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.
Lenmeldy™ / Libmeldy® (atidarsagene autotemcel), formerly known as OTL-200
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 Lenmeldy™ / Libmeldy® (atidarsagene autotemcel)
Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the U.S. for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
For additional details about Lenmeldy, please refer to the full Prescribing Information.
In Europe, Lenmeldy is known as Libmeldy®, where it has been approved by the European Commission (EC), UK Medicines and Healthcare products Regulatory Agency (MHRA), and Swiss Agency for Therapeutic Products (Swissmedic). For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
The program was originated by and developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
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.
Several additional research programs under development.
**Lenmeldy™ is approved in the U.S. for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Libmeldy® is approved in the European Union, UK, Iceland, Switzerland, Liechtenstein and Norway.
***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 Lenmeldy™ / Libmeldy® (atidarsagene autotemcel), formerly known as OTL-200
Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the U.S. for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
For additional details about Lenmeldy, please refer to the full Prescribing Information.
In Europe, Lenmeldy is known as Libmeldy®, where it has been approved by the European Commission (EC), UK Medicines and Healthcare products Regulatory Agency (MHRA), and Swiss Agency for Therapeutic Products (Swissmedic). For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.
The program was originated by and developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.
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