2021 Annual Report: CEO Geoff MacKay on how gene therapy’s promise propels us forward

2021 challenged us all in many ways. As successive waves of SARS-CoV2 variants swept around the globe, we saw a monumental scientific achievement in our industry – the development and distribution of a completely new vaccine – accompanied by various failures and missteps that undermined progress against the pandemic. It was an imperfect and at […]

Moving gene therapy forward: Predictions for the next phase of development

Gene therapy has encountered plenty of speed bumps in the past year, with companies across the industry dealing with safety issues, regulatory setbacks, chemistry, manufacturing and control (CMC) challenges and difficult questions over commercialization. AVROBIO has not been immune, as we recently deprioritized one of our clinical programs due to several factors, including emerging clinical […]

A periscope for gene therapy: How high-resolution methods like single-cell sequencing may help predict the fate of gene therapy drug product

In recent years, tools that enable deeper analysis of single cells have made incredible progress. We can now look at individual cells from a single sample and see how they differ from one another in terms of their DNA, gene expression and protein production. With these tools, each cell tells its own story about where […]

Inclusivity is key to scientific advancements: Why I helped start “Out Toxicologists and Allies”

In the modern world of science, things move fast. There is always a new innovation or discovery just around the corner, ready to change the field. And it’s all the result of people working together: a combination of great dedication, rigorous research, and steadfast support from colleagues. However, diversity of thought is the silent understructure […]

“Commercial” is not a dirty word: Why all biotech companies need a strong commercial mindset from the earliest stages

Does commercialization strategy matter when you are years from having a commercial product? In my experience in biotech, the answer is a resounding yes. Biotech is an industry that has a high rate of failure — 90 percent of drug candidates do not get approval.  Even if approval comes, a study by Deloitte Center for Health Solutions […]

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 […]