CRISPR Therapeutics AG
CRISPR Therapeutics AG works on 11 rare diseases tracked on Trial Friend, including Dermatomyositis, Hodgkin Lymphoma, Homozygous Familial Hypercholesterolemia and 8 more, with 5 recruiting clinical trials.
CRISPR Therapeutics develops CRISPR/Cas9-based gene-editing therapies for serious diseases. Casgevy (exagamglogene autotemcel), co-developed with Vertex Pharmaceuticals, was the first CRISPR-based therapy approved by the FDA for sickle cell disease (December 2023) and transfusion-dependent beta thalassemia (January 2024). The pipeline includes CTX112, an allogeneic CAR-T therapy for B-cell malignancies, and CTX310 targeting ANGPTL3 for cardiovascular disease, and CTX320 targeting lipoprotein(a).
CRISPR Therapeutics AG Drug Pipeline
CRISPR Therapeutics AG has 5 active clinical trials across 3 development stages, with 5 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of CRISPR Therapeutics AG's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
CRISPR Therapeutics AG Clinical Trials (5)
Active and recruiting clinical trials sponsored by CRISPR Therapeutics AG, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
CRISPR Therapeutics AG Trial Locations
CRISPR Therapeutics AG clinical trials are running at 46 sites in 7 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (11)
Diseases targeted by CRISPR Therapeutics AG's clinical trial and drug development programs
Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...
Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells. It typically starts in lymph nodes in the neck, chest, or armpits and can spread to...
Homozygous Familial Hypercholesterolemia is a severe genetic disorder causing extremely high cholesterol levels from birth, leading to early heart disease and cholesterol deposits in tendons and aroun...
Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production. It causes easy bruising, pinp...
Mesothelioma is a rare, aggressive cancer that develops in the mesothelium, the thin layer of tissue covering most internal organs. It most commonly affects the lining of the lungs (pleural mesothelio...
Multiple sclerosis is a chronic autoimmune disease where the immune system attacks the protective myelin sheath surrounding nerve fibers in the brain and spinal cord. This disrupts communication betwe...
Patient Resources
Organizations and resources related to CRISPR Therapeutics AG's rare disease focus areas
Frequently Asked Questions About CRISPR Therapeutics AG
Common questions about CRISPR Therapeutics AG's rare disease programs, clinical trials, and treatments.