INTERPHEX 2025: Advancing Treatments for Ultra-Rare Diseases

According to Joe Katakowski, director of Research at the RTW Foundation, parents of children with rare diseases are the driving force behind the development of treatments for ultra-rare diseases. “[RTW Foundation has] funded a lot of academic research grants, but the missing component was the embedment of these parents and patients in those efforts. So, we've restructured our granting approach now to make the rare disease parents and foundations, the owners, and the accountable party to the academic researchers,” Katakowski explains. “And we think this model is going to be far more successful than just funding academic researchers directly. Even though our current model still gets funding to the academic researchers, it makes those rare parents and the rare disease foundations the central player in the space. We empower them with knowledge related to drug development, and we empower them with funding and resources, and we engage with them for a prolonged period. It's not just a quick boot camp; it's not a weekend course. We're working with these foundations, oftentimes in excess of 16 months, perhaps two years, to really help them solve all the challenges that will come up inevitably during their drug development campaign.”

Katakowski and Deanna Portero, VP, Partnerships & Innovation, Orphan Therapeutics Accelerator, gave a presentation titled, “A Novel Pathway for Ultra-Rare Disease Treatment Development, Commercialization and Access” at INTERPHEX 2025, in which they discussed approaches to advancing treatments for ultra-rare diseases. PharmTech Group sat down with Katakowski to discuss how organizations like the RTW Foundation work to advance the development of treatments for rare diseases.

INTERPHEX 2025 was held in New York City from April 1–3.

Click the video above to watch part one of PharmTech’s interview with Joe Katakowski.

Watch Part One of the interview here.

About the speaker

Joe Katakowski is director of Research at the RTW Foundation