Leading the way in bringing gene therapy to the CNS with conditioning: a renaissance for busulfan?

Effective treatment of the central nervous system (CNS) through gene therapy is a promising approach for the many genetic diseases with neurological manifestations. It’s a particular hurdle for diseases with both CNS and systemic involvement, where an effective therapy would need to work on both sides of the blood–brain barrier. Prominent among this group are […]

Picking the right path: The importance of identifying the right vector for gene therapy

I recently had the opportunity to participate in a terrific conversation with two of my fellow CEOs on a Meeting on the Mesa panel from other leading gene therapy companies entitled “State of Play in the Vector Arms Race: Perspectives on the Future of Gene Delivery.” From that title, you might have been expecting fireworks, […]

Removing Barriers To Achieve Widespread Clinical Adoption Of Lentiviral Gene Therapy

Lentiviral gene therapy, involving the genetic modification of stem cells outside of the body, requires a conditioning regimen in order to create the necessary space for engraftment of gene-modified cells in the bone marrow. Busulfan is an established conditioning agent, building on decades of use in bone marrow transplants as well as the treatment of […]

A recipe for managing data disclosure successfully with academic partners: A public gene therapy company perspective

When AVROBIO went public in June 2018, I found myself on the steep end of an unexpected but interesting learning curve regarding data disclosure. I had spent years leading Shire’s business development team, so I was well-versed in the regulatory requirements for public companies. However, I suddenly had to reckon with the complexities that ensued […]