This nonprofit is building the ecosystem to cure epidermolysis bullosa

Epidermolysis bullosa, a condition characterized by skin so fragile that even gentle contact results in blisters and wounds, has long remained incurable and received minimal serious pharmaceutical investment. Martin Steiner, Managing Director of DEBRA Research, a non-profit organization dedicated to curing EB, explains that the organization does not conduct lab research or clinical trials but instead builds the ecosystem necessary for drug development.

This includes funding research, investing in biotech companies, establishing shared infrastructure, and forging connections among relevant parties. DEBRA Research's efforts have resulted in a growing portfolio of investments, dedicated clinical trial infrastructure, and a strategic position in key deals benefiting patients who have endured long waits.

Steiner elaborates on what it means to live with EB, DEBRA Research's role in drug development, their investment strategies, the partnership between Replay and LEO Pharma, and the challenges of fostering collaboration in rare disease research. He also discusses gene therapy and read-through molecules as potential therapeutic approaches. Finally, he explores the possibility of replicating DEBRA Research's model for other rare diseases.