It is estimated that 25,000-50,000 Americans have a debilitating skin disorder called epidermolysis bullosa (EB). EB is typically present at birth, as children with EB are missing the essential proteins that bind their skin together. Some children are born with missing areas of skin that leave large wounds that never heal. For children with EB, the basic activities of life—walking, eating, playing, sleeping—can be painful, if not impossible.

Currently, there are no treatments that target the underlying causes of EB. Treatments on the market today instead focus on disease management, prevention of complications, and prevention of the formation of new blisters.

The encouraging news for children with EB and their families is that scientists know the causes of EB and have a growing knowledge of how to address it. However, treatments or a cure for EB have been elusive primarily due to a lack of funding for research.

Like so many disease organizations, EB Research Partnership (EBRP)—the largest global nonprofit organization dedicated to treating and curing EB—is stepping in to fill the research funding gap. EBRP is among a growing group of nonprofits that have pioneered innovative models to further advance their missions to bring treatments and cures to patients.

EBRP employs a venture philanthropy model under which it participates in the commercial success of the research projects it funds. When EBRP provides a grant to a research project, it retains the right to share in financial value generated by a successful project; these proceeds are then used to fund more EB research. This model has enabled EBRP to fund larger projects than it would otherwise.

In 2019, for example, EBRP funded the formation of a new company: Wings Therapeutics. Wings is focused on advancing the clinical development of exon skipping treatments for EB. Its lead drug, QR-313, was incubated at ProQR Therapeutics. This publicly-traded rare disease company sought a strategic partner to continue the development of its drug for EB so that it could focus on its pipeline of ophthalmology drugs. EBRP provided the capital and secured the management team to form Wings. Under the terms of the deal, ProQR retained a minority stake in the new company and is eligible for milestone and royalty payments.

When priorities within a drug company change, there is a question about what happens to the drugs that are no longer a strategic fit. In this case, a strong relationship between the CEO of ProQR and the chairman of EBRP created the path for a transaction. By providing transparency into its corporate objectives, proactively seeking a strong partner to advance the drug’s development, and consummating a transaction on mutually beneficial terms, ProQR acted in the best interests of the community that it intended to help. EBRP garnered the resources needed to agree to such a transaction within a two-week window.

To the extent drug companies have a line of sight into the drugs that will be discontinued as part of strategic or business shifts, companies should consider how they can facilitate greater access to those drugs, as ProQR did. While there could be barriers to doing so, EBRP and ProQR’s partnership illustrates that obstacles are surmountable and solutions can be reached that meet all constituents’ objectives.

At FasterCures, we’re acutely aware of the human cost of these delays. Through our BRIDGE initiative, we are exploring ways to enable more transactions for deprioritized drugs so that the promise of these assets can be fully explored. We need your help—if you have a similar story, share it with us!