At Our Core

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 these articles, members of our community share their perspectives on our work, our mission, and how we can achieve that transformative future. Click here for a complete list of company press releases.
Science & Innovation

Crossing the Blood-Brain Barrier to Treat Disease

Crossing the blood-brain barrier is a vital prerequisite to solving some of the world’s most devastating and difficult to treat diseases. Frontotemporal dementia and Alzheimer’s ar...


Landmark Studies Shine A Light on the Need to Modernize the US Newborn Screening System

Recent publications in JAMA Network Open, Expert Evaluation of Strategies to Modernize Newborn Screening in the United States call for urgent change to ensure the U.S. newborn scre...


Rightsizing the U.S. Newborn Screening System

In the first 24 to 48 hours of a baby’s life, a small blood sample is taken to detect serious genetic conditions, some of them deadly, that can be treated if diagnosed early.1 Than...

Science & Innovation

Going Viral: Understanding Lentiviral Vectors in Gene Therapy

Gene therapy is designed to insert functional copies of a faulty gene that is not working properly. Genes are the genetic blueprints for proteins and sometimes a mutation can resul...

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Science & Innovation

Trends and Issues Shaping the Gene Therapy Industry

The gene therapy field is on the cusp of something special in realizing the transformative potential of gene therapies for patients and their families. After a year as president an...

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Our Orchard

The Importance of Orchard’s Core Values for Our Bold Vision

As we pass the one-year mark of the beginning of the COVID-19 pandemic, the collective shift to at-home work, and my transition to the role of chief executive officer at Orchard, I...

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