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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A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions.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
$430 Million Cell and Gene Therapy Center Set to Launch on Long Island, NY
Governor Kathy Hochul has announced New York Biogenesis Park, a $430 million cell and gene therapy hub set to transform the field on Long Island. Backed by a $150 million state investment, this 700,000-square-foot facility in Lake Success will focus on developing life-saving therapies, including treatments for cancer, genetic diseases, and chronic illnesses. The park will revolutionize medicine by repairing damaged DNA, modifying mutated genes, and engineering therapeutic cel
Dec 2, 2024
AI Creates Precise DNA “Switches” for Gene Therapy - A Step Closer to Targeted Treatments
Imagine controlling genes with the precision of a light switch— controlling specific parts of the body as you will. That’s what the researchers at Yale, Jackson Laboratory, and the Broad Institute aimed to achieved with a new AI tool called CODA (Computational Optimization of DNA Activity). This newly designed AI tool creates custom DNA “switches” that control when and where genes turn on or off, which could greatly improve gene therapy. What is CODA? CODA is a groundbreaking
Oct 23, 2024
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 19, 2024
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