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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.
19 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.
19 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


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Scientists Cure Type 1 Diabetes in Mice Using a Gentle Immune System “Reset”
Stanford Medicine researchers cured Type 1 diabetes in mice using a gentle immune-reset approach that combines donor blood stem cells and pancreatic islet transplants. The method prevented autoimmunity, required no immunosuppressive drugs, and may pave the way for future treatments for diabetes and other autoimmune diseases.
Dec 2, 2025


Vertex’s Stem Cell Therapy: A Bold Leap Toward a Type I Diabetes Cure
Vertex Pharmaceuticals has announced a pivotal milestone for its VX-880 therapy, a stem-cell-derived islet cell treatment to restore insulin production in people with type 1 diabetes (T1D). The treatment, which previously underwent a Phase 1/2 clinical trial, is now entering a Phase 1/2/3 trial, making it the first scalable T1D cure to reach this advanced stage. This combined trial streamlines the research process, integrating safety, efficacy, and confirmatory studies into a
Jan 13, 2025


CRISPR Gene-Editing Therapies Make Strides, but Cost and Accessibility Remain Key Challenges
As CRISPR-based therapies edge closer to becoming mainstream, groundbreaking treatments for blood disorders and cancer are making their mark. One of the most exciting developments is Casgevy, a gene-editing therapy that has shown remarkable success in treating sickle-cell disease and β-thalassaemia. Approved in both the UK and US, Casgevy works by reactivating fetal hemoglobin, offering patients a chance at long-term relief with fewer blood transfusions and hospital visits. W
Dec 19, 2024
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