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LATEST NEWS
FDA Proposes One Approval for Many Mutations in Personalized Gene Editing
A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.
3 days ago4 min read


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.
Oct 224 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.
Oct 224 min read


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GENE TECH TIMES
The Future Has Arrived


Enhance CAR T-Cell Therapy Efficacy and Reduce Toxicity by Using the Microbiome
Emerging research shows that the intestinal microbiome plays a vital role in the success of CAR T-cell therapy, influencing both efficacy and toxicity. Dr. Melody Smith’s studies reveal that certain antibiotics can disrupt the microbiome, reducing overall survival and increasing neurotoxicity risks. Optimizing gut health through diet or microbiome-targeted interventions may enhance CAR T-cell outcomes for cancer patients.
Feb 24, 2025


A Life Saved: Woman in Remission for 18 Years After Pioneering CAR T-Cell Therapy for Neuroblastoma
In an inspiring breakthrough for cancer treatment, a woman who underwent an experimental immunotherapy trial as a child has now been in remission for 18 years. Diagnosed with neuroblastoma, an aggressive form of nerve tissue cancer typically seen in children under 5, she is believed to be the longest-surviving patient treated with CAR T-cell therapy. This life-changing therapy, which uses genetically engineered white blood cells to target cancer, was part of a clinical trial
Feb 18, 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 27, 2024


Revolutionary Gene Therapy Targeting Galectin 1 Shows Promise in Reducing Liver Cancer Incidence in Animal Models
New Approach for Liver Cancer Treatment and Possible Prevention: Gene Therapy Targets Biomarker for Hepatocellular Carcinoma (HCC) Researchers at the UC Davis Comprehensive Cancer Center have unveiled a promising gene therapy approach that significantly reduces the incidence of hepatocellular carcinoma (HCC) in animal models, specifically mice. This innovative research targets a specific protein known as galectin 1 (Gal1), which has long been established as a critical biomark
Feb 2, 2024
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