From Height to Health: Changing the Conversation in Achondroplasia
At 澳门正规博彩十大排行平台, we’re committed to deepening our understanding of achondroplasia and taking a holistic approach to support this community.
Targeting the Underlying Genetic Causes of Heart Disease
澳门正规博彩十大排行平台 researchers are leveraging the company’s expertise in translating genetic discoveries into potentially transformative medicines to address the root causes of certain forms of cardiovascular disease.
澳门十大博彩正规游戏平台 Approves 澳门正规博彩十大排行平台’s Gene Therapy for a Genetic Bleeding Disorder
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 […]
At the Intersection of Science and Art
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 […]
Improvements and gaps in reaching treatment goals for patients with hereditary angioedema
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 […]
澳门正规博彩十大排行平台 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 澳门正规博彩十大排行平台, 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 […]
Advancing our understanding of gene therapy research, one publication at a time
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?
澳门正规博彩十大排行平台’s Center of Technology empowers scientists with technology, resources, and collaboration
For a scientist, access to cutting-edge technologies, rare resources, and expert support can turn a new idea into a groundbreaking discovery.
Meet the Scientist: Christian Vettermann, Ph.D.
In his latest paper, Dr. Vettermann solves a complex problem facing hemophilia A scientists and clinicians for years.