Category GENE THERAPY RARE DISEASES

Novartis ousted top scientists over manipulation of data for gene therapy

Source STAT Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for

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The Landscape of Cellular and Gene Therapy Products: Authorization, Discontinuations, and Cost

Source Human Gene Therapy Clinical Development Background: Cell and gene therapy products belong to a diverse class of biopharmaceuticals known as advanced therapy medicinal products. Cell and gene therapy products are used for the treatment and prevention of diseases that until recently were only managed chronically. The objective of this study was to examine the characteristics

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Sangamo and Pfizer report data for haemophilia A gene therapy

Sangamo Therapeutics and Pfizer have reported additional findings from the Phase I/II Alta clinical trial of investigational gene therapy SB-525 to treat patients with severe haemophilia A. SB-525 is made up of a recombinant adeno-associated virus serotype 6 vector (AAV6) that encodes the complementary deoxyribonucleic acid for B domain deleted human Factor VIII (FVIII). During

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How will we pay for the coming generation of potentially curative gene therapies? Bill Cassidy US Senator proposals

We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such

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Gene Therapies clinical trials see 17% jump year-over-year, with two approvals near and pipelines expanding

Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned. ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were

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