Rare Ventures Launches in Pittsburgh with $25 Milllion in Backing

Rare Daily Staff

A new Pittsburgh-based initiative backed by up to $25 million in philanthropic funding aims to speed the development of treatments for rare diseases, which collectively affect hundreds of millions of people worldwide.

Called Rare Ventures, the effort brings together Carnegie Mellon University, the University of Pittsburgh, UPMC Children’s Hospital of Pittsburgh, Stanford Medicine, biotechnology company ElevateBio and the EB Research Partnership, a nonprofit founded by Jill Vedder and Pearl Jam singer Eddie Vedder.

The Richard King Mellon Foundation has committed up to $25 million to launch the program, which organizers describe as a venture-philanthropy platform. Its goal is to link scientific research, artificial intelligence, clinical care, drug development and manufacturing in a more coordinated effort to move potential treatments toward patients.

“Rare diseases represent one of the greatest unmet challenges in medicine,” said Sam Reiman, director and trustee of the Richard King Mellon Foundation. “Rare Ventures brings together an exceptional group of partners around a bold vision.”

Rare Ventures builds on the EB Research Partnership’s experience funding rare-disease research. The organization said it has supported more than 180 research projects in 22 countries over the past 14 years and helped expand the number of active clinical trials in epidermolysis bullosa from two to more than 50. It also said three FDA-approved therapies for the condition have reached patients in the past three years.

The partnership plans to reinvest potential proceeds from successful treatments into new research, creating a continuing source of funding for additional rare-disease programs.

Carnegie Mellon will serve as the initiative’s technology center, using its strengths in artificial intelligence, computational biology, chemistry and automated science. University researchers will work with clinicians at UPMC and Stanford Medicine to identify disease targets, design potential treatments and develop better ways to deliver therapies to the right cells in the body.

The program’s initial focus will include epidermolysis bullosa, a rare genetic disorder that causes extremely fragile, blistering skin. The EB Research Partnership was created to fund research into the condition.

Organizers said six other rare diseases identified by UPMC partners will also become priorities during the initiative’s first three years, though they did not name the conditions.

Michael Hund, CEO of the EB Research Partnership and a co-founder of Rare Ventures, said the project is intended to address challenges that often slow rare-disease research, including small patient populations, fragmented data and the high cost of testing and manufacturing treatments.

“By bringing together artificial intelligence, venture philanthropy, therapeutic development and world-class institutions, we are building a new model designed to accelerate therapies and create hope for millions of families around the world,” Hund said.