Fabry Disease, Gaucher Disease, Pompe Disease and Cystinosis: AVROBIO has One Gene Therapy Platform to Treat them All

November 1, 2018

Geoff MacKay is President and Chief Executive Officer of AVROBIO, Inc. Mr. MacKay is an experienced CEO with proven success leading innovative businesses. While CEO of Organogenesis Inc., the company treated 1 million patients with living cell therapies, received the first FDA CBER allogeneic cell-therapy approval and achieved an unparalleled position within regenerative medicine.

Mr. MacKay was founding CEO of eGenesis, applying CRISPR Cas-9 gene editing to xenotransplantation. Mr. MacKay spent 11 years at Novartis in senior leadership positions within the Global Transplantation & Immunology franchise. Past activities include Chairman of the board of MassBio, Chairman of the board of the Alliance of Regenerative Medicine and Advisory Council to the Health Policy Commission for Massachusetts.

In this exclusive 3,272 word interview with the Wall Street Transcript, Geoff MacKay describes the exciting breakthrough treatments his company is developing.

“The target product profile for all four of our gene therapy programs is to cure these rare diseases in a single dose. This is achievable because the lentiviral gene therapy approach is one that results in the permanent integration of the therapeutic transgene into the chromosomes of the patient’s blood stem cells.

These cells engraft long term in the bone marrow, where they produce nucleated daughter cells, such as white blood cells, which in turn make supra-normal levels of the therapeutic protein. To date, this approach has been used in clinical trials across a growing number of diseases where long-term results have been reported. We do believe that the ex vivo lentiviral gene therapy approach, if successful, has the potential to be a lifelong cure.”

These disease therapies represent billion of dollars of potential annual revenue to AVROBIO:

“…We are currently targeting lysosomal storage disorders, specifically four unique lysosomal storage disorders: Fabry disease, Gaucher disease, Pompe disease and cystinosis. We are applying one technology platform across these four lysosomal storage disorders, as well as building toward developing gene therapies for other diseases in the future.”

Get the complete detail and the most recent status of these treatments by reading the entire 3,272 word interview in the Wall Street Transcript.