Following Luxturna and slate of eye disease approvals, Roche adds another ophthalmology partner for AAV gene therapies
Roche’s ophthalmology ambitions have grown steadily in recent years with two eye disease approvals in the past year and the acquisition of Spark and its Luxturna gene therapy in 2019, and the Big Pharma is doubling down in the space with another gene therapy tie-up.
Instead of Philadelphia, Roche has headed to Pittsburgh for this deal, linking arms with a University of Pittsburgh Medical Center spinout developing new AAV gene therapy vectors that the biotech’s CEO hopes will address limitations of existing treatments, including Luxturna. The deal also adds to Roche’s deep ophthalmology pipeline, which includes late-stage work attempting to secure more indications for recently approved Susvimo and Vabysmo.
While the upfront payment is relatively small at $7.5 million, the fledgling Avista Therapeutics can secure more than $1 billion in back-end payments if the biotech eclipses certain milestones and can get its gene therapies across the finish line, the companies announced Tuesday morning. The company has been seeded with $10 million from UPMC Enterprises to date and anticipates going to investors in mid-2023 to raise a Series A, CEO Rob Lin tells Endpoints News.
“Pretty good to be sitting out the fundraising market at the present moment,” Lin, who is also a VP at UPMC Enterprises, said. “Between the upfront monies from the Roche partnership and the seed round, we’re pretty good for now.”
Leah ByrneDerived from avistar, which means “to catch sight of” in Spanish and Portuguese, Avista is built upon the work of UPMC researchers Leah Byrne, José-Alain Sahel and Paul Sieving, who previously led the National Eye Institute. Byrne’s PhD work at UC-Berkeley led to the creation of vector capsid AAV.7m8, which was licensed to Adverum Biotechnologies, a biotech that has gone through a series of hurdles with its ocular disease gene therapies. Sahel leads UPMC’s Eye Center and has co-founded a bevy of biotechs, including SparingVision, which expects to file an IND on its first inherited retinal disease gene therapy by the end of this year, said Lin, who is a board observer.
Avista’s computationally-driven in vivo platform, aptly dubbed scAAVengr, aims to create new cell-specific AAVs. In the Roche partnership, Avista will deploy the single-cell engineering platform to create intravitreal AAV capsids. The Big Pharma can then choose which ones it wants to license and will be responsible for all preclinical, clinical and commercialization work that might come out of the gene therapy programs using those capsids. Avista’s internal pipeline will remain distinct from the work with its Swiss partner.
The goal is to “open the window of diseases” that gene therapies can treat, Lin said.
“One of the things we saw was that subretinal delivery involves sticking a hole in the retina and creating a little bubble there to deliver the gene therapy product, and while it’s been shown to be effective in a product like Luxturna, the surgical complications of that make it limiting both in terms of how many people can be treated with that but also with the types of diseases that perhaps maybe don’t want to stick a hole in the retina,” the CEO said.
The three-employee biotech will grow to about 10 employees next year as Avista searches for additional C-suite members and begins the hunt for additional financing, Lin said. Meanwhile, he said, Avista is heads down on delivering on its Roche agreement, aiming to have “a number of vectors” in the next 18 months that the pharma can opt into.
Barbara Lueckel“Our scientists were familiar with the innovative work by Leah Byrne, and when the spin-out of Avista was being contemplated, we were eager to be Avista’s first partner and develop enabling capsids to address debilitating ocular diseases together,” said Barbara Lueckel, head of research tech at Roche Pharma Partnering, in a statement.
Avista is among a slate of gene therapy players looking at diseases in the eye. Along with Adverum and SparingVision, the field also includes Coave Therapeutics and Opus Genetics, founded by Luxturna inventor Jean Bennett.
