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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
New Gene Therapy Breakthrough Paves Way for Better Treatment of Brain Disorders
In a project funded by the National Institute of Mental Health (NIMH) and the National Institute of Neurological Disorders and Stroke (NINDS), a group of researchers have created a novel adeno-associated viral (AAV) vector to address brain disorders. This vector is more effective at reaching brain cells, opening doors for the development of treatment for brain disorders such as Parkinson's, Alzheimer's, and Huntington's Disease. Adeno-associated viral (AAV) vectors are common
Aug 20, 2024
Two New CRISPR-Based Gene Therapies for Sickle Cell Anemia Approved by the FDA
The FDA recently approved two groundbreaking CRISPR-based gene therapies for curing Sickle Cell Disease (SCD): Casgevy - developed by Vertex Pharmaceuticals and CRISPR Therapeutics Lyfgenia - developed by Bluebird Bio By targeting the BCL11A gene, these therapies successfully correct SCD mutations and increase fetal hemoglobin (HbF) levels in patients. This is exciting news for patients and families living with this painful disease, as it presents a potential cure rather than
Mar 25, 2024
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