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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Roche Holding has entered a definitive merger agreement with Spark Therapeutics, as the Swiss drugmaker seeks to expand its ability to treat rare diseases through gene therapies, as well as build its hemophilia portfolio.
Source: PharmaManufacturing.com - 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
There are approximately 7,000 distinct rare diseases affecting 350 million people worldwide, approximately 80 percent of which are caused by faulty genes. Scientific advances such as the CRISPR/Cas9...(PRWeb January 10, 2019)Read the full story at https://www.prweb.com/releases/gene_therapy_for_rare_diseases_considerations_for_both_clinical_and_post_marketing_studies_new_webinar_hosted_by_xtalk/prweb16025009.htm
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
Conclusion:We report a first in human study with a novel form of cellular therapy, mitochondrial augmentation, in which we enrich HSCs with organelles encoding non-mutated version of the mtDNA sequence. We show the ability of mitochondrial augmentation to improve in vitro PS-derived HSC function, and improvement in metabolic determinants, aerobic capacity and quality of life of two patients treated. Together, these preliminary clinical data suggest that mitochondrial augmentation therapy is safe, and may alter the clinical course for patients with mitochondrial deletions/mutations including PS.FigureDisclosuresJacoby: Nova...
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
We are in a wondrous age of medicine where CURES for certain types of cancer, rare diseases, blindness and other scourges are imminent. We now need to figure out how to pay for them.
Source: Forbes.com Healthcare News - Category: Pharmaceuticals Authors: Tags: NASDAQ:AVXS NYSE:NVS Source Type: news
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