231: From IND to BLA: The Biologics CMC Decisions That Determine Regulatory Success with Henri Kornmann - Part 1

Seventy percent of FDA Complete Response Letters have a CMC root cause. Most of those failures trace back to decisions made years earlier. Decisions that felt minor at the time and proved impossible to fix later.Henri Kornmann has spent two decades making those decisions the right way. From junior CMC scientist at Merck to leading Ferring Pharmaceuticals' first gene therapy approval for bladder cancer, Henri has crossed between CMC development, GMP manufacturing, and due diligence across some of the industry's most complex programs. His conclusion: a CMC program is like building a house. Get the foundation wrong and no amount of late-stage effort will save you.In Part 1, Henri reveals the decisions that cannot be undone and how to get them right from the start.What you will learn:Evolution of cell bank technology and regulatory expectations (00:33)The impact of weak CMC foundations on late-stage failure (00:51)Lessons learned from Ferring’s gene therapy approval and CMC gap analysis (06:51)FDA statistics on CMC issues in INDs and response letters (08:07)Critical early decisions: cell bank clonality and proper storage practices (10:22)The importance of comprehensive raw material documentation (12:29)Early analytical characterization and discovering molecular “funkiness” before phase trials (13:41)Supply strategy for phase 2—why stability and batch knowledge matter (14:49)Introduction to critical quality attributes (CQA), process parameters, and quality-by-design principles (15:52)Common pitfalls in CQA identification and continued process verification (17:01)Smart insight:The therapies that reach patients aren't built on heroic late-stage rescues. They're built on disciplined early decisions: the right cell bank, the right analytics, the right documentation. Henri's message is unambiguous: there are CMC mistakes you can fix later, and there are CMC mistakes you cannot. Knowing the difference is the foundation of every successful biologics program.In Part 2, Henri walks through scale-up to commercial manufacturing, process validation stages 1 through 3, post-approval control strategy, and the project management and regulatory fluency that separate successful CMC leaders from the rest.If this topic resonates with you, here are a few related episodes where we dive deeper into building strong CMC foundations and avoiding costly development mistakes:Episodes 199 - 200: Mastering Quality by Design: From Product Failures to Commercial Success in Biologics CMC DevelopmentEpisodes 189 - 190: Why Smart Biotech Founders Plan CMC First (While Competitors Burn Cash Later)Episodes 23 - 24: Strategies for Success: Master CMC Development with Gene LeeEpisodes 57 - 58: Crafting a Solid CMC Strategy: Key Factors and Common Pitfalls with Matthias MüllnerConnect with Henri Kornmann:LinkedIn: www.linkedin.com/in/henri-kornmann-9b6869Next step:Need fast CMC guidance? → Get rapid CMC decision support hereSupport the show