Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies (Day 1)

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing for rare disease gene therapies. Workshop sessions will explore: prominent manufacturing formats and technologies; major logistical bottlenecks; strategies to streamline scale transitions; drug master file best practices and opportunities; analytics and lot release testing; prospects for innovation; and barriers to innovation.Air date: 1/28/2020 8:30:00 AM
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video

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Publication date: March 2020Source: The Lancet Haematology, Volume 7, Issue 3Author(s): Michael Makris
Source: The Lancet Haematology - Category: Hematology Source Type: research
Publication date: March 2020Source: The Lancet Haematology, Volume 7, Issue 3Author(s): The Lancet Haematology
Source: The Lancet Haematology - Category: Hematology Source Type: research
In today's research materials, scientists report on the discovery of a maladaptive response to the presence of tau aggregates in brain cells, one that makes the situation worse than it would otherwise be. Tau is one of a small number of proteins that can become altered in a way that ensures other molecules of the same protein also alter. They join together and precipitate into solid structures, known as neurofibrillary tangles in the case of tau, accompanied by a halo of disrupted biochemistry that is harmful to cell and tissue function. This spreads, seeding dysfunction as it moves from cell to cell, or throughout a tissu...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Condition:   Adenovirus Infections, Human Intervention:   Sponsor:   Chimerix Completed
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: Available online 26 February 2020Source: Urology Case ReportsAuthor(s): Erdem Aktas, Murat Ucar, Bahar Akkaya, Erol Guntekin, Orkun Batmaz
Source: Urology Case Reports - Category: Urology & Nephrology Source Type: research
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
Conclusion This study described four Chinese LINCL siblings who were diagnosed by WES. The patients of these four families had similar disease courses started from motor regression or seizures to cognition regression and visual loss but carried mutations in different genes i.e. CLN2, CLN5, CLN6, and CLN7. The clinical features of LINCLs in these four Chinese siblings were not significantly different from those of Western patients. However, all Chinese LINCL patients in this study presented similar clinical course despite the affected genes. We assumed it as an ethnic specific clinical course according to our observation. ...
Source: Frontiers in Genetics - Category: Genetics & Stem Cells Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
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is Marfany Many rare diseases course with affectation of neurosensory organs. Among them, the neuroepithelial retina is very vulnerable due to constant light/oxidative stress, but it is also the most accessible and amenable to gene manipulation. Currently, gene addition therapies targeting retinal tissue (either photoreceptors or the retinal pigment epithelium), as a therapy for inherited retinal dystrophies, use adeno-associated virus (AAV)-based approaches. However, efficiency and safety of therapeutic strategies are relevant issues that are not always resolved in virus-based gene delivery and alternative methodol...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
Abstract Rare-disease drug development is both scientifically and commercially challenging. This case study highlights Agilis Biotherapeutics (Agilis), a small private biotechnology company that has developed the most clinically advanced adeno-associated virus (AAV) gene therapy for the brain. In an international collaboration led by Agilis with National Taiwan University (NTU) Hospital and the Therapeutics for Rare and Neglected Diseases (TRND) program of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health, Agilis' gene therapy for aromatic L-amino acid decarboxyl...
Source: Drug Discovery Today - Category: Drugs & Pharmacology Authors: Tags: Drug Discov Today Source Type: research
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