Develop Potency Assays Early for ATMP Success

FDA has reportedly shelved its Commissioner’s National Priority Voucher pilot and paused publishing complete response letters, Zai Lab’s IL-13/IL-31Rα bispecific ZL-1503 shows a 40- to 50-day half-life that points to dosing every 12 weeks, and Wave Life Sciences reports 1-year INLIGHT data for its INHBE-silencing siRNA WVE-007 in obesity.

Merck's Dr Dave Maraldo outlines 3 principles for placing manufacturing capacity and how end-to-end ADC supply chains could shift platform thinking.

Donidalorsen sustained a 95% reduction in hereditary angioedema attacks over 2 years, and patients switching from other prophylaxis saw a 71% drop.

FDA has effectively paused its Commissioner’s National Priority Voucher pilot and appears to have halted new complete response letter postings, according to reports. HHS says work on the program is ongoing.

A single 240 mg dose of WVE-007 cut visceral fat while preserving lean mass for 1 year, as Activin E fell by up to 88% across INLIGHT dose groups.

A single dose of Zai Lab’s long-acting bispecific ZL-1503 produced at least 90% inhibition of an atopic dermatitis signaling biomarker through Week 12 in healthy volunteers. Its 40- to 50-day half-life supports a target of dosing every 12 weeks.

In the first part of a two-part interview, Paz Vellanki of Precision for Medicine explains what sponsors should bring to early FDA meetings and how manufacturing and assay readiness can delay rare disease trials.

FDA clears the IND for Biolojic Design’s BD200, an AI-designed Trop2/Nectin-4 multibody ADC; Blackstone Life Sciences agrees to provide Pfizer up to $1.5 billion to develop its monthly GLP-1 receptor agonist berobenatide; and enGene Therapeutics plans a year-end BLA for detalimogene voraplasmid in BCG-unresponsive non-muscle invasive bladder cancer.