Novartis replaces top scientists at Avexis after drug data manipulated

Novartis AG said on Wednesday it replaced the two top research and development executives at its Avexis unit after some data was manipulated from early testing of a gene therapy for infants that costs more than $2 million.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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Leaning into its success with gene therapy, Pfizer (NYSE: PFE) has announced a half-billion dollar expansion of its Sanford, North Carolina, footprint, which is expected to bring 300 jobs to the region. The $500 million investment will be used for the construction of a gene therapy manufacturing facility. Pfizer already employs about 3,600 in North Carolina at sites such as Chapel Hill and Morrisville. In Sanford, the company already employs about 650. Pfizer, based in New York, has research…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
The Massachusetts-based gene therapy company celebrated its new 25,000-square-foot space with a ribbon-cutting Aug. 16.
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
The Massachusetts-based gene therapy company celebrated its new 25,000-square-foot space with a ribbon-cutting Aug. 16.
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
AbstractLong QT syndrome (LQTS) is an inherited primary arrhythmia syndrome that may present with malignant arrhythmia and, rarely, risk of sudden death. The clinical symptoms include palpitations, syncope, and anoxic seizures secondary to ventricular arrhythmia, classicallytorsade de pointes. This predisposition to malignant arrhythmia is from a cardiac ion channelopathy that results in delayed repolarization of the cardiomyocyte action potential. The QT interval on the surface electrocardiogram is a summation of the individual cellular ventricular action potential durations, and hence is a surrogate marker of the abnorma...
Source: Pediatric Cardiology - Category: Cardiology Source Type: research
Pfizer is injecting $500 million into a manufacturing facility in Sanford, North Carolina to position itself as a bigger player in the gene therapy market.
Source: PharmaManufacturing.com - Category: Pharmaceuticals Source Type: news
Leaning into its success with gene therapy, Pfizer (NYSE: PFE) has announced a half-billion dollar expansion of its Sanford footprint, which is expected to bring 300 jobs to the region. The $500 million investment will be used for the construction of a state-of-the-art gene therapy manufacturing facility in Sanford. Pfizer already employs about 3,600 in North Carolina at sites such as Chapel Hill and Morrisville. In Sanford, the company has about 650 employees. The expansion is necessitated to…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Hailey-Hailey disease (HHD), or familial benign pemphigus, is a heritable skin condition characterized by chronic waxing and waning blisters in intertriginous areas. The disease is caused by a mutation of the ATP2C1 gene, encoding a Ca+2-ATPase. Many treatments have been used for the disease, yet there is no effective treatment due to the lack of gene therapy. There are several reports of the use of low-dose naltrexone (LDN) to treat HHD. We add 3 cases of biopsy-proven HHD treated with naltrexone to the current literature.
Source: Journal of the American Academy of Dermatology - Category: Dermatology Source Type: research
XPD, coded by ERCC2, is a helicase component of the basal transcription factor, TFIIH, involved in both DNA repair and transcription. Mutations in XPD that cause xeroderma pigmentosum (XP) may be predominately associated with repair-affecting mutations while transcription-affecting XPD mutations may be predominately associated with trichothiodystrophy (TTD). XP is characterized by extreme sensitivity to sun damage, sun-induced cancers and neurodegeneration in some patients. The diagnostic symptom of TTD is brittle, sulfur-deficient hair, but subjects have a broad range of features including skeletal, developmental, and immune abnormalities.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
The epidermal differentiation complex (EDC) includes over fifty genes encoding proteins involved in keratinocyte development. These belong to three gene families: the cornified envelope precursor family, the S100 protein family and the S100 fused type protein (SFTP). Of these fifty genes, filaggrin (FLG) located on chromosome 1 q21, a member of the SFTP family, is the most studied in the context of skin barrier dysfunction. We investigated the frequency and effect of rare loss-of-function (LOF) variants within the EDC in patients of a clinical study, VP-VLY-686-2102.
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
Cutaneous neurofibromas (cNF) are complex tumors of the skin in individuals with neurofibromatosis type 1 (NF1), a syndrome affecting approximately 1:3,000 persons worldwide. There are no drug therapies for these tumors, and current treatment is limited to various procedure-based approaches. A critical need in conducting meaningful translational studies of these tumors is to have high quality tissue samples representing the diversity of human cutaneous tumors. Multiple challenges exist, including a lack of standardized sample collection or analysis protocols, non-universal terminology for cNF, different methods for the rem...
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
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