Our Portfolio and Focus

Unlocking the curative potential of HSC gene therapy.

Our hematopoietic stem cell, or HSC, gene therapy approach harnesses a patient’s own blood stem cells which are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment. We are developing a diversified portfolio of HSC gene therapies for the treatment of genetic and other severe diseases where the burden is immense for patients, families and society 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 patient.advocacy@orchard-tx.com.

Explore our pipeline below.

Preclinical
Clinical proof of concept
Registrational trial
Commercialization
Designations
Neurometabolic/Neurodegenerative Disorders
RPD
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.

For more information about MLD, click here.

About OTL-200/ Libmeldy® (EU, UK, Iceland, Switzerland, Liechtenstein and Norway)

Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of metachromatic leukodystrophy (MLD) in patients 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 is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Switzerland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

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. 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

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

Several additional research and preclinical programs under development.

*New investments in this program are currently limited.

**Libmeldy® (OTL-200) is approved in the European Union, UK, Iceland, Switzerland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S. All other therapies in our pipeline are investigational and have not been approved by any regulatory agency or health authority.

***Program partnered with Pharming Group N.V.

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

RMAT Regenerative Medicine Advanced Therapy Designation

PRIME Priority Medicine Designation

About OTL-200/ Libmeldy® (EU, UK, Iceland, Switzerland, Liechtenstein and Norway)

Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of metachromatic leukodystrophy (MLD) in patients 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 is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Switzerland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

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