Category REGENERATIVE MEDICINE

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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An emerging view of RNA transcription and splicing

P.M. Fornasari 08/10/2019 Whitehead Institute scientists find chemical modification contributes to trafficking between non-membrane-bound compartments that control gene expression. Cells often create compartments to control important biological functions. The nucleus is a prime example; surrounded by a membrane, it houses the genome. Yet cells also harbor enclosures that are not membrane-bound and more transient, like

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ICER closely monitoring FDA’s Zolgensma investigation

Following an announcement by the FDA that it is investigating ‘data manipulation’ of Novartis’ Zolgensma, the Institute for Clinical and Economic Review (ICER) has commented that this could lead to a re-evaluation of its initial cost-effectiveness assessment of the drug.   Novartis’ gene therapy Zolgensma was approved by the FDA to treat spinal muscular atrophy (SMA) in children under two

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Centers for Medicare & Medicaid Services (CMS) Make CAR T-Cell Cancer Therapy Available to Medicare Beneficiaries Nationwide

Decision ensures consistency in access to the innovative new cancer therapy, and CMS is working closely with sister agencies to monitor outcomes for patients receiving the therapy Today the Centers for Medicare & Medicaid Services (CMS), under the leadership of President Trump and Secretary Azar, finalized the decision to cover FDA-approved Chimeric Antigen Receptor T-cell, or “CAR

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Industrial Biotechnology: To What Extent Is Responsible Innovation on the Agenda?

The UK Industrial Biotechnology (IB) Strategy presents a consistent plan to develop the IB sector but fails to endorse an innovation process that allows for input from multiple publics. This could be disadvantageous for the bioeconomy: there are notable cases where negligence to address societal dimensions has caused innovation failure. In 2018, the UK Industrial

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Muscular contribution to adolescent idiopathic scoliosis from the perspective of stem cell-based regenerative medicine.

Source https://www.liebertpub.com/doi/pdf/10.1089/scd.2019.0073 Adolescent idiopathic scoliosis (AIS) is a relatively frequent disease within a range 0.5-5.0% of population, with higher frequency in females. While a resultant spinal deformity is usually medically benign condition, it produces far going psychosocial consequences, which warrants attention. The etiology of AIS is unknown and current therapeutic approaches are symptomatic only, and

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Car-T: the first agreement for new therapies, based on actual results, is ready. Martini (Ex Dg Aifa): “A new negotiation model that puts Italy at the forefront”

The first agreement (with Novartis) for therapies that allow to reprogram some cells of the patient (the T lymphocytes), in order to make them able, once reinfused, to recognize and target the tumors, is approved by the CPR and CTS of Aifa. Now the agreement will have to be ratified by the AIFA Board and

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University of Minnesota achieves transplant in monkeys without long-term immune-fighting drugs

The technique could greatly increase the number and safety of organ transplants.  University of Minnesota researchers are reporting a breakthrough in what has long been viewed as a holy grail in the field of organ transplantation — success without the permanent need for immunosuppressive drugs. Their technique could greatly increase the number and safety of

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Characterizing Direct-to-Consumer Stem Cell Businesses in the Southwest United States

There are currently hundreds of businesses across the United States offering direct-to-consumer stem cell treatments that have not been through regulatory approval by the Food and Drug Administration (FDA). Here, we provide a detailed characterization of nearly 170 stem cell businesses operating in the Southwest United States. We draw specific attention to two as-yet understudied

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Injectable ‘bone spackling’: A cell therapy approach to heal complex fractures

Large, complex bone wounds are hard for doctors and patients alike to contend with. They often require grafts and multiple surgeries. Jan Stegemann, a University of Michigan professor of biomedical engineering, is reprogramming adult cells from bonemarrow so that they can be injected directly into a wound and grow into bone. The marrow-derived cells are known as progenitor cells, a type

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