At the presentation of the financial results for the quarter ended 30 June 2021 and the upcoming milestones Orchard Therapeutics’ CEO, Bobby Gaspar, M.D., Ph.D., says:

“This past quarter Orchard has shown great progress against multiple core strategic objectives across the portfolio. In our earlier stage pipeline, we’re very excited for our new collaboration with Pharming exploring the potential of HSC gene therapy in HAE.”

On 1 July 2021 Orchard Therapeutics and Pharming Group N.V. announced a strategic collaboration to research, develop, manufacture and commercialize OTL-105, a newly disclosed investigational ex vivoautologous HSC gene therapy for the treatment of HAE. OTL-105 is designed to increase C1 esterase inhibitor (C1-INH) in HAE patient serum to prevent HAE attacks. In preclinical studies, to date, OTL-105 demonstrated high levels of SERPING1 gene expression via lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs.

Under the terms of the collaboration, Pharming has been granted worldwide rights to OTL-105 and will be responsible for clinical development, regulatory filings, and commercialization of the investigational gene therapy, including associated costs. Orchard will lead the completion of IND-enabling activities and oversee manufacturing of OTL-105 during pre-clinical and clinical development, which will be funded by Pharming.
(Source: Orchard Therapeutics)