GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics

GSK and Orchard Therapeutics have announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. This acquisition strengthens Orchard's position as a global leader in gene therapy for rare diseases.
Source: World Pharma News - Category: Pharmaceuticals Tags: Featured GlaxoSmithKline Business and Industry Source Type: news

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Source: - Category: Pharmaceuticals Source Type: news
Abstract Recent innovations in cutting-edge sequencing platforms have allowed the rapid identification of genes associated with communicable, noncommunicable and rare diseases. Exploitation of this collected biological information has facilitated the development of nonviral gene therapy strategies and the design of several proteins capable of editing specific DNA sequences for disease control. Small molecule-based targeted therapeutic approaches have gained increasing attention because of their suggested clinical benefits, ease of control and lower costs. Pyrrole-imidazole polyamides (PIPs) are a major class of DN...
Source: Advanced Drug Delivery Reviews - Category: Drugs & Pharmacology Authors: Tags: Adv Drug Deliv Rev Source Type: research
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Source: The British Journal of Ophthalmology - Category: Opthalmology Authors: Tags: Br J Ophthalmol Source Type: research
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Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news
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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Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
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Source: Blood - Category: Hematology Authors: Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research
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Source: Asbestos and Mesothelioma News - Category: Environmental Health Authors: Source Type: news
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Source: The Health Care Blog - Category: Consumer Health News Authors: Tags: Artificial Intelligence Pharmaceuticals Physicians AI David Shaywitz Health Tech Source Type: blogs
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Source: Healthcare News - Category: Pharmaceuticals Authors: Tags: NASDAQ:AVXS NYSE:NVS Source Type: news
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