News and Trends 18 Dec 2017 British Biotech Takes Over Development of Gene Therapy for Rare Immune Disease Orchard Therapeutics has licensed a gene therapy for the rare genetic disorder X-linked chronic granulomatous disease (X-CGD) from the non-profit Généthon. London-based Orchard Therapeutics is adding a new program to its pipeline of gene therapies. The company has obtained an exclusive license to a lentiviral vector to treat X-linked chronic granulomatous disease and the clinical trial data […] December 18, 2017 - 2 minutesmins - By Clara Rodríguez Fernández Share WhatsApp Twitter Linkedin Email
News and Trends 22 Jun 2017 French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy Noble Généthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease. Dedicated to rare diseases since 1990 and more recently to gene therapy, Généthon is one of very few not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is […] June 22, 2017 - 3 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email
News and Trends 28 Mar 2017 Why did Biotech Beat Pharma to Bring the First Gene Therapy to Market? Pharma would seemingly best biotech in any competition. A few CEOs explain why it left the honor of “The First Gene Therapy” to biotech. On July 20, 2012, the western world’s first gene therapy, Glybera, received marketing authorization from the EMA. The treatment was designed and developed by uniQure as one-off treatment for Lipoprotein Lipase Deficiency (LPLD), a […] March 28, 2017 - 5 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email
In Depth 21 Mar 2017 Rethinking Profits: Can a Socially Conscious Model work in Biotech? As biopharma continues to weather a maelstrom of public scrutiny for its prices and profits, I asked executives about alternative models. Conventional wisdom in biotech is that the non-profit model can’t work in the industry: it takes a decade and billions in cash to develop a drug, with no guaranteed success. No investor would sink […] March 21, 2017 - 7 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email