Also known as: Casgevy, CASGEVY
Casgevy (exagamglogene autotemcel) is a CRISPR/Cas9 genome-edited autologous CD34+ hematopoietic stem-cell therapy approved for sickle cell disease with recurrent vaso-occlusive crises and for transfusion-dependent beta thalassemia in patients 12 years and older. It is administered as a one-time infusion after myeloablative conditioning.
CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
CRISPR Therapeutics reported a $581.6 million net loss in 2025 with revenue of just $3.5 million, while continuing to advance its gene-editing pipeline including the approved CASGEVY therapy and next-generation CAR T cell programs. The company maintains strategic partnerships and expects current funds to support operations for at least 24 months.
CRISPR Therapeutics reported a Q4 2025 loss of $1.37 per share, missing estimates, with revenues of $0.9 million falling short of expectations. The company's partner Vertex recorded $54 million in Casgevy sales for the quarter, with regulatory submissions for pediatric label expansion planned for early 2026.
The cell therapy manufacturing market is projected to reach $14.01 billion by 2035, with CAR-T therapies dominating at 65% market share. Recent FDA approvals for new CAR-T indications and Japanese regulatory acceleration highlight growing clinical adoption, while research advances include new anti-aging protein platforms and CRISPR-based treatments.
The immuno-oncology market is projected to grow from $65.22 billion in 2025 to $170.19 billion by 2032, driven by checkpoint inhibitors, CAR-T cell therapy, and cancer vaccines, with North America commanding 43% market share.
The FDA has issued draft guidance that may speed approval pathways for rare disease therapies using genome editing technologies, potentially benefiting CRISPR Therapeutics' pipeline of six clinical-stage candidates.
Approved gene-editing therapies for sickle cell disease priced at $2.2 million remain out of reach in Africa, where 80% of the world's cases occur, as Uganda launches mandatory newborn screening.
Switzerland-based CRISPR Therapeutics stands to benefit from increased Casgevy sales following partner Vertex Pharmaceuticals' fourth-quarter results, with five additional gene-editing therapies in clinical trials targeting larger patient populations.
Vertex Pharmaceuticals and CRISPR Therapeutics reported $116 million in full-year 2025 sales for gene-editing therapy Casgevy, with 64 patients receiving infusions and patient initiations nearly tripling compared to 2024.
