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Sickle Cell Drug Development Gains Momentum Across Disease Spectrum

Transplants An Alternative To Genetic Medicines For Some

 
• By Mandy Jackson

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...

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