Gene editing expert Charlie Albright tells us why he hopped from Editas to a new role as CSO of an upstart biotech out to engineer gene therapy 2.0
We now know where Charlie Albright was headed when he jumped ship at CRISPR gene editing pioneer Editas a few weeks ago.
Albright has skipped across town to Waltham, MA-based Affinia, the newly hatched gene therapy biotech — from AveXis vets Sean Nolan and Rick Modi — looking to develop next-gen AAV delivery vehicles for a new wave of once-and-done therapies. And he’s been joined on the team by Petra Kaufmann, a vet from Novartis Gene Therapies — until recently known as AveXis.
“We’re really at the beginning of a new era of drug development with gene therapy,” Albright tells me, pointing to some big advances, as well as acknowledging some daunting setbacks that have occurred.
“It’s not surprising we’re seeing both successes and some setbacks,” he adds. “I came to Affinia because I thought we had the potential to address some of the fundamental issues in AAV gene therapy. And after my first 4 weeks here I believe that even more than when I took the job.”
The big idea at Affinia, what attracted Albright to the company, is the belief that the insights Luk Vandenberghe made in his Harvard lab can translate into new and much better capsids that can significantly improve efficacy, guard against toxicity and target a variety of new tissues that can open the door to a host of diseases now outside the reach of the currently available tech. The tech also has the potential to avoid resistance mechanisms, naturally reoccurring antibodies, and help solve manufacturing challenges.
“My job is to take that technology from an academic setting and industrialize that technology and at the same time point it toward the right combination of where the technology works and the unmet need is high,” says Albright.
“The platform is based on AAV capsids that come from the reconstruction of the ancestral sequences… We take this library of potential ancestors and we barcoded them so we can see which ancestor we’re looking at, or which capsids we’re looking at, and then we can inject them into animals and see which ones get more broadly distributed in the central nervous system, which ones allow better penetration into cardiac tissue, and thereby identify capsids that allow us to get the dose down and the amount of gene therapy we deliver way up.”
These variants based on ancestral sequences have high functionality, he notes. “We have evidence that that actually will work. In particular, we found a capsid that de-targets the liver, one of the major sites of toxicity that comes from high doses of systemic AAV. Another distributes gene therapy more broadly in the brain, though lumbar puncture, that outperforms AAV9.” Both are examples of what differentiates Affinia from the rest of the field, which has been exploding with new programs over the last few years.
Affinia still has a long way to go, but at this point in the journey, Albright feels he’s found a spot right on the cutting edge of the gene therapy boom. And he plans to ride it out to the clinic.