Inside The Orphan Drug Revolution The Promise of Patient-Centered Biotechnology

by Geraghty, James A.

• ISBN13: 9781621825005
• ISBN10: 1621825000
• Format: Paperback
• Copyright: 2022-11-30
• Publisher: Cold Spring Harbor Laboratory Press

Table of Contents

Foreword: How It All Started, 40 Years Ago
Introduction: Was It Really a Revolution?

Part One: THE VANGUARD
1. Pre-Revolutionary Stirrings
The deep roots of the orphan drug revolution
2. Leading in the Revolution
The historic importance of hemophilia therapies
3. Somehow a Miracle Happens
Gaucher disease and the emergence of Genzyme

Part Two: HARD-WON SUCCESSES
4. Patients Are Everywhere
Product launches in Europe, and well beyond
5. Broader Fronts Emerge
Alphabet soup: CF, PNH, SMA, HAE, DMD, TTR
6. Making Medicines in Milk
A long, unlikely path to two small orphan drugs
7. The End of the Beginning
Genzyme tries to do too much, and loses it all

Part Three: A SECOND WAVE
8. Starting from Scratch
After a vacuum, "nothing is impossible" at TRV
9. To Boldly Go: Into the Brain
The challenges of CNS diseases at Voyager
10. Learning to "Regulate" DNA
Going well beyond "junk DNA" at Fulcrum
11. Into "Ultra-Orphan" Diseases
Taking on ultra-rare conditions at Orchard
12. To China, and Hopefully Back
Pursuing a 20-year mission at CANbridge
13. Where Patients Are Waiting
New start-ups tackle diseases yet unserved
14. Some Brief Lessons Learned
Observations for orphan drug entrepreneurs

Part Four: THE CHALLENGES AHEAD
15. The Challenge of Affordability
Ensuring availability for patients and society
16. Toward Long-Term Sustainability
With a revolution comes new responsibilities

Epilogue: Heading to 50 Year
What might the next ten years bring?

Acknowledgments
Sources
Index
Photo Section follows page 106