Mind your manners: The lost art of etiquette in business negotiations

It’s tempting to think that the first skill you need to learn in business development is negotiating. That’s important, of course, but it should not be your first stop. Business development, at its core, is all about building relationships. And while that may sound simple, I’ve seen many up-and-comers stumble because they fail to understand […]

FDA and the CMC goalposts: CMC should accelerate, not curb, gene therapy progress

There’s no getting around it: This has been a challenging stretch for gene therapy companies on the chemistry, manufacturing and controls (CMC) front. We’ve all seen the headlines of clinical trial and BLA filing delays while companies respond to CMC concerns. Behind these headlines are real consequences: Patients have had to cope with heartbreaking delays […]

Moonlighting proteins: A surprising finding in a clinical trial suggests new insights into gene therapy

Science is full of surprises – it’s one of the things I love about this work. I’ve been reminded of this recently in light of an initial observation from the Phase 1/2 clinical trial[1] of AVR-RD-04, our investigational lentiviral gene therapy for cystinosis. Sponsored by our academic collaborator Stephanie Cherqui, Ph.D. at the University of […]

Cruella’s at the door

That’s what I call her: Cruella, my inner critic, the woman who’s perched in my psyche just dying to point out how much I suck. She raised her mean head just this week after I flubbed a presentation at a critical meeting.  I was mortified at my own mistake, seeing in my mind’s eye how […]

You want to be a CBO? Four tips to set you on your way

Most of the titles in a biotech C-suite are generally self-explanatory. The Chief Technical and Manufacturing Officer runs operations in CMC and analytics. The Chief Financial Officer handles financial strategy and investor relations. The Chief Commercial Officer prepares for bringing investigational therapies to market. Then there’s the title I hold: Chief Business Officer (CBO). This […]

Treating the whole body: The case for lentiviral gene therapy’s head-to-toe reach

We hear it regularly from patients and caregivers: There’s an urgent need for therapies that can treat the whole body to address the full range of symptoms in diseases that manifest in both the central nervous system (CNS) and peripheral tissues. Biologically, that’s a tall order. The brain is protected by an intricate blood-brain barrier […]

Our second wave: How tag technology is driving exciting preclinical results in Pompe disease research

At AVROBIO, we’ve been highly strategic in selecting our second wave of programs for our leading lentiviral gene therapy pipeline. We’re targeting three devastating lysosomal disorders in this next wave – Pompe disease, Hunter syndrome and Gaucher disease type 3 – each of which impacts multiple organ systems throughout the body and is marked by […]

You can’t yoga your way out of burnout: Reflections on keeping a team energized and productive during a pandemic

This pandemic year has been tough on all of us. But as I reflect back on 2020, I’ve realized that the strains also brought opportunity. The profound dislocations of the year prompted new ways of thinking about nurturing team culture, buoying morale and maintaining strong forward momentum as a business amid steep challenges. As chief […]