The ‘Haves And Have-Nots’ Are Becoming Clearer In Cell And Gene Therapy

While 2023 saw several cell and gene therapy approvals that were genuine scientific and clinical breakthroughs, many smaller companies in the sector bore the brunt of the ongoing biopharma downturn.

Most notable among the sector’s milestones were the US Food and Drug Administration’s accelerated approval of Sarepta’s Duchenne muscular dystrophy gene therapy Elvidys (delandistrogene moxeparvovec) and two potentially curative sickle cell disease (SCD) treatments – Vertex and CRISPR