Ultra-rare diseases often receive limited attention from the pharmaceutical industry due to the small number of patients affected. The RTW Foundation (RTWF) aims to bridge this gap by funding drug discovery projects focused on ultra-rare diseases, pairing this with free scientific and advisory services to patient advocacy groups seeking to develop therapies for their communities. As Director of Research at RTWF, Joe leads the development strategy and manages internal projects, ensuring the foundation’s mission to empower patient advocacy groups in driving therapeutic research. Joe believes that well-informed and engaged patient advocates are essential to advancing treatment options for ultra-rare diseases. To facilitate this, RTWF builds partnerships between advocacy groups, academic researchers, CROs, and biopharmaceutical companies, optimizing the pathway from discovery to development. Through its scientific advising services, RTWF provides sustained support to patient advocacy organizations, enabling them to lead therapeutic initiatives and establish their influence within their respective disease areas. Before joining RTWF, Joe was a staff scientist at Regeneron, where he led preclinical efforts in gene therapy development, AAV vector engineering, and other genetic medicine modalities. Prior to Regeneron, he served as a principal scientist at Pfizer, where he spearheaded immuno-oncology programs from discovery through IND application, working with a wide array of modalities, including lipid/polymeric nanoparticles, PROTACs, ADCs, antibodies, and small molecules. Joe holds a BS in human biology from Michigan State University, an MS in cellular & molecular biology from Eastern Michigan University, and a PhD in immunology and biomedical sciences from the Albert Einstein College of Medicine.