re: Generation

Category: Science

AllSCIENCECULTURESTORIES
Toward a new paradigm in treatment for hemophilia A
A CONVERSATION WITH OUR CHIEF MEDICAL OFFICER DOUG KERR, MD, PHD, MBA
In March we recognize Bleeding Disorders Awareness Month and the communities affected by these conditions, including hemophilia. As we continue to advance our efforts toward a durable, redosable gene therapy for hemophilia A, we reflect on how we hope to serve these communities.
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Our quality culture enables pioneering science
by SONIA RAZZETTI, PhD, VICE PRESIDENT OF QUALITY
Quality is a mindset that enables our science at Generation Bio. It’s all about rigor. Responsibility. Integrity. And a commitment not just to do great work but to document that science so it can be understood, replicated, and built upon for years to come.
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Special Delivery: Engineering a non-viral delivery platform for a new class of gene therapy
by MATTHEW STANTON, PhD, CHIEF SCIENTIFIC OFFICER
I stared at the display of cell culture images on the monitor. Every one of them was dark. Sometimes a breakthrough comes from what is not there.
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Manufacturing a global revolution in gene therapy
by MARK ANGELINO, PhD, FOUNDER & CHIEF OPERATING OFFICER and GEOFF MCDONOUGH, MD, PRESIDENT & CHIEF EXECUTIVE OFFICER
In the early 1940s, Pfizer executive John Smith wrote of the revolutionary drug penicillin: “[T]he yields are low, the isolation is difficult, the extraction is murder, the purification invites disaster, and the assay is unsatisfactory.”1
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Bringing the light of gene therapy to more patients
Thomas Edison’s first lightbulb in 1879 sparked a revolution. Using electricity that was passed through a platinum filament, his glass bulb required less power than the existing lamps of the day. But it also lasted only a few hours before burning out.
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Genetic diseases steal too many kids' futures. It doesn’t have to be this way.
by GEOFF MCDONOUGH, MD, President & Chief Executive Officer and DOUG KERR, MD, PhD, MBA, Chief Development Officer
This piece was originally published on WBUR’s Cognoscenti. I want to live in a world where children have never heard of hemophilia or cystic fibrosis, because we have all but eradicated those diseases.
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Right from the beginning: returning to the origins of the orphan drug movement
by GEOFF MCDONOUGH, MD, PRESIDENT & CHIEF EXECUTIVE OFFICER
This article was originally published as part of the From The Trenches feature of LifeSciVC. Our industry stands at the beginning of the most significant opportunity to impact genetic and metabolic disease we have ever encountered –
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Imagine a world without genetic disease.

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