Ultragenyx Pharmaceutical Inc. (RARE) Presents at Morgan

Company Overview - Ultragenyx has been dedicated to developing new treatments for rare diseases for 15 years, focusing on areas with high unmet medical needs [2] - The company has a diverse platform approach, including enzyme replacement therapy with Mepsevii, small molecule therapy with Dojolvi, and gene therapy programs [2] Product Pipeline - The foundational product, Mepsevii, is an enzyme replacement therapy, followed by Dojolvi as a small molecule treatment [2] - Recent advancements include the Angelman program utilizing antisense oligonucleotides (ASO) and an antibody program for osteogenesis imperfecta (OI) [2]