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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Date: Monday, 08 20, 2018; Speaker: Christopher P. Austin, MD, Director , NCATS/NIH; Wilson Bryan, MD, Director, Office of Tissues&Advanced Therapies, CBER/FDA; Anne Pariser, MD, Director, Office of Rare Diseases Research, NCATS/NIH; Peter Marks, MD, Director, Center for Biologics Evaluation and Research, FDA; Building 10; Masur Auditorium; Videocast Event
Source: NIH Calendar of Events - Category: American Health Source Type: events
New drugs and gene therapies designed to treat cancer, rare diseases, etc. are justifiably commanding high prices. However, if they don ’t work, payments shouldn’t be required.
Source: Forbes.com Healthcare News - Category: Pharmaceuticals Authors: Tags: NYSE:NVS NASDAQ:AMGN Source Type: news
Japan has taken measures for specially defined rare diseases, called Nan-Byo (difficult-illness) in Japanese since 1972. While this governmental support has benefited Nan-Byo patients, those suffering from unidentified conditions do not fall into the scheme and cannot receive subsidies nor chances for consultation. To identify such rare and often undiagnoseddiseases, we must integrate systematic diagnosis by medical experts through global data matching, thereby solving the Nof1 problem. Thus, AMED launched the Initiative on Rare and Undiagnosed Diseases (IRUD) to construct a holistic medical network utilizing the existing ...
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
man D Abstract Many rare monogenic diseases are treated by protein replacement therapy, in which the missing protein is repetitively administered to the patient. However, in several cases, the missing protein is required at a high and sustained level, which renders protein therapy far from being adequate. As an alternative, a gene therapy treatment ensuring a sustained effectiveness would be particularly valuable. Liver is an optimal organ for the secretion and systemic distribution of a therapeutic transgene product. Cutting edge non-viral gene therapy tools were tested in order to produce a high and sustained le...
Source: European Journal of Medical Genetics - Category: Genetics & Stem Cells Authors: Tags: Eur J Med Genet Source Type: research
Japan has taken measures for specially defined rare diseases, called Nan-Byo (difficult-illness) in Japanese since 1972. While this governmental support has benefited Nan-Byo patients, those suffering from unidentified conditions do not fall into the scheme and cannot receive subsidies nor chances for consultation. To identify such rare and often undiagnoseddiseases, we must integrate systematic diagnosis by medical experts through global data matching, thereby solving the Nof1 problem. Thus, AMED launched the Initiative on Rare and Undiagnosed Diseases (IRUD) to construct a holistic medical network utilizing the existing ...
Source: Journal of Investigative Dermatology - Category: Dermatology Authors: Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research
GlaxoSmithKline is divesting its rare disease gene therapy drugs to Orchard Therapeutics, it said on Thursday, as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.Reuters Health Information
Source: Medscape Pathology Headlines - Category: Pathology Tags: Pathology & Lab Medicine News Source Type: news
GlaxoSmithKline is getting out of the rare disease gene therapy business. The London-based pharmaceutical company, which has large operations in Philadelphia and Montgomery County, said Thursday it has entered into an agreement to transfer its portfolio of approved and investigational rare disease gene therapies to Orchard Therapeutics. Under the terms of the deal, GSK will receive a 19.9 percent equity stake in Orchard — which is based in London and has U.S operations in California — and receive…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
LONDON (Reuters) - Britain's Orchard Therapeutics, which has already raised more than $140 million to fund its work in gene therapy, plans another private sale of shares following its acquisition of a portfolio of GlaxoSmithKline rare disease medicines.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
LONDON (Reuters) - GlaxoSmithKline is divesting its rare disease gene therapy drugs to Orchard Therapeutics, it said on Thursday, as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
Biotech group Orchard Therapeutics acquires €594,000 treatment for ‘bubble boy’ syndrome
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news
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