The BioPharm Brief: A Potency Rulebook, a CDMO Buyout, and a Nasal Antibody for MS

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today, we’re looking at new FDA expectations for cell and gene therapy potency, a major expansion of US manufacturing capacity, and mixed results for a nasal antibody in progressive MS.

First, FDA has finalized its guidance on potency assurance for cell and gene therapy products. It replaces the agency’s 2011 guidance on potency testing. Instead of treating potency as a single release test, FDA wants manufacturers to follow a risk-based potency assurance strategy across the full product lifecycle, from early IND submissions through BLA approval and postmarket verification. That strategy covers raw material variability, in-process testing, and assays confirming biological activity. The guidance also has dedicated sections on short-shelf-life products, viral vector gene therapies, and patient-specific therapies. FDA plans a webinar on December 10 to support implementation.

Next, Lonza has agreed to acquire Merck KGaA’s US contract development and manufacturing business. The deal brings in five sites and about 1,100 employees. The sites cover bioconjugation in St. Louis, high-potency ADC payload-linkers in Wisconsin, viral vector manufacturing in Carlsbad, California, and lipid nanoparticle and liposome production in Indianapolis. It takes Lonza’s US footprint from four sites to nine and fills a US bioconjugation gap the company flagged just weeks ago. The deal is expected to close in mid-2027, pending regulatory approvals.

And, Tiziana Life Sciences reported Phase 2a results for foralumab, a fully human anti-CD3 monoclonal antibody given as a nasal spray, in non-active secondary progressive MS. In the 48-patient INFORM-MS trial, the 50-microgram dose significantly reduced microglial activation in white matter on PET imaging compared with placebo. The 100-microgram dose did not reach statistical significance on that endpoint. No serious adverse events were related to the study drug. The nasal route is designed to stimulate regulatory T cells rather than broadly suppress the immune system. Tiziana plans to meet with FDA to discuss next steps.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

• Potency over the full lifecycle: FDA’s final guidance expects cell and gene therapy developers to build potency assurance from IND through postmarket, not just test at release.
• US capacity consolidation: Lonza’s purchase of Merck KGaA’s US CDMO business more than doubles its US network, adding ADC, viral vector, and LNP capabilities.
• A dose puzzle in progressive MS: Low-dose intranasal foralumab reduced brain inflammation on PET imaging, but the higher dose missed, which raises questions about the dose-response.