CF Foundation, Longwood team on new incubator for companies with cutting-edge CF treatments
Nine months after launching a $500 million hunt for a cure for cystic fibrosis, the Cystic Fibrosis Foundation said it will use a portion of those funds to do something it has never done before: help launch new companies.
The CF Foundation, whose venture philanthropy efforts helped fund Vertex’s line of powerful CF drugs, is teaming with Longwood Fund to create a CF incubator. The incubator will identify new companies with platforms or technologies that can be applied in the rare genetic condition. The partners can then finance early development in exchange for a commitment from the companies to focus on applications in cystic fibrosis.
CF Foundation has committed up to $20 million to the effort, although the companies selected could also receive funding through Longwood’s traditional early-stage VC efforts.
“This is new, so we’ll have to see how it plays out in practice,” Longwood partner David Steinberg told Endpoints News.
The idea for the incubator emerged last October at Harvard Medical School, where Longwood was holding their biannual meeting with non-profits and patient groups. At the time, the CF Foundation was preparing to launch its $500 million Path to a Cure initiative. Trikafta, the third of Vertex’s three small-molecule CF drugs, was about to hit market, but although the new drug meant 90% of CF patients could now be treated, that still left 10% untreated. Armed with royalties from Vertex, the Path initiative would try to back technologies that could treat that last group of patients and ultimately, perhaps, lead to a cure.
The CF Foundation had long backed academic work and particular programs at established companies — Vertex kept up their early CF work in part because the foundation was funding it — but it had never tried to found new ones. But such an early-stage approach made particular sense for what the foundation was now trying to accomplish, foundation CEO Mike Boyle told Endpoints.
Cystic fibrosis involves a malfunctioning transport protein in the lungs, called CFTR. Vertex’s drugs either help that protein fold in the right way or guide it to the right place. The remaining 10% of patients, though, either have a protein misfolded in such a way it doesn’t respond to existing small molecules or they simply don’t have the protein at all.
That meant patients would have to be treated with technologies that can target the gene: gene therapy, gene editing, and RNA drugs, among others. Boyle said the foundation is in early talks with 17 biotechs, mostly small, with such platforms. Many larger companies with these platforms are focused in other areas.
“One of the challenges right now is that there are easier targets for gene therapy than CF,” Boyle said.
It’s difficult to get gene therapy into the lungs, where the immune response is unique and cells turn over at a higher rate than in other parts of the body. But the incubator could allow Longwood and the foundation to steer companies that are just starting out to this challenge in exchange for the funding they need to get off the ground. Those startups would also have access to the foundation’s scientific advisors, their CF lab in Boston and their CF clinical trial network.
In addition to specific therapies, the incubator may also invest in drug delivery technology, such as exosomes, that could make delivering DNA, RNA or other types of drugs to the lungs.
There is precedent for the approach. The Gates Foundation’s venture arm has long invested in early-stage immunology platforms in exchange for a commitment from the companies to focus part of their work on infectious disease and not just cancer or autoimmune disorders.
Boyle said they’re hoping to launch companies within a couple years, but they’re also continuing to solicit partnerships with academic centers and more established biotechs. The incubator’s $20 million is just a small portion of Path’s $500 million vault.
“This is the beginning of the process,” Boyle said. “We are open for business.”
Social image: Mike Boyle, Cystic Fibrosis Foundation via YouTube