AVROBIO Announces New Positive Clinical Data and Preclinical Data, as Well as Expanded Leading Lysosomal Disorder Gene Therapy Pipeline

Excerpt from the Press Release:

CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced positive new data across its clinical programs in Gaucher disease type 1, Fabry disease and cystinosis, further reinforcing the potential of ex vivo lentiviral gene therapy for lysosomal disorders. Additionally, AVROBIO is further expanding its lysosomal disorder pipeline with a new program in Gaucher disease type 3, which joins the recently announced program in Hunter syndrome in a synergistic portfolio of six programs designed to prevent, halt or reverse genetic disease.

“We’re delighted to report substantial new data across our three clinical programs. Three months post-gene therapy infusion, the first Gaucher disease patient’s levels of the toxic metabolite plasma lyso-Gb1, as well as plasma chitotriosidase, were lower than the baseline levels when the patient was still on enzyme replacement therapy (ERT). With our Fabry disease data continuing to reflect sustained and durable results, with our first patient now out 3.5 years from dosing, we are planning our strategy to seek accelerated approvals in one or more major markets,” said Geoff MacKay, president and CEO of AVROBIO. “Additionally, the first patient in the investigator-sponsored trial for cystinosis, now out one year, remains off both oral and eye drop cysteamine and we are thrilled to announce that a third patient has been dosed.

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