Category GENE THERAPY RARE DISEASES

Boston University researchers create new protocol to improve gene therapy tool production

Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus

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Zylka Lab awarded $6.1 million from NIH to develop CRISPR/Cas9 gene therapy for Angelman syndrome, study UBE3A autism gene with Philpot Lab

Mark Zylka, PhD, in The UNC Neuroscience Center was awarded NIH funding for two 5-year projects, one jointly with Ben Philpot, PhD, to develop new knowledge with the potential to advance treatments for neurodevelopmental disorders. The National Institutes of Health have awarded two separate grants totaling $6.1 million to Mark Zylka, PhD, director of the

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Gene Therapy Trials

Applications and the limitations of real-world data. Source Contract Pharma There are approximately 7,000 distinct rare diseases that exist affecting 350 million people worldwide, and approximately 80% of those rare diseases are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system1 have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies,

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Gene therapy may help functional recovery after stroke

A new gene therapy turns glial cells — abundant support cells in the brain — into neurons, repairing damage that results from stroke and significantly improving motor function in mice. A paper describing the new therapy, which uses the NeuroD1 gene, appears online in the journal Molecular Therapy. Once further developed, this NeuroD1-based gene therapy potentially

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Success of gene therapy for a form of inherited blindness depends on timing

An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients’ sight. But new research underscores the importance of further investigation to halt the progression of the disorder. Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was

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Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations

Gene therapies constitute a new concept of transformative therapies, administered once in a lifetime. The value assessment of these innovative therapies constitutes a challenge for health technology assessment (HTA) bodies. The HTA reports for all seven gene therapies that have to date been granted a market authorization in the European Union (EU) and/or the United

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Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study Ultragenyx Pharmaceutical, a

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Four U.S. CRISPR Trials Editing Human DNA to Research New Treatments

Breaking down how the gene editing technology is being used, for the first time in the United States, to treat patients with severe medical conditions Source Smithsonian.com Last fall, the birth of genetically edited twin girls in China—the world’s first “designer babies”—prompted an immediate outcry in the medical science community. The change to the twins’ genomes,

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