Category REGENERATIVE MEDICINE

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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Multiple myeloma treatment granted FDA Orphan Drug Designation

Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Glenmark Pharmaceuticals’ bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies. “Patients who are struggling with diseases like

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FDA Grants Breakthrough Therapy Designation for Genentech’s Gazyva (Obinutuzumab) in Lupus Nephritis

The designation is based on the results of the Phase II NOBILITY study that showed Gazyva, in combination with standard of care, helped more people achieve a complete renal response than standard of care alone Genentech today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to Gazyva® (obinutuzumab) for

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SanBio Granted Regenerative Medicine Advanced Therapy Designation from the U.S. FDA for SB623 for the Treatment of Chronic Neurological Motor Deficits Secondary to Traumatic Brain Injury

The SanBio Group, a scientific leader in regenerative medicine for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation for SB623 cell therapy for the treatment of chronic neurological motor deficits secondary to traumatic brain injury (TBI). The designation is based on clinical results

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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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