Crizanlizumab

CRISPR Therapeutics stock is down 47% since March due to slow Casgevy launch, but it has five other therapy candidates in clinical-stage testing. Despite slow sales, Casgevy's potential and a $1.9 billion cash reserve make CRISPR Therapeutics a risky but potentially lucrative investment.

Beam Therapeutics reported a patient death in a Phase I/II trial for BEAM-101, attributing it to the preconditioning regimen, not the treatment. The FDA allowed the trial to continue without changes. Researchers seek less toxic alternatives to busulfan, crucial in cell and gene therapy protocols.

Pfizer's withdrawal of Oxbryta from global markets due to increased risk of deaths and complications has left the sickle cell disease community reeling. Despite recent setbacks, hope for SCD treatment lies in next-gen transplantation and gene therapy, with St. Jude developing its own gene therapy. Pfizer's inclacumab and osivelotor, Agios Pharmaceuticals' mitapivat, Novo Nordisk's etavopivat, and Fulcrum Therapeutics' pociredir are among investigational therapies in the SCD pipeline.

Oxbryta (voxelotor), a sickle cell drug hailed as transformative, was withdrawn due to safety concerns, highlighting the need for cautious reporting on new drugs, especially those approved through the FDA's accelerated pathway. The drug's withdrawal caused confusion and heartbreak among patients and physicians, emphasizing the importance of neutral language and thorough reporting on clinical trial details, side effects, and FDA terminology to avoid misleading the public.

Pfizer withdraws Oxbryta (voxelotor) for sickle cell disease from all markets due to imbalance in vaso-occlusive crises and fatal events, discontinuing all clinical trials and expanded access program.