rare diseases, says Peter Ginsberg, vice president, business & technology development at the North Carolina Biotechnology Center. The center has limited funding capabilities but it works with foundations to connect them to technologies or other resources that can help. Ginsberg says a nonprofit, typically started by a family touched by a rare disease, has the drive to advocate for promising early-stage research in a way a pharma company might not.
Yet foundations can fall short on the expertise needed to prepare a compound for pharmas. BioPontis will work with patient groups to identify promising research at universities; in many cases the foundations are already in close contact with leading researchers in their disease. Universities will benefit from the expertise of BioPontis scientists, who bring experience working with pharmaceutical companies. Where necessary, BioPontis may also provide funding.
With so many rare diseases needing treatments, BioPontis will choose where it goes by looking for the most promising science, says Richard Basile, co-founder and chief operating officer of BioPontis. The research must have enough testing behind it, including some animal studies, to suggest that with help, it could advance far enough to start clinical trials. That’s a point where a pharma company could be interested in taking a look at licensing the technology, Basile says.
Basile has held several executive positions at Bayer (XETRA: [[ticker:BAYN]]) and was later the first CEO of blood therapeutics startup Hemocellular Therapeutics, which is now the Research Triangle Park, NC, company Entegrion.
Handelin, for her part, views rare diseases through the lens of a geneticist. She founded the DNA testing laboratory at Integrated Genetics, which is now Genzyme, part of Sanofi (NYSE: [[ticker:SNY]]). She later co-founded gene therapy company Genovo. BioPontis’s board of directors also includes Marlene Haffner, who served 20 years as director of the FDA’s Office of Orphan Products Development, and Eric Tambuyzer, whose experience at Genzyme includes work in Europe on orphan products regulation.
BioPontis took its first run at turning university research into viable drug candidates several years ago as a venture fund. Handelin says the for-profit model at the time was to invest in a portfolio of projects, not companies. BioPontis eventually lined up 10 university partners around the country, which would work with BioPontis to advance academic research far enough to attract pharmaceutical company interest. Securities filings show that a 2012 fundraiser targeting $60 million raised just $7 million. Handelin now says neither the industry nor the investment community was ready for that model.
Two years later, a refocused BioPontis operates as a nonprofit focusing exclusively on rare diseases—changes Handelin believes will help the organization find an audience. BioPontis isn’t the only one embracing a nonprofit approach. For example, Bridges to Cures, a Wisconsin nonprofit, formed earlier this year to mentor and fund university researchers spinning out new companies developing promising new drugs.
Big pharma may have ceded rare disease drug discovery to smaller biotechs, but venture capital hasn’t completely forsaken rare disease investments. Earlier this year, Chiesi Ventures partnered with Durham, NC-based A.M. Pappas & Associates on a new fund focusing on rare disease drugs. Venture capital veteran Art Pappas, who manages the fund, told Xconomy that rare diseases and orphan drugs are
