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.
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 pediatric 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 buildup 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 pediatric 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.
Wiskott Aldrich syndrome (WAS) is a life-threatening inherited immune disorder characterized by recurrent and severe infections, autoimmunity, eczema and severe bleeding episodes. It is caused by a mutation in the gene that produces the Wiskott Aldrich syndrome protein, which results in abnormal function of white blood cells and low platelets.
OTL-103 is an ex vivo autologous gene therapy being investigated for the treatment of WAS. It uses a modified virus to insert a working copy of the WAS gene into a patient’s cells. OTL-103 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. It has received rare pediatric disease designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Orchard is conducting ongoing clinical trials of OTL-103 for the treatment of WAS.
OTL-103 is an investigational therapy and has not been approved by any regulatory agency or health authority.
