Ex-Novartis business head hops over to a gene therapy startup — and she's reeled in $80M for a dash to the clinic
Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.
“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News.
In 2019, he launched AviadoBio along with molecular neurobiologist Youn Bok Lee and vector biologist Do Young Lee to pursue a new take on gene therapy for neuro disorders. And on Thursday, they unwrapped an $80 million Series A round led to bring their lead candidate for frontotemporal dementia (FTD) into the clinic.
“I was an instant believer,” said Lisa Deschamps, who left her role as CBO of Novartis Gene Therapies to take the helm at AviadoBio in October.
The team is targeting diseases that have an aggressive early progression, Shaw said. While most of their competitors are putting vectors into the cerebrospinal fluid — a liquid found within the tissue that surrounds the brain and spinal cord — AviadoBio is looking to deliver the vectors right to the brain.
“We get fantastic expression by doing that,” Shaw said. “It also allows us to microdose — we’re giving about a thousandth of the dose of our competitors.”
Their lead candidate, AVB-PGRN, and an intrathalamically dosed therapy designed to deliver a functional copy of the progranulin (GRN) gene. FTD is caused by a deficiency of progranulin, which plays a role in lysosomal function, or the protein recycling part of the cell.
“Neurons are incredibly dependent on having good recycling because the neurons you die with are the neurons you’re born with,” Shaw said. “So you know, they can’t be adolescents. They can’t have a messy room. They’ve got to have a really, really crisp, tidy space … So what we’re trying to do is just return back to physiological levels this really important recycling protein.”
AviadoBio will compete with a handful of players working on potential treatments for FTD, including GSK-partnered Alector, which unveiled data this summer showing its antibody AL001 brought progranulin levels back to near-normal levels after a half-a-year. And Expansion Therapeutics unveiled an $80 million Series B round just a couple months ago to bring its small molecule RNA platform one step closer to the clinic – including a candidate for FTD.
Deschamps said AviadoBio’s candidate should enter the clinic by the end of next year, while another preclinical candidate for ALS is a bit further behind. The Series A round was led by New Enterprise Associates and Monograph Capital. A handful of big-name backers pitched into a $16.5 million seed round last year, including Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation (JJDC), and LifeArc.
Despite Big Pharma’s retreat from neuro a few years ago, the industry seems to be warming back up to the field, Deschamps said — and new companies are emerging with gene therapies to take a shot at difficult-to-treat conditions. This past summer, for example, Alcyone Therapeutics emerged from stealth with $23 million and 12 gene therapies in tow, with a rare neurodevelopmental condition called Rett syndrome up first.
“I was leading the global neuroscience business at Novartis … one of the things we had talked about years ago was how many Big Pharma companies were exiting the space. It is quite the opposite these days,” Deschamps said. “We see certainly a resurgence across big and small companies, you know, coming back into the neuroscience space.”