Deerfield-backed biotech emerges from stealth with gene therapy delivery platforms from Harvard and MIT
As a PhD student at Tufts, Dave Greenwald studied the use of adeno-associated virus (AAV) vectors to deliver gene therapies to people with retinitis pigmentosa, a group of rare genetic eye diseases that causes the retina to break down over time.
While he later left lab life, he now sits at the helm of Apertura Gene Therapy, a biotech that hopes to customize AAV vectors that are used widely in delivering most gene therapies. Apertura announced its launch from stealth with $67 million in Series A financing from Deerfield, where Greenwald is also a vice president of business development.
The company acquired its AAV technology from Ben Deverman, a Broad Institute scientist who has spent nearly 15 years studying AAV vectors.
“When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids,” Deverman said in a press release.
On the other hand, his lab’s technology designs “custom AAV capsids that have the chosen characteristics for treating specific diseases,” he said, “and we believe this approach will result in new and effective gene therapies.”
When asked which specific diseases Apertura plans on going after with the custom AAV capsids, Greenwald said, “The nice thing about these platforms is that they are agnostic to cell type and organ, so we actually use them in pretty much any disease.”
However, he told Endpoints News, “we’re not disclosing specific therapeutic areas or indications at this time.”
Giving slightly more insight, Apertura’s director of corporate development Kristina Wang said, “Natural serotype AAVs don’t reach certain tissues very well, such as CNS, kidney, and a bunch of other tissues. We are engineering AAVs to get to those difficult-to-access tissues.”
Apertura also plans to incorporate another gene therapy technology, known as PESCA, that it acquired from a different Cambridge-area scientist — Michael Greenberg, the chair of Harvard’s neurobiology department.
While the AAV technology is about what goes on around the capsid, “the technology from Harvard is inside the capsid, so it’s more about the regulation,” Greenwald said. “So once we get the gene therapy to the right place, do we turn it on in the right cells at the right amount?”
PESCA can fine-tune the genetic payload of a therapy, “potentially avoiding off-target toxicities, and combined with our AAV capsids, can potentially avoid some of the high systemic dosage requirements as in some of the current gene therapies,” added Wang, who worked in Greenberg’s lab while she attended grad school at Harvard.
The toxicity from high doses of AAV-delivered gene therapies has been an ongoing safety concern. In a report published last year, FDA regulators noted that 35% of nearly 150 trials with AAV gene therapies reported serious adverse events, including deaths, related to the AAV-delivered treatments.
Apertura hopes to present its preclinical data later this year, Greenwald said.