Ultragenyx Pharmaceutical Inc. (RARE) Presents at Morgan


Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Morgan Stanley 23rd Annual Global Healthcare Conference September 9, 2025 4:05 PM EDT

Company Participants

Eric Crombez – Chief Medical Officer & Executive VP

Conference Call Participants

Maxwell Skor – Morgan Stanley, Research Division

Presentation

Maxwell Skor
Equity Analyst

Great. Thank you, everyone, for joining. I’m Max Skor, biotech analysts with Morgan Stanley. And I’m happy to be hosting this session with Ultragenyx, Eric Crombez, Chief Medical Officer. Thank you very much for joining us today. And I just wanted to briefly touch on important disclosures. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. So great. Eric, if you don’t mind I’ll open the — if you’d like to open up the conversation, anything you’d like to highlight?

Eric Crombez
Chief Medical Officer & Executive VP

Yes. So great. I appreciate the opportunity. I appreciate the time. Just, I guess, a very just brief introduction on Ultragenyx. So we’ve been around for 15 years, fully dedicated to the development of new treatments for patients suffering from rare diseases where there really is very high unmet medical need. Foundational to the company was Mepsevii in enzyme replacement therapy and then moving on to Dojolvi, a small molecule and then really rounding out with our gene therapy programs and then more recently, our Angelman program as an ASO, OI as an antibody. So really looking at a diversity of platforms there. So again, we can really focus on these rare diseases, figuring out the right way to go after them and then pursuing those into a commercialized setting.

Question-and-Answer Session

Maxwell Skor
Morgan Stanley, Research Division

Great. So Ultragenyx is really balancing near-term growth with a broad pipeline. So what 3



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