Inspiration and Impact

We aspire to end the devastation caused by genetic and other severe diseases through the curative potential of hematopoietic stem cell, or HSC, gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment. Orchard has a portfolio spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

We recognize and deeply value the impact that patient communities have on us through the inspirational stories and dedicated advocacy we see every day.

Meet the children and families who inspire us to imagine limitless possibilities.

Educational Brochures

What is Gene Therapy?

What is MLD?

What is MPS?

Learn more about our gene therapy approach >

Clinical Trials

Orchard and our research partners are conducting clinical trials of investigational gene therapies for a number of genetic and other severe diseases, including several neurometabolic disorders. For more information about these trials, explore our pipeline or contact us at

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