Our approach has the potential to provide one-time treatments 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.
Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the body’s metabolic system. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of fats called sulfatides in the brain and other areas of the body, leading to loss of sensory, motor and cognitive function.
About Libmeldy™ (atidarsagene autotemcel) (EU, UK, Iceland, Liechtenstein and Norway)
Libmeldy (atidarsagene autotemcel) is an ex vivo autologous hematopoietic stem cell gene therapy approved by the European Medicines Agency (EMA) in 2020 and is currently the only approved treatment for MLD. Libmeldy 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.
Libmeldy 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 Libmeldy 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.
OTL-203 (MPS-I)
About MPS-I
Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to break down sugar molecules called glycosaminoglycans (also known as GAGs). The accumulation of GAGs across multiple organ systems results in symptoms including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications.
OTL-203 is an ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare paediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome.
OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority.
OTL-201 (MPS-IIIA)
About MPS-IIIA
Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare, life-threatening neurometabolic disease characterized by intellectual disability and loss of motor function. It is caused by a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, resulting in the build-up of sugar molecules called mucopolysaccharides in the brain and other tissues. There are currently no approved treatment options for MPS-IIIA.
OTL-201 is an ex vivo autologous gene therapy being developed for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s cells. OTL-201 has received rare paediatric disease designation from the FDA and is currently being evaluated in an ongoing proof-of-concept clinical trial.
OTL-201 is an investigational therapy and has not been approved by any regulatory agency or health authority.
Several additional research and preclinical programs under development.
Libmeldy® (OTL-200) has 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.
About OTL-200/ Libmeldy® (EU, UK, Iceland, Liechtenstein and Norway)
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. Libmeldy is known as OTL-200 in the U.S., where it is currently under Priority Review by the 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.
