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Latest From AveXis, Inc.
Gene therapy remains attractive when data show new technologies can advance the field, but gene regulation, editing and RNA therapies are grabbing investors’ attention, panelists said at ARM’s Cell and Gene Meeting on the Mesa.
Bluebird bio’s decision to focus on the US market is being seen as a broader signal of the need for changes in market access policies to enable gene therapies to be made available for European patients. Only collaborative work will create solutions acceptable to payers and companies, and finding an acceptable price that both can live with will be crucial.
bluebird bio's decision to wind down commercial initiatives for its gene therapy business in Europe is a blow for the sector in the region as a whole.
Health technology assessment body NICE has welcomed further discussion between Orchard Therapeutics and the National Health Service to resolve its concerns over the company’s one-time gene therapy for the neurodegenerative disorder, metachromatic leukodystrophy, in children.
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- BioLife Cell Bank, Inc.