Inspiration and Impact

Orchard is dedicated to empowering life and inspiring new possibilities for people affected by certain rare inherited diseases. Through our mission to develop one-time, potentially curative therapies for a range of devastating genetic conditions, we seek to have a lasting effect on children, families, caregivers and society. We also recognize and deeply value the impact that the rare disease community has on us through the inspirational stories and dedicated advocacy we see every day.

Meet the children and families who inspire us to grow beyond rare disease.


Read Orchard’s access policy

We are proud to partner with advocates and organizations that share our goal of bringing one-time, potentially curative gene therapies to people affected by rare, often-fatal genetic diseases around the world. Visit the websites of these groups for more information and to get involved in the fight against rare inherited disease.


Educational Brochures

What is Gene Therapy?

What is MLD?

What is MPS?

What is ADA-SCID?

What is WAS?

What is X-CGD?

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 rare, inherited diseases, including primary immune deficiencies, neurometabolic disorders and blood disorders. For more information about these trials, explore our pipeline or contact us at

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