Archives September 2019

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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Funded by FDA, C-Path and NORD to Launch Rare Disease Data and Analytics Platform

The collaborative project between the organizations will kick off at a launch meeting in September and will aim to reduce barriers for the development of new treatments and cures for rare diseases The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD

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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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Novartis’ Phase III studies of ofatumumab achieve primary goals in relapsing multiple sclerosis

Novartis has reported positive findings from the Phase III ASCLEPIOS I and II clinical trials of ofatumumab (OMB157), which met their primary endpoints in relapsing forms of multiple sclerosis (RMS) patients. Ofatumumab is an anti-CD20 monoclonal antibody (mAb) designed to cause B-cell lysis and depletion. It has been formulated for once-monthly, subcutaneous self-administration. The identical

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University of Pennsylvania Announces Completion of Exclusive R&D Alliance With Novartis and Development of New Focused Relationship

Penn to Broaden Pathways for Development of New CAR T Cell Therapies Following a landmark, first-of-its-kind collaboration that spawned the global CAR T cell therapy industry, the University of Pennsylvania and Novartis have concluded their seven-year research and development alliance, which resulted in the world’s first approved gene therapy for cancer. They will continue to

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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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Report: Providers need strategy for disruptive digital health tech

If healthcare organizations want to succeed in an environment of digital transformation, they must bring about participatory health. That shift will require providers to be grounded in patient engagement and patient-centered care—by being “on-demand, connected and data-driven.” That’s the contention of a new report from the American Hospital Association’s Center for Health Innovation, and EY, which examines

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