The Challenge of Finding the Unknown

Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.

The FDA approved Roche's satralizumab (Enspryng), an IL-6 receptor-targeting monoclonal antibody, for adults with thyroid eye disease based on the Phase 3 SatraGO-1 and SatraGO-2 trials, giving patients the first at-home subcutaneous treatment option.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.

Nathan Edwards of Arya Consulting moderates a discussion with Elisabeth Gardiner of Tevard Biosciences and Donny Xu of the RTW Institute about rare disease treatment development and navigating the FDA’s Rare Pediatric Disease Priority Review Voucher Program.

The FDA has accepted Pierre Fabre Pharmaceuticals’ resubmitted BLA for tabelecleucel, an allogeneic T-cell immunotherapy for EBV+ post-transplant lymphoproliferative disease. The FDA’s target action date is March 31, 2027.

MT027, T-MAXIMUM's B7-H3 CAR-T therapy, earns a second FDA-cleared phase 2 trial, now testing lung and breast cancer brain metastases.

Genglycos, the gene therapy that earned the voucher, will fund new Ultragenyx therapies for other rare and ultra-rare diseases.