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, including neurometabolic disorders, primary immune deficiencies and blood disorders.

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.

Preclinical
Clinical proof of concept
Registrational trial
Commercialization
Designations
Neurometabolic Disorders
RPD
BKT
PRIME
RMAT
OTL-200 MLD

OTL-200 (MLD)

About MLD

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. Currently, there are no approved treatments for MLD.

For more information about MLD, click here.

About OTL-200

OTL-200 is an ex vivo autologous gene therapy being investigated for the treatment of MLD. It uses a modified virus to insert a functional copy of the ARSA gene into a patient’s cells. OTL-200 was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-200 has received rare pediatric disease designation from the FDA. Orchard is currently conducting clinical trials to investigate the safety and efficacy of OTL-200 for the treatment of MLD.

OTL-200 is an investigational therapy and has not been approved by any regulatory agency or health authority.

OTL-203 MPS-I

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. 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

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.

OTL-201 is an investigational therapy and has not been approved by any regulatory agency or health authority.

OTL-202 MPS-IIIB

OTL-202 (MPS-IIIB)

About MPS-IIIB

Mucopolysaccharidosis type IIIB (MPS-IIIB, also known as Sanfilippo syndrome type B) is a rare and life-threatening neurometabolic disease. Patients with MPS-IIIB are born with a mutation in the alpha-N-acetylglucosaminidase (NAGLU) gene, causing sugar molecules called mucopolysaccharides to build up in the brain and other tissues, leading to intellectual disability and loss of motor function. Currently, there are no approved treatment options for MPS-IIIB.

For more information about MPS-IIIB, click here.

About OTL-202

OTL-202 is an ex vivo autologous gene therapy being developed for the treatment of MPS-IIIB. It uses a modified virus to insert a functional copy of the NAGLU gene into a patient’s cells.

OTL-202 is an investigational therapy and has not been approved by any regulatory agency or health authority.

Primary Immune Deficiencies
Strimvelis®

Strimvelis®

Strimvelis® is a gammaretroviral vector-based gene therapy approved by the European Medicines Agency in 2016. It was the first ex vivo autologous gene therapy approved by the EMA. Strimvelis® has not been approved by the FDA.

For more information about Strimvelis®, visit the EMA website.

OTL-101 ADA-SCID

OTL-101 (ADA-SCID)

About ADA-SCID

Adenosine deaminase severe combined immunodeficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the adenosine deaminase (ADA) enzyme that result in a lack of, or minimal, immune system development. Children born with ADA-SCID experience recurrent severe infections and overall failure to thrive.

For more information about ADA-SCID, click here.

About OTL-101

OTL-101 is an ex vivo autologous gene therapy being investigated for the treatment of ADA-SCID. It uses a modified virus to insert a functional copy of the ADA gene into a patient’s cells. OTL-101 has received both breakthrough therapy designation and rare pediatric disease designation from the FDA. Orchard is conducting ongoing clinical trials of OTL-101 for the treatment of ADA-SCID.

OTL-101 is an investigational therapy and has not been approved by any regulatory agency or health authority.

OTL-103 WAS

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-102 X-CGD

OTL-102 (X-CGD)

About X-CGD

X-linked chronic granulomatous disease (X-CGD) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the cytochrome B-245 beta chain (CYBB) gene. Because of this genetic defect, the neutrophils, or white blood cells, of patients with X-CGD are unable to kill bacteria and fungi, leading to repeated chronic infections.

For more information about X-CGD, click here.

About OTL-102

OTL-102 is an ex vivo autologous gene therapy being investigated for the treatment of X-CGD. It uses a modified virus to insert a working copy of the CYBB gene into a patient’s cells. Orchard is planning to initiate a clinical trial to evaluate OTL-102 for the treatment of X-CGD.

OTL-102 is an investigational therapy and has not been approved by any regulatory agency or health authority.

Blood Disorders
OTL-300 TDT

OTL-300 (TDT)

About beta thalassemia

Beta thalassemia is a genetic blood disorder caused by mutations in the beta-globin gene that cause ineffective red blood cell production. Patients with the most severe mutations are not able to produce hemoglobin, and rely on frequent red blood cell transfusions in order to survive. Because of these transfusions, patients must also receive treatments to remove excess iron from the body.

For more information about beta thalassemia, click here.

About OTL-300

OTL-300 is an ex vivo autologous gene therapy being investigated for the treatment of transfusion-dependent beta thalassemia. It uses a modified virus to insert a functional copy of the beta-globin gene into a patient’s cells. OTL-300 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-300 has received Priority Medicines (PRIME) designation from the EMA. Orchard in conducting a clinical proof-of-concept trial to evaluate OTL-300 for the treatment of transfusion-dependent beta thalassemia, and is currently conducting long-term safety and efficacy follow-up.

OTL-300 is an investigational therapy and has not been approved by any regulatory agency or health authority.

* Several additional research and preclinical programs under development

Strimvelis® has been approved by the European Medicines Agency. It has not been approved by the U.S. Food and Drug Administration. The other therapies in our pipeline are currently in clinical trials and have not been approved by any regulatory agency or health authority.

RPD Rare Pediatric Disease Designation; eligible for a Priority Review Voucher

BKT Breakthrough Therapy Designation

PRIME Priority Medicine Designation

RMAT Regenerative Medicine Advanced Therapy Designation

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