On a trip to Basel, Switzerland, Philip catches up with Stefano Portolano, CEO of the European firm Azafaros. They chat about Azafaros’ massive $147M Series B round in May 2025, which is being used to develop a small molecule treatment for rare diseases known as lysosomal storage disorders.Stefano also explains how rare diseases went in and out of fashion with big pharma, the revival of small molecule drugs relative to gene therapies and biologics, and his extensive experience in big biotech firms.⭐️ ABOUT THE SPEAKERWith more than 20 years of experience in orphan diseases under his belt, Stefano Portolano joined Azafaros in 2021. He previously served as Vice President, Strategy & Commercial Operations at Celgene and Head of Europe at Ultragenyx Pharmaceuticals.Stefano first received his Doctor of Medicine from Università degli Studi di Napoli ‘Federico II,’ Italy and carried out research in autoimmunity as an adjunct assistant professor of Medicine at the University of California, San Francisco, USA.🔗 LINKS MENTIONED- Azafaros website - https://www.azafaros.com/- Azafaros Series B round - https://www.azafaros.com/news/azafaros-secures-132m-in-oversubscribed-series-b-financing-to-advance-phase-3-clinical-programs-of-innovative-therapies-in-lysosomal-storage-disorders/l211c14- Joris Pezzini, Alira Health | Hottest Biotech Insights - https://flot.bio/episode/joris-pezzini-alira-health-biotech-trend/- Marc de Garidel, Abivax | How to make two Billion-dollar exits (“smoothly”) - https://flot.bio/episode/marc-de-garidel-abivax-exit/📜 TRANSCRIPTRead the full transcript here: https://flot.bio/episode/stefano-portolano-azafaros-rare-disease/💸 DONATEWe welcome donations from individuals and businesses to support our podcast and allow us to create even more content: https://www.paypal.me/PhilipHemme⭐️ SPONSORSHIPReady to amplify your brand to influential leaders in biotech and biopharma? Contact nick@flot.bio and unlock sponsorship opportunities in our podcasts!🫶 FOLLOW US- Newsletter: http://eepurl.com/h_fnmH- LinkedIn: https://www.linkedin.com/company/flot-bio/- X (Twitter): https://x.com/FlotBio🙏 LIKE/FOLLOW/REVIEWDid you enjoy the episode? Please hit the like and subscribe button. Also, please let me know your thoughts in the comments below.🎙️ ABOUT FLOT.BIOFlot.bio is a show to watch the best Europeans in biotech to help you grow. Hosted by Philip Hemme. 20k monthly views. Free.⏰ TIMESTAMPS- [00:00:00] Intro- [00:01:45] Azafaros’ origin and $147M B round- [00:04:39] Developing nizubaglustat- [00:11:17] Competition in Niemann-Pick disease type C- [00:18:44] The small molecule revival- [00:24:29] Keeping options open- [00:27:23] Europe’s growing role in biotech investments- [00:30:12] The rare disease fashion- [00:51:20] Getting personal with Stefano Portolano- [01:11:11] Quickfire