Novartis executive sold shares before drug data manipulation made public

An unnamed Novartis executive sold 925,400 Swiss francs ($946,000) worth of shares less than three weeks before the U.S. Food and Drug Administration (FDA) announced data from tests of its gene therapy Zolgensma had been manipulated.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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Publication date: Available online 14 September 2019Source: Biochimica et Biophysica Acta (BBA) - General SubjectsAuthor(s): James J. Miller, Adam J. Kanack, Nancy M. DahmsAbstractBackgroundFabry disease is caused by α-galactosidase A deficiency. Substrates of this lysosomal enzyme accumulate, resulting in cellular dysfunction. Patients experience neuropathic pain, kidney failure, heart disease, and strokes.Scope of reviewThe clinical picture and molecular features of Fabry disease are described, along with updates on disease mechanisms, animal models, and therapies.Major conclusionsHow the accumulation of α-ga...
Source: Biochimica et Biophysica Acta (BBA) General Subjects - Category: Biochemistry Source Type: research
Publication date: Available online 12 September 2019Source: Biochimica et Biophysica Acta (BBA) - Molecular Basis of DiseaseAuthor(s): Kauss Valerjans, Dambrova Maija, Medina Diego LuisAbstractNeuronal ceroid lipofuscinoses, also collectively known as Batten disease, are a group of rare monogenic disorders caused by mutations in at least 13 different genes. They are characterized by the accumulation of lysosomal storage material and progressive neurological deterioration with dementia, epilepsy, retinopathy, motor disturbances, and early death [1]. Although the identification of disease-causing genes provides an important ...
Source: Biochimica et Biophysica Acta (BBA) Molecular Basis of Disease - Category: Molecular Biology Source Type: research
This study aims to evaluate cell proliferation and the downstream expression pattern of hPTTG1 gene at the mRNA and protein levels after specific down-regulation of hPTTG1 by siRNA.Main methodsThe human breast cancer MDA-MB-231 cell line was transfected with siRNA against hPTTG1. The mRNA and protein expression levels were examined by Real-time PCR and Western blot, respectively. The cell proliferation was assayed by MTS. To investigate the pattern of protein expression, total cellular protein was analyzed by 2D gel electrophoresis and mass spectroscopy. Subsequently, the possible biological consequences were determined by...
Source: Life Sciences - Category: Biology Source Type: research
Castle Creek Pharmaceuticals Holdings has reached an agreement to acquire Chester County cell and gene therapy company Fibrocell Science Inc. in a deal valued at $63.3 million. According to documents filed with the Securities and Exchange Commission, Castle Creek will pay Fibrocell stockholders $3 for each held and assume Fibrocell's debt. The purchase price represents a 63% premium compared to the company’s average trading levels during the last month. The deal, whic h still requires shareholder…
Source: Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
CORNERSTONE program integrates process development expertise and novel technology to remove development bottlenecks in the manufacture of Gene Therapy Medicinal Products (GTMPs). Portfolio includes...
Source: The Scientist - Category: Science Tags: The Scientist The Marketplace Source Type: news
Abstract The deletion of Arginine 14 of the phosholamban gene (PLN p.R14del) is associated with the pathogenesis of an inherited form of cardiomyopathy with prominent arrhythmias. Patients carrying the PLN R14del mutation are at risk of developing dilated cardiomyopathy or arrhythmogenic right ventricular cardiomyopathy. Although the genetic etiology is well defined, the molecular mechanism underlying the pathogenesis of PLN R14del-cardiomyopathy is unknown. Our CURE PLaN network, funded by the Foundation Leducq, will bring together leading scientists, clinicians, and patients to elucidate the genotype-phenotype r...
Source: Circulation Research - Category: Cardiology Authors: Tags: Circ Res Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
On Sept 17. 1999, Jesse Gelsinger died after receiving an experimental gene therapy from James Wilson's lab at the University of Pennsylvania. That tragedy waylaid Wilson's career and almost shut down the whole field. Wilson and his team put their heads down and spent the next decade searching for safer gene therapies. Today, his lab's $70 million annual budget and bevy of biotech partnerships are fueling the gene therapy explosion. The resurgence was never a sure thing. Here's how it happened.
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
Management to host conference call today at 8:30 a.m. Eastern SAN DIEGO, Sept. 12, 2019 -- (Healthcare Sales &Marketing Network) -- Tocagen Inc. (Nasdaq: TOCA), a clinical-stage, cancer-selective gene therapy company, today announced that the Toca 5 P... Biopharmaceuticals, Oncology Tocagen, glioma, brain cancer, gene therapy
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
Publication date: December 2019Source: Molecular Genetics and Metabolism Reports, Volume 21Author(s): Yvonne L. Latour, Robin Yoon, Sarah E. Thomas, Christina Grant, Cuiling Li, Miguel Sena-Esteves, Maria L. Allende, Richard L. Proia, Cynthia J. TifftAbstractGM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile to late onset and is uniformly fatal, with no effective therapy currently available. Although animal models have been useful for under...
Source: Molecular Genetics and Metabolism Reports - Category: Genetics & Stem Cells Source Type: research
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