Orchard Therapeutics Global Website

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.

Learn more about MLD

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

Meet the Price family

Amy Price, a mother of eight, is a vocal advocate for expanding newborn screening for the rare diseases. She knows firsthand how critical early diagnosis can be.

Read More

Meet the Elsons

Connie was bright, energetic and active. She enjoyed reading books, helping her parents make shopping lists, climbing trees and going down the slides at the playground.

Read More

Living with Metachromatic Leukodystrophy (MLD)

Discover the challenging journey of Nala’s family as they grapple with the devastating diagnosis of metachromatic leukodystrophy (MLD), and the lack of treatment options due to the advanced stage of Nala’s condition.


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.