We’re thrilled to share that the U.S. Food and Drug Administration has approved our gene therapy for a genetic bleeding disorder. Developing the one-time therapy and bringing it over the finish line, first in Europe and now the United States, has been a global effort. Our teams have made unprecedented scientific, medical, manufacturing and regulatory […]
It should not come as a surprise to anyone that art and science are siblings. Faced with a lump of clay, a potter has a world of infinite possibility at their beck and call. Every revolution of the potter’s wheel is the beginning of a journey, of an experiment. With much trial and error, with […]
The contents of this post were first presented at a poster talk during the annual scientific meeting of the American College of Allergy, Asthma & Immunology’s November 2022 symposium. Hereditary angioedema (HAE) is a genetic disorder that results in unpredictable episodes of severe swelling and can be life threatening. The swelling can affect different parts […]
BioMarin Scientist Spotlight: Drs. Sylvia Fong & Soumi Gupta Discuss the Significance of AAV Therapies
By interrogating the human genome, we’ve designed therapies that address the root cause of genetic disease. That’s a guiding principle for how we approach our research at BioMarin, and nowhere is that more true than in our work researching gene therapy. As part of our commitment to expanding our technical expertise, we’re looking into many […]
Forty years ago, the idea of gene therapy seemed like science fiction. Deliver a genetic blueprint to human cells using modified viruses or other molecular tools?
For a scientist, access to cutting-edge technologies, rare resources, and expert support can turn a new idea into a groundbreaking discovery.