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GeneTherapy news

Amicus CEO Crowley bags a gene therapy company, a new pipeline — and a great photo op — for $100M in cash

Endpoints News - Sep 20, 2018
The key figure you shouldn't lose sight of here is Celenex co-founder Brian Kaspar of Nationwide Children's Hospital in Columbus, who spearheaded the science work on AveXis' gene therapy for spinal muscular atrophy — since bought out by Novartis for ...

Nightstar gene therapy used in first US patient with X-linked retinitis pigmentosa

Healio - Sep 19, 2018
A patient at Bascom Palmer Eye Institute has become the first in the United States to receive an innovative gene therapy treatment for X-linked retinitis pigmentosa as part of a Nightstar Therapeutics clinical trial, according to a press release. Julio ...

Amicus Expands into Gene Therapy with Celenex Acquisition

Genetic Engineering & Biotechnology News - Sep 20, 2018
Amicus Therapeutics said today it has acquired Nationwide Children's Hospital spinout Celenex for up to $452 million, in a deal that expands the rare disease drug developer into gene therapy with a portfolio of 10 clinical and preclinical programs in ...

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Gene Therapy videos

Gene Therapy -- The time is now: Nick Leschly at TEDxBoston


Gene Therapy Explained

Many diseases have a genetic basis, which means that the disease is caused by mutated genes which provide incorrect instructions that cause the cell to ...

What is gene therapy?


Gene therapy lecture 2 | ex vivo gene therapy

Gene therapy explained. This gene therapy lecture explains about ex vivo gene therapy. http://shomusbiology.weebly.com/ Download the study materials here- ...

Gene therapy part 1 : basics of gene therapy

Gene therapy explained. This gene therapy lecture explains the basic principles of gene therapy http://shomusbiology.weebly.com/ Download the study ...