Sickle Cell Drug Development Gains Momentum Across Disease Spectrum

Transplants An Alternative To Genetic Medicines For Some

The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options.

Vertex and bluebird booths at ASH 2023 — Vertex and bluebird presented longer-term data at ASH • Source: Scrip

The approval of the first genetic medicines for sickle cell disease (SCD) on the eve of the American Society of Hematology (ASH) annual meeting set off a celebratory feel for the conference, held 9-12 December in San Diego, and drew increased attention to data presented at ASH for multiple novel drug candidates for the disease.

Key Takeaways

The pipeline of new medicines for sickle cell disease is growing rapidly as evidenced by data presented for gene-editing medicines, gene therapy and small molecules at ASH.

The FDA approved Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s CRISPR/Cas9 gene editing medicine Casgevy (exagamglogene-autotemcel, exa-cel) and bluebird...