genetic diseases.”
Dr. Joris van Arensbergen, Annogen’s founding CEO added, “This successful outcome is a testament to the power of our SuRE™ platform and a demonstration of the synergy achieved between Annogen and partners like Orchard. We are proud to contribute to the advancement of Orchard’s innovative HSC therapy pipeline and look forward to seeing these promoters play a key role in the development of potentially life-changing treatments for patients in need.”
