Source STAT A common saying at the Food and Drug Administration is: “In God we trust, all others must bring data.” The independent evaluation of science is an essential element of the FDA’s dual role of protecting the public health and promoting innovation to bring new therapies into practice. In fact, with certain exceptions, the
New research reveals how increasing brain stiffness as we age causes brain stem cell dysfunction, and demonstrates new ways to reverse older stem cells to a younger, healthier state. New research, published today in Nature, reveals how increasing brain stiffness as we age causes brain stem cell dysfunction, and demonstrates new ways to reverse older stem
The results of an early trial in 31 brain cancer patients finds immune activity boosted in the tumor, and possibly longer survival. An inducible, tumor-localized gene therapy has been tested for the first time in glioblastoma patients. The two-part approach, which involves receiving an injection of an immune-activator gene into the brain tumor site and
The gene editor targets cancer, blood disorders and blindness Since its debut in 2012, CRISPR gene editing has held the promise of curing most of the over 6,000 known genetic diseases. Now it’s being put to the test. In the first spate of clinical trials, scientists are using CRISPR/Cas9 to combat cancer and blood disorders
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
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
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
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
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
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
