Voxelotor

Recent FDA approvals have expanded treatment options for sickle cell disease beyond hydroxyurea, including L-glutamine, crizanlizumab, and two gene therapies, marking significant progress in disease management.

Pfizer has voluntarily withdrawn its sickle cell disease therapy Oxbryta (voxelotor) from all global markets after discovering an imbalance in deaths during clinical trials.

Lilly's Jaypirca demonstrated superior efficacy in relapsed/refractory CLL patients previously treated with BTK inhibitors, nearly doubling the time to disease progression compared to older regimens.

• CRISPR Therapeutics' stock has declined despite the FDA and EU approvals of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • The initial launch of Casgevy, developed with Vertex Pharmaceuticals, has been slower than anticipated due to the complexity of the treatment and conditioning regimen. • CRISPR Therapeutics is advancing five other clinical-stage therapy candidates, including CTX112 for blood cancers, with promising early trial results. • Despite current losses, CRISPR Therapeutics maintains a strong cash position, providing a runway for Casgevy sales growth and pipeline development.

• CRISPR Therapeutics' stock has declined despite FDA and EU approvals of Casgevy for sickle cell disease and transfusion-dependent beta thalassemia. • Casgevy's initial sales have been slow due to the complexity of the therapy and a conditioning regimen that carries risks. • Promising Phase 1 trial results for CTX112, a blood-cancer treatment, and a strong cash position offer potential for future growth. • Analysts suggest caution due to the high-risk nature of the stock, advising investors to consider their risk tolerance.