Novartis says delayed telling FDA of Zolgensma concern due to 'mistake'

Novartis scientists learnt months ago about safety concerns surrounding its gene therapy Zolgensma but delayed telling the U.S. Food and Drug Administration (FDA) due to what the Swiss drugmaker said was a "mistake".
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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We are living in a new era of medicine where innovations are transforming our ability to attack the cause of a disease, not just the symptoms. Today, concepts, such ascell and gene therapies,that once sounded like science fiction are now becoming reality with the potential to cure or fundamentally alter the trajectory of many life-threatening conditions. This rapid state of innovation is due in part to the vast array of partnerships between government, the biopharmaceutical industry and academia that make up the biomedical research and development (R&D) ecosystem, which enables us to expand the frontier of science.
Source: The Catalyst - Category: Pharmaceuticals Authors: Tags: Research and Development Pelosi Plan & D Focus Source Type: news
Gene Therapy, Published online: 13 November 2019; doi:10.1038/s41434-019-0110-7A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Eyevensys, a clinical-stage biotechnology company based in France, has developed a method to perform non-viral gene therapy in the eye, with the aim of treating ocular diseases. The system uses ocular electrotransfection to deliver therapeutic genes ...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Genetics Ophthalmology Source Type: blogs
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Source: The Scientist - Category: Science Tags: News & Opinion Source Type: news
The workshop is organised by CARAT in collaboration with BIO.NRW.Advances in CAR-T cell develoment made with in CARAT will be presented. Further, various aspects such as production, application and reimbursement options for this novel therapeutic approach will be examined. In addition to scientific lectures, a panel discussion on opportunities and threats of the broad application of CAR-T cell therapies from different viewpoints will be held. Please register until November 26, 2019 by email: Dr Martina Wessling: m.wessling@bio.nrw.deTel: +49 211 385 469-9205Venue: Representation of the State of North Rhine-Westphalia to th...
Source: EUROPA - Research and Innovation: Events - Category: Research Tags: Health - D72D1707-FE0F-3C39-4E55830C194C2E7C Horizon 2020 - 719491AD-9C69-558D-B05B8AC79345ED08 Source Type: events
Shares in Solid Biosciences Inc plunged 71% on Tuesday after a trial testing its gene therapy for a muscle-wasting disorder was halted for the second time in less than two years.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Abstract An ongoing concern of in vivo gene therapy is adaptive immune responses against the protein product of a transgene, particularly for recessive diseases in which antigens are not presented to lymphocytes during central tolerance induction. Here we show that Toll-like receptor 9 (TLR9) signaling activates T cells against an epitope tagged mitochondria-targeted ornithine transcarbamylase (OTC) following the administration of a systemic adeno-associated virus (AAV) vector. Using a transgenic mouse model system, we demonstrate that TLR9 signaling extrinsic to T cells induces a robust cytotoxic T-cell response ...
Source: Cellular Immunology - Category: Allergy & Immunology Authors: Tags: Cell Immunol Source Type: research
Gene therapy approaches are being deployed to treat recessive genetic disorders by restoring the expression of mutated genes. However, the feasibility of these approaches for dominantly inherited diseases — where treatment may require reduction in the expression of a toxic mutant protein resulting from a gain-of-function allele — is unclear. Here we show the efficacy of allele-specific RNAi as a potential therapy for Charcot-Marie-Tooth disease type 2D (CMT2D), caused by dominant mutations in glycyl-tRNA synthetase (GARS). A de novo mutation in GARS was identified in a patient with a severe peripheral neuropath...
Source: Journal of Clinical Investigation - Category: Biomedical Science Authors: Source Type: research
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