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.

Orchard Therapeutics of the US and Dutch biotech Pharming have agreed to jointly research, develop, manufacture and commercialize the former’s newly disclosed investigational ex vivo autologous hematopoietic stem cell gene therapy as a treatment for hereditary angioedema.

Vertex and CRISPR can now get access to the advantages afforded by the European Medicines Agency’s priority medicines scheme for both of the indications they are targeting with their investigational gene therapy, CTX001.

The proposed $39bn acquisition of Alexion by AstraZeneca has a particular interest for its chief financial officer Marc Dunoyer who recently spoke to Scrip about his extensive experience in the rare disease space.