At AVROBIO, we often talk about how our gene therapy platform, plato®, includes a variety of cutting-edge technologies that we can bring to bear on our investigational gene therapy programs. This includes the use of proprietary peptide tags designed to help therapeutic protein get to where it needs to go – a kind of fast-track […]
Author Archives: avrobioprod
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, […]
Nurturing a culture of “smart speed” to bring gene therapy to the world
As we approach our 5th anniversary and surpass our 125th hire, we have come to recognize that one of the most important characteristics for success at AVROBIO is a commitment to our mantra of “smart speed.” We’ve been thinking a lot about what that term means for us – and for the field of gene […]
Optimization is the name of the game in lentiviral gene therapy
In the field of lentiviral gene therapy, there is considerable focus on the most technical and novel steps of the process: transducing the patient’s stem cells and building a manufacturing system that can deliver optimized drug product at scale. AVROBIO is no exception; we’ve spent quite a lot of time driving improvements in these areas. […]
Strategic thinking in the age of COVID-19
Even though you couldn’t see everyone at the same time in the Zoom meeting, you could tell the AVROBIO R&D team was on fire. People were leaning into their screens, all devices and apps off, using the chat function, ready to talk. We had moved a long-planned offsite into the virtual world and condensed the […]
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 […]
A year of promise: Five key gene therapy developments to watch in 2020
The new year always brings new promise – and that’s certainly true in our fast-moving field of gene therapy. We saw some tremendous advances last year, such as regulatory approvals of two new therapies – as well as several less splashy developments worth keeping an eye on. I have high hopes that the field will […]
