top of page
LATEST NEWS
Estuary Launches With $103 Million to Bring Gene Therapy to Polycystic Kidney Disease
Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.
21 hours ago4 min read
Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.
21 hours ago4 min read
Optogenetic Gene Therapy Gives Some Blind Patients Back a Sense of Light
In the NEJM-published PIONEER trial, an algae-derived light-sensing gene plus amber-light goggles improved light sensitivity in 6 of 10 people with late-stage retinitis pigmentosa, and 4 of 8 got better at finding objects. Here is how optogenetics works.
Oct 115 min read
LEARN MORE
LEARN MORE
GENE TECH TIMES
The Future Has Arrived
FDA Approves BEQVEZ: A Breakthrough One-Time Gene Therapy for Adults with Hemophilia B
Gene therapy is transforming the landscape of treatment options, and the recent FDA approval of BEQVEZ marks a significant milestone for individuals living with hemophilia B. This innovative one-time gene therapy replaces the need for regular infusions of clotting factor, offering new hope for patients suffering from this genetic disorder. Understanding Hemophilia B Hemophilia B is a genetic condition caused by mutations in the human factor IX gene, leading to a deficiency i
May 1, 2024
bottom of page