Pearl Jam frontman Eddie Vedder is backing Rare Ventures, a Pittsburgh-based start-up created to find cures for rare diseases. (Louis B. Ruediger | TribLive)
A Pittsburgh-based coalition of medical experts, industry veterans and philanthropists — with the backing of star grunge musician Eddie Vedder of the band Pearl Jam — plans to fight rare diseases as part of a new start-up
The project, Rare Ventures, is backed by an investment from the Richard King Mellon Foundation of up to $25 million, the Strip District-based philanthropic organization announced Wednesday
Collaborating with other local players like the University of Pittsburgh, UPMC Children’s <a href="https://healthylife7.com/cone-health-moves-forward-with-plans-to-build-mebane-hospital/” title=”Cone Health moves forward with plans to build Mebane hospital”>Hospital of Pittsburgh and Carnegie Mellon University, Rare Ventures will seek to accelerate funding and research of rare diseases in an effort to rapidly develop therapies for illnesses that collectively affect hundreds of millions
The project’s model is inspired by the EB Research Partnership, a research and investment organization founded by Eddie Vedder and his wife Jill to fast-track development of treatments for epidermolysis bullosa, a rare genetic skin disease
The project has raised more than $80 million, funding numerous clinical trials, including two genetic therapies that recently earned FDA approval, according to Rare Ventures co-founder Michael Hund, who also heads up EB Research Partnership
Rare Ventures will use its same financial model, Hund said
That involves seeking philanthropic support — in this case from the RK Mellon Foundation — to fund the most promising disease research projects in exchange for a financial stake in the work. Rare Ventures will then reinvest its returns from those investments in further research, Hund said
“The ultimate goal is to create a self-sustaining model,” Hund told TribLive Wednesday
To unlock all $25 million from the RK Mellon Foundation, Rare Ventures will have to hit a series of benchmarks over the next three years, which Hund declined to outline
Globally, more than 400 million people live with a rare disease, yet around 95% of known rare diseases have no treatment, according to a press release from the foundation
Hund called the situation “perhaps the largest unmet need in global healthcare.”
But with the combined efforts of Pittsburgh’s medical and science communities — in addition to collaboration with Stanford University
“We’re building an engine for rare disease,” he said
Much of that work will take place at UPMC Children’s Hospital, where Dr. Terence Dermody said doctors will research the cause of rare diseases and develop cures
Rare disease isn’t something new for the hospital, but the rapid pace of the Rare Ventures collaborative could be a game-changer, said Dermody, who serves as chair of pediatrics at the University of Pittsburgh School of Medicine and physician-in-chief and scientific director at Children’s
“We have experience in these areas,” Dermody told TribLive. “We’re sort of an anchor part of this initiative.”
If done right, the formula created in Pittsburgh with Rare Ventures could create a model that could be replicated to treat other illnesses elsewhere, Dermody said
“We’re going to bring cures to these kids,” he said. “I’m confident of it.”
Rare Ventures’ work will begin on seven rare diseases, Hund said
Those include epidermolysis bullosa — the focus of Vedder’s foundation — VLCAD deficiency, PACS1 syndrome, autosomal dominant leukodystrophy with autonomic disease, a type of retinitis pigmentosa, oculocutaneous albinism type 1A, and pyruvate dehydrogenase deficiency
Though Carnegie Mellon won’t be involved in direct clinical trials, Barbara Shinn-Cunningham said the school’s experts will work to provide technology that helps guide research
That includes use of artificial intelligence to recognize patterns in vast data sets — like a human genome — that a person may not be able to pick out, said Shinn-Cunningham, dean of CMU’s Mellon College of Science
“This is an amazing opportunity to do science in the world,” she said
According to RK Mellon, AI will be a fundamental part of Rare Ventures
The project will use a platform called Curator, described as an “AI-powered, genomics-driven patient platform developed through collaborations with leading technology and academic partners.”
It will provide patients with information on specialists, trials and research, while offering data to researchers and industry developers to speed up trials, the release said
The placement of the project in Pittsburgh signals a robust and growing biotechnology sector that could further help the region, Shinn-Cunningham said
“Discovery that changes the world starts in Pittsburgh,” Dermody said, citing Jonas Salk’s famed development of the world’s first polio vaccine in the city
Based in New York City, Hund said he’ll travel between there and Pittsburgh
For now, he said Rare Ventures will likely make its home in the spaces of its various local partners, but it could eventually expand to its own office
Hund said the initiative will soon hire three to five full-time staff members
About the Writer
James Engel is a TribLive staff writer. He can be reached at jengel@triblive.com
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