Emerging Therapies 2023

Gene-corrected nula-cel freed all 3 RESTORE patients from transfusions, with sickle hemoglobin below 20% in the 2 followed for over 6 months.

Beam Therapeutics’ base-edited cell therapy risto-cel meets its primary endpoint in the BEACON sickle cell disease trial, Amgen’s CD40L antagonist dazodalibep reduces systemic disease activity in the phase 3 OASIZ-301 Sjögren’s disease trial, and ImvivaBio’s allogeneic CD19/BCMA CAR-T CTA313 sustains lupus remission through 12 months.

Matt Truppo, Richard Jaenisch, and Brian Drapeau examine AI's impact on drug success, human oversight, data governance, and biopharma's future.

ProBio grants AbTis exclusive global rights to a discovery-stage oncology antibody for development as a site-specific antibody-drug conjugate.

ImvivaBio’s off-the-shelf CD19/BCMA CAR-T cell therapy CTA313 kept 88% of evaluable patients with refractory lupus in DORIS remission at Month 12, according to longer-term phase 1 data presented at ACR Convergence 2026.

Beam’s base-edited cell therapy met its primary endpoint in the BEACON trial, and a U.S. biologics license application is planned as early as year-end.

Full phase 3 OASIZ-301 results show both dosing regimens of Amgen’s CD40L antagonist fusion protein dazodalibep reduced systemic disease activity in Sjögren’s disease at Week 48. The every-four-weeks dose also improved patient-reported dryness.

AstraZeneca acquired preclinical biotech Omniose, adding a bioconjugation platform for conjugate vaccines against serious bacterial infections.