BioPharm Weekly News Roundup—Week of Sept. 29, 2025

Welcome to the BioPharm International® weekly news roundup for the week of Sept. 29, 2025. In this new video feature, the editors will highlight each week’s industry news in an easy-to-consume, fun format. New roundups will drop every Friday, so be sure to come back each week. And be sure to check the links below for more in-depth coverage of each story.

AAVantgarde Bio’s dual-vector gene therapy AAVB-039 gained FDA orphan drug designation and UK Medicines and Healthcare products Regulatory Agency clinical trial authorization approval, accelerating complex Stargardt disease treatment.

FDA guidance promotes adaptive, Bayesian, and single-arm clinical trial designs for cell and gene therapy (CGT) development in rare diseases.

New FDA guidance mandates long-term CGT surveillance, leveraging real-world data, registries, and AI for data quality.

FDA RMAT guidance expedites regenerative therapies, including VeonGen Therapeutics’ drug, while demanding rigorous CMC standards.

Novartis expands its immunology pipeline with Tourmaline Bio acquisition and tests Direct-to-Patient model for Cosentyx distribution.

Naobios and Olon France partnered to develop a custom process-specific HCP ELISA kit for a viral vaccine, ensuring reliable biopharma analytics.

Integra Therapeutics successfully used generative AI to design synthetic proteins, enhancing gene-editing tools beyond natural capabilities.

Pfizer is the first major firm to agree to White House MFN pricing, boosting US investment and lowering drug costs.