UK regulator selects tiny Massachusetts biotech's potential Gaucher gene therapy for accelerated pathway
The UK’s Medicines and Healthcare products Regulatory Agency is increasingly granting drugmakers access to its exclusive Innovative Licensing and Access Pathway, with Cambridge, MA-based Avrobio announcing Tuesday the addition of its investigational gene therapy that uses patients’ own hematopoietic stem cells to treat a rare disease.
Under the pathway, which in early 2021 began awarding so-called “passports” to the five-month, accelerated review pathway, patients could potentially gain access to rare disease treatment more quickly.
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