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.
- Recent approvals for Casgevy and Lyfgenia provide new options for severe SCD, but leave room for oral drugs that may be more accessible to the broader patient population
The FDA approved Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s CRISPR/Cas9 gene editing medicine Casgevy (exagamglogene-autotemcel, exa-cel) and bluebird bio inc