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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.
3 days 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.
3 days 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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GENE TECH TIMES
The Future Has Arrived
Gene and Cell Therapy Clinical Trials to Notice in 2025
Several clinical trials in 2025 are pushing the boundaries of gene and cell therapy, aiming to address a range of complex diseases. Below is an overview of five major trials that could provide significant breakthroughs for patients suffering from genetic disorders, cancer, and autoimmune conditions. Intellia Therapeutics’ Gene-Editing Therapy for Hereditary Angioedema Hereditary angioedema (HAE) is a rare genetic disorder that causes episodes of severe swelling, often in the
Jan 31, 2025
Afami-cel: First T-Cell Therapy Approved for Solid Tumors
On August 2nd, 2024, the FDA granted accelerated approval for afami-cel -- the first gene-engineered T-cell Therapy for advanced synovial sarcoma and the first T-cell therapy ever to get approved for solid tumors. Developed by Adaptimmune, afami-cel uses the patient's immune system to fight cancer, a mechanism similar to other existing T-cell therapies on the market. In this treatment, the patient's T cells are genetically modified to recognize and attack sarcoma cells that e
Aug 28, 2024
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