Company Spotlight

Pittsburgh-based Avista Therapeutics is taking on one of gene therapy’s toughest challenges: how to get genetic medicine to the right cells—accurately, safely and effectively. 

Spun out of UPMC Enterprises in 2021, Avista is focused initially on rare, inherited retinal diseases—starting with X-linked retinoschisis (XLRS), a condition that causes retinal degeneration in young boys. The company aims to file its first Investigational New Drug application (IND) with the FDA in early 2026.

“Our goal is to tackle blinding diseases that have no cures today,” said cofounder and chief scientific officer Leah Byrne, Ph.D. “We’re developing therapies for people who currently have no options.”

At the heart of Avista’s approach is scAAVengr, a next-gen AAV (adeno-associated virus) engineering platform. Traditional AAV vector development can take years, but Avista’s platform—driven by computational tools and single-cell analytics—screens millions of viral vector candidates in parallel. This speeds up discovery while raising the bar for what makes a vector viable. 

“We don’t just look at whether a vector gets into a cell—we track whether it actually reaches the nucleus and expresses the therapeutic gene,” said Byrne. “That’s a much higher standard.”

This platform is what caught the attention of Roche, which signed a $7.5 million collaboration deal with Avista in 2022. The partnership focuses on applying Avista’s platform to develop gene therapies for other inherited retinal diseases.

Though the initial focus is ocular, Avista’s team believes its innovative platform has broader applications.

“We’re building what we think are best-in-class vectors for retinal gene therapy, but we’re already exploring how to apply our approach to other tissues,” Byrne said.

Deep Roots in Pittsburgh

Avista traces its roots to Dr. Byrne’s lab at the University of Pittsburgh, where early funding from UPMC Enterprises supported development of the scAAVengr platform. The company was co-founded by Byrne, José-Alain Sahel, M.D., and Paul Sieving, M.D., Ph.D. Rob Lin, Ph.D., who helped launch Avista from UPMC Enterprises, now serves as CEO. Sahel, who directs the UPMC Vision Institute, is one of the world’s leading ophthalmologists and received the Wolf Prize in Medicine in 2024 for his pioneering work in vision restoration. 

Pittsburgh’s life sciences ecosystem continues to power Avista’s growth.

“Pittsburgh has been essential to our success,” said Byrne. “We’ve hired incredible talent from the University of Pittsburgh and Carnegie Mellon, and we’ve found that people here stay because they’re invested in the region.”

Avista is betting on its cutting-edge science and local roots to help it achieve its long-term vision of bringing new hope to patients. After all, it’s in the company’s name: “Avista” means “to see far” in Spanish.