At Orchard, we’re committed to working closely with physicians and others in the healthcare community to advance research into ex vivo autologous gene therapy and improve outcomes for people affected by rare inherited diseases.
Healthcare providers requesting information on Orchard’s approved or pipeline programs may contact firstname.lastname@example.org.
Strimvelis (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence) is a gammaretroviral vector-based gene therapy approved by the European Medicines Agency (EMA) in 2016. It was the first ex vivo autologous gene therapy approved by the EMA. Strimvelis has not been approved by the U.S. Food and Drug Administration (FDA) or any other Health Authority.
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA) matched related stem cell donor is available. Strimvelis is intended solely for autologous use and must be given in a specialized hospital by a doctor who is experienced in treating patients with ADA-SCID and in using this type of medicine.
Serious adverse reactions include autoimmunity (e.g., autoimmune haemolytic anaemia, autoimmune aplastic anaemia, autoimmune hepatitis, autoimmune thrombocytopenia and Guillain-Barré syndrome). The most commonly reported adverse reaction was pyrexia.
For more information about Strimvelis, please see the EU Summary of Product Characteristics available on the EMA website.
Independent Medical Education
Orchard supports independent medical education on our therapeutic areas of interest and on emerging developments in gene therapy. For more information and to apply, please contact email@example.com.
Orchard supports investigator-initiated research on the diagnosis, natural history and other topics relevant to our indications. For more information and to apply, please contact firstname.lastname@example.org.
Orchard and our research partners are conducting clinical trials of investigational gene therapies for a number of rare, inherited diseases, including primary immune deficiencies, diseases of the brain and blood disorders. For more information about these trials, explore our pipeline or contact a member of Orchard medical information at email@example.com.