R&D On incentivisation and investment in rare disease R&D To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.
News Evidence builds behind Regeneron's deafness gene therapy Ten out of 11 children with congenital deafness treated with a gene therapy developed by Regeneron have seen "notable" improvements in hearing.
News Pfizer drops Beqvez, leaving its gene therapy cupboard bare Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.
News Troubled gene therapy player bluebird sold for a song At risk of defaulting on financial commitments, gene therapy firm bluebird bio has agreed to sell itself to private equity firms at a knockdown price.
R&D JP Morgan Week 2025 – Steve Pakola On the sidelines of JP Morgan in San Francisco, editor-in-chief Jonah Comstock caught up with Steven Pakola, REGENXBIO’s chief medical officer.
Market Access The value of a partner, globally and locally A new podcast looks at recent trends related to drug approvals, as well as challenges and opportunities both small and large pharma companies face