Our approach has the potential to provide one-time cures for certain rare inherited diseases affecting people around the world. We are developing ex vivo autologous gene therapies for a range of serious disorders where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.
Explore our pipeline below.
Several additional research and preclinical programs under development. Libmeldy™ (OTL-200) and Strimvelis® have been approved by the European Medicines Agency and have not been approved by the U.S. Food and Drug Administration or any other health authority. In the U.S., OTL-200 is an investigational therapy. All other therapies in our pipeline are investigational and have not been approved by any regulatory agency or health authority.
OTL-200 (atidarsagene autotemcel), referred to as Libmeldy in the European Union, is an ex vivo autologous haematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020 and is currently the only approved treatment for MLD. OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA).
In the EU, Libmeldy is indicated for the treatment of patients with metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.
OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA). In addition to the risks associated with the gene therapy, treatment with OTL-200 is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.
For more information about Libmeldy, please see the EU Summary of Product Characteristics available on the EMA website.
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.