ORCHARD THERAPEUTICS PLC.

Company Overview

Orchard is a biotech firm specializing in in vivo autologous hematopoietic stem cell (HSC) gene therapies. Its focus lies in rare genetic disorders, encompassing neurometabolic disorders, primary immune deficiencies, and blood disorders such as beta thalassemia. Its portfolio consists of two commercially available products approved in Europe, along with seven lentiviral-based product candidates undergoing clinical-stage development and other candidates in preclinical stages.

In January 2024, Orchard was acquired by Kyowa Kirin Co., Ltd., a Japan-based global specialty pharmaceutical company. Orchard becomes a fully owned Kyowa Kirin subsidiary.

Orchard is engaged in ex vivo autologous HSC gene therapy, particularly targeting severe neurometabolic diseases and initiating early research programs. Leading this effort is the OTL-200 program, which has received approval in the EU, the U.K., Iceland, Liechtenstein and Norway under the trade name Libmeldy. This approval extends to eligible patients afflicted with early-onset metachromatic leukodystrophy (MLD).

Financial Highlights (FY 2022)

Company's Business Segments

• Gene Therapy : The company provides hematopoietic stem cells for treating genetic and other severe diseases where the burden for patients is immense.