Importance of Data Reproducibility for Development of Safe Gene Therapies

FDA grants Ultomiris accelerated approval for primary immunoglobulin A nephropathy, a pharma funding roundup covers Alcon’s $550 million acquisition of Neurotech and AI Proteins’ collaboration with Astellas, and Incubate’s John Stanford on which therapeutic areas MFN deals might hit hardest.

Alcon is acquiring Neurotech Pharmaceuticals to add the only FDA-approved MacTel cell therapy to its retina portfolio, while AI Proteins has enters a collaboration with Astellas to design novel miniprotein therapeutics.

FDA has granted accelerated approval to Ultomiris based on Phase III data showing a 43.4% placebo-adjusted reduction at 34 weeks.

A new ICER and West Health report finds median drug launch prices climbed to $257,000, the FDA approves GSK’s Hibsago as the first functional-cure therapy for chronic hepatitis B, and CSL names Deborah Waterhouse as CEO and managing director.

Median net launch prices for new drugs climbed between 2022 and 2025 to $257,000, according to a new ICER and West Health report that also found persistent coverage and access barriers for patients.

The FDA approved GSK's bepirovirsen for adults with chronic hepatitis B on nucleoside therapy, based on phase 3 trials showing 19% functional cure.

Deborah Waterhouse is leaving ViiV Healthcare to become CEO and managing director of CSL.

Incubate’s John Stanford on why MFN’s first casualty is the moonshot — and why cancer, Alzheimer’s, and rare disease bear the cost.