Pipeline

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.

For more information about clinical trials of Orchard’s investigational therapies, please visit www.clinicaltrials.gov or contact us at info@orchard-tx.com.

Explore our pipeline below.

Neurometabolic/Neurodegenerative Disorders

Preclinical

Clinical proof of concept

Registrational trial

Commercialization

OTL-203 MPS-I

Designation: RPD; PRIME

Clinical Trial Identifier: NCT03488394

OTL-201 MPS-IIIA

Designation: RPD

Clinical Trial Identifier: NCT04201405

OTL-204 FTD

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.

For more information about MPS-I, click here.

About OTL-203

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.

For more information about MPS-IIIA, click here.

About OTL-201

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.

Immunological Disorders

Preclinical

Clinical proof of concept

Registrational trial

Commercialization

OTL-103 WAS

Designation: RPD; RMAT

Clinical Trial Identifier: NCT03837483, NCT01515462, EudraCT 2009-017346-32

OTL-104 CROHN’S

OTL-103 (WAS)

About WAS

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.

For more information about WAS, click here.

About OTL-103

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.

OTL-105 (HAE)

Hereditary angioedema

Program partnered with Pharming Group N.V

IE-002-2200005, June 2023