pharmaphorumGene therapy trials held back by fragmented EU rulespharmaphorumBiotech and pharma organisations are calling for EU countries to harmonise regulations, or otherwise face falling behind in cutting-edge gene and cell therapy trials. The Europ… Continue reading
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Gene therapy treatment can reach $500K for certain diseases
Genetic Literacy Project The idea behind gene therapy is that genetic material is used as a “living drug” to treat disease. Scientists have been trying to get gene therapies to work for decades, so Kymriah’s approval is historic. But the therapy’s $475,000 price tag was … |
Labiotech.eu (blog) |
French Gene Therapy for Wilson’s Disease gets Orphan Status
Labiotech.eu (blog) Vivet Therapeutics has been granted Orphan Drug Designation for its gene therapy for Wilson’s disease, VTX-801, by both the FDA and the European Commission. The French biotech Vivet Therapeutics is developing what could eventually become the first … |