CRISPR Therapeutics AG

This is a single-arm, open-label, multicenter, ascending dose Phase 1/2 trial evaluating the safety and preliminary efficacy of CTX112 or Zugocabtagene geleucel (zugo-cel) in adult participants with relapsed/refractory primary Immune Thrombocytopenia (ITP) and relapsed/refractory primary Warm Autoimmune Hemolytic Anemia (wAIHA).Show more

This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).Show more

This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX112™ in subjects with relapsed or refractory B-cell malignancies.

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

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 with recurrent vaso-occlusive crises (December 2023) and transfusion-dependent beta thalassemia (January 2024). In July 2026 the FDA widened both approvals to patients aged 2 and older. The pipeline includes zugocabtagene geleucel (zugo-cel, formerly CTX112), a donor-derived CAR-T therapy in trials for B-cell cancers and for autoimmune diseases such as lupus, immune thrombocytopenia and warm autoimmune hemolytic anemia; CTX460 for alpha-1 antitrypsin deficiency, in a Phase 1 trial; CTX310, which targets ANGPTL3 to lower blood fats, in Phase 1b; and CTX321, a lipoprotein(a) program not yet in human trials.

CRISPR Therapeutics AG has active programs across 12 rare diseases: Alpha-1 Antitrypsin Deficiency, Dermatomyositis, Hodgkin Lymphoma, Homozygous Familial Hypercholesterolemia, Immune Thrombocytopenia, Mesothelioma, Multiple Sclerosis, Neuromyelitis Optica, Sickle Cell Disease, Stiff Person Syndrome, Systemic Sclerosis, Thalassemia. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Yes. CRISPR Therapeutics AG is a rare disease specialist headquartered in Zug, Switzerland, founded in 2013. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Alpha-1 Antitrypsin Deficiency, Dermatomyositis, Hodgkin Lymphoma among others.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by CRISPR Therapeutics AG, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

CRISPR Therapeutics AG was founded in 2013 and is headquartered in Zug, Switzerland. The company has been active for 13 years. It trades publicly under the ticker symbol CRSP. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.