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 […]

Challenging the central dogma

You may have caught Malcolm Gladwell’s The Obscure Virus Club on his Revisionist History podcast. I heard it this past year and thought of it again recently after listening to one of our internal short-course training sessions at AVROBIO on the Principles of Biology. A day later, I caught Short Wave’s podcast on Ignaz Semmelweis, […]

Burnout or exhaustion: Learn the difference to help ourselves and colleagues address the issues

I recently listened to this Harvard Business Review podcast – an interview with Christina Maslach, a professor of psychology at the University of California, Berkeley, who studies the causes and impact of burnout. This is an important topic for all of us, personally and professionally. This podcast is timely as we continue our cadence 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 […]

Overcoming the blood-brain barrier: New approaches to treating CNS complications of lysosomal disorders

My colleagues and I returned last month from the WORLDSymposium, a bustling scientific conference devoted exclusively to the dozens of rare genetic diseases classified as lysosomal disorders. We heard inspiring stories from families and advocates and reviewed the latest research from around the world. It is impossible not to come away optimistic for the future […]