Orphan: The Quest to Save Children with Rare Genetic Disorders

Orphan: The Quest to Save Children with Rare Genetic Disorders

By Philip R. Reilly, MD, JD
ISBN 978-1-621821-37-3 • 408 pages, illustrated (12 page insert of B&W images)

Philip R. Reilly, MD, JD

Philip R. Reilly, MD, JD, is a Venture Partner at Third Rock Ventures in Boston, Massachusetts. Trained in internal medicine and clinical genetics, he specializes in starting and developing companies to develop breakthrough therapies for orphan genetic diseases. Dr. Reilly has served as CEO and Chairman of Interleukin Genetics, Inc., was Executive Director of the Eunice Kennedy Shriver Center for Mental Retardation, and was on the faculty at Harvard Medical School and Brandeis University, among other positions. He is the author of more than 100 scientific articles and seven books.

The title Orphan is meant to convey two messages. The first is that there are a vast number of genetic disorders that burden only a few people, but for which we must find a way to harness our biomedical research engine to develop cures. The second is to emphasize what we all know—that nothing is more precious than our children, and we must not let a bad ticket in.

Hardcover, eBook, and Audiobook Now Available

CSHL Press

What people are saying about Orphan

"A physician, attorney, and biotech entrepreneur, Philip Reilly clearly illuminates rare genetic disorders, the struggle to understand and treat them, and the promise of the latest advances in genetic testing and therapy. Engaging and compassionate, Orphan brings the exciting advances in biomedical research to life. This excellent volume is enriched by the often heroic stories of parents and physician-scientists determined to find answers."

- David J. Skorton, MD, Secretary, The Smithsonian Institution

"A fascinating, engaging, and at times inspiring look at the past, present, and future of genetic medicine told through the stories of scientists, doctors, patients, and parents who were heroes in the fight against genetic disease. Thoroughly enjoyable."

- Hank Greely, Stanford Law School, an authority on public policy issues in genetics

"Reilly's Orphan provides a very interesting and readable history of the various approaches used in efforts to first understand and then treat children who suffer from rare genetic diseases. Research efforts to identify rare genetic changes that cause disease used to require decades and large families, but with the rapid advances in genome sequencing over the past 15 years, this can now be done in weeks. Orphan discusses how these new discoveries might be coupled with gene editing or cell therapy to offer new hope to families."

- J. Craig Venter, PhD, Chairman and CEO, J. Craig Venter Institute, and a pioneer in human genomics

"Written with clarity and from first hand experience by a pioneer who is helping to develop new breakthrough therapies for children with rare genetic diseases, this book takes you by the hand and pulls you into the world of these children and their heroic parents, many of whom helped to champion new treatments working with equally heroic scientists and physicians. The story is truly compelling."

- Donald Ingber, MD, PhD, Founding Director of the Wyss Institute for Biologically Inspired Engineering at Harvard University