Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products

Source: What's New at CBER - Category: Biomedical Science Source Type: news

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Basel, 27 May 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the phase III Archway study, evaluating Port Delivery System with ranibizumab (PDS) in people living with neovascular or “wet” age-related macular degeneration (nAMD). PDS is a permanent refillable eye implant, approximately the size of a grain of rice, which continuously delivers a customised formulation of ranibizumab over a period of months. The Archway trial met its primary endpoint, demonstrating that patient s with PDS who received refills every six months achieved visual acuity outcomes equivalent to tho...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news
Conditions:   SARS-CoV 2;   Viral Infection;   COVID 19 Intervention:   Biological: Partially HLA-matched Virus Specific T cells (VSTs) Sponsors:   Baylor College of Medicine;   Center for Cell and Gene Therapy, Baylor College of Medicine;   The Methodist Hospital System;   AlloVir Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
AbstractThe current US drug innovation financing framework rests on the notion that a defined period of marketing exclusivity combined with the expectation of reimbursement for clinically valuable, cost-effective therapies, followed by vigorous price competition from generic drugs and biosimilars ensures a sufficient return on investment (ROI) to incent private sector risk-based investment and research and development activities while providing access for new treatments to patients. While periodically, alternatives such as government prizes, direct purchases or development, and limits on certain incentives have been propos...
Source: PharmacoEconomics - Category: Health Management Source Type: research
Recombinant lentiviruses serve as important tools for diverse applications in vitro and in vivo. In research contexts, examples include ectopic protein expression [1], shRNA inhibition of targeted transcripts [2], gRNA / Cas9 CRISPR lentivirus libraries [3], and progenitor cell barcoding for lineage and tumor cell tracking studies [4, 5]. In clinical trials, significant progress is being made with lentiviruses as gene therapy vectors for select inherited diseases (e.g., thalassemia, SCIDX-1) [6, 7] and for CAR-T cell engineering [8].
Source: Experimental Hematology - Category: Hematology Authors: Source Type: research
Conditions:   SARS-CoV 2;   Viral Infection;   COVID 19 Intervention:   Biological: Partially HLA-matched Virus Specific T cells (VSTs) Sponsors:   Baylor College of Medicine;   Center for Cell and Gene Therapy, Baylor College of Medicine;   The Methodist Hospital System;   AlloVir Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
In conclusion, our results suggest a previously unknown mechanism whereby the canonical NF-κB cascade and a mitochondrial fission pathway interdependently regulate endothelial inflammation. Lin28 as a Target for Nerve Regeneration https://www.fightaging.org/archives/2020/05/lin28-as-a-target-for-nerve-regeneration/ Researchers here show that the gene Lin28 regulates axon regrowth. In mice, raised levels of Lin28 produce greater regeneration of nerve injuries. Past research has investigated Lin28 from the standpoint of producing a more general improvement in regenerative capacity. It improves mitoch...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
CONCLUSION: We demonstrated the anti-cancer effect of CAPE in human SCLC cells and studied the mechanism by acquiring a comprehensive transcriptome profile of CAPE-treated cells. PMID: 32445454 [PubMed - as supplied by publisher]
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
Protective effect of hypoxia inducible factor-1α gene therapy using recombinant adenovirus in cerebral ischaemia-reperfusion injuries in rats. Pharm Biol. 2020 Dec;58(1):438-446 Authors: Li YQ, Hui ZR, Tao T, Shao KY, Liu Z, Li M, Gu LL Abstract Context: Hypoxia-inducible factor-1α (HIF-1α)-induced genes can improve blood circulation.Objective: To investigate brain protective effect of recombinant adenovirus-mediated HIF-1α (AdHIF-1α) expression and its mechanism.Materials and methods: Male SD rats were used to establish focal cerebral ischaemia-reperfusion (CIR) injury mo...
Source: Pharmaceutical Biology - Category: Drugs & Pharmacology Tags: Pharm Biol Source Type: research
CONCLUSIONS: Our studies have shown that small regulatory molecules with organelle-specific effect (such as miRs) need to be targeted, and that the resultant effect is comparable to silencing of "general" hypoxic transcription factors. PMID: 32432754 [PubMed - in process]
Source: European Review for Medical and Pharmacological Sciences - Category: Drugs & Pharmacology Tags: Eur Rev Med Pharmacol Sci Source Type: research
I am a few months late in noting this: videos from last year's Longevity Forum, one of the events in the Longevity Week held in London in November 2019, in what now seems an entirely different world, were posted online earlier this year. The Longevity Forum is organized by Jim Mellon and allies, a part of his diverse efforts to advance the growth of a longevity-focused medical industry capable of turning back aging and significantly lengthening healthy life spans. The Forum is focused on non-profits, regulatory concerns, and government policy rather than on industry, but there are nonetheless interesting presentatio...
Source: Fight Aging! - Category: Research Authors: Tags: Healthy Life Extension Community Source Type: blogs
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