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CRISPR Therapeutics AG

Rare disease drugmaker · based in Zug, Switzerland · founded 2013 · ticker CRSP · crisprtx.com

CRISPR Therapeutics AG works on 13 rare diseases we track, including Alpha-1 Antitrypsin Deficiency, Dermatomyositis and 11 more. It has 7 active clinical trials, and 5 are looking for volunteers now.

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.

CRISPR Therapeutics AG pipeline

Each row lists the diseases CRISPR Therapeutics AG is studying at that stage, from the ones closest to approval down to the earliest work.

Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

Alpha-1 Antitrypsin Deficiency recruiting · Warm Autoimmune Hemolytic Anemia recruiting · B-cell Lymphoma recruiting

Phase 1
First studies in people, mostly checking safety.

Lupus Nephritis recruiting · Cardiovascular recruiting

CRISPR Therapeutics AG clinical trials

These are CRISPR Therapeutics AG trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

5 trials looking for volunteers now

Looking for volunteersPhase 1/2Recently updatedNCT07850076

A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)

Tests CTX460

For Alpha-1 Antitrypsin Deficiency (AATD)

A Study of CTX460 in Adults with Emphysema Associated with Severe Alpha-1 Antitrypsin Deficiency (AATD).

Ages 18 Years - 75 Years1 location
View full study
Looking for volunteersPhase 1Recently updatedNCT06925542

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

Tests CTX112

For SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis, Systemic Sclerosis and Inflammatory Myopathy, Idiopathic

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).

Ages 18 Years - 70 Years17 locations
View full study
Looking for volunteersPhase 1/2Recently updatedNCT07549698

Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease

Tests CTX112

For Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia and Immune Thrombocytopenic Purpura

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).

Ages 18 Years+12 locations
View full study
Looking for volunteersPhase 1Recently updatedNCT07491172

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

Tests CTX310

For Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder and Hypertriglyceridemia

This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.

Ages 18 Years - 75 Years17 locations
View full study
Looking for volunteersPhase 1/2No updates in a whileNCT05643742

A Safety and Efficacy Study Evaluating CTX112 in Subjects With Relapsed or Refractory B-Cell Malignancies

Tests CTX112

For B-cell Lymphoma, Non-Hodgkin Lymphoma, B-cell Malignancy, Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) and Follicular Lymphoma

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.

Ages 18 Years+7 locations
View full study

2 trials underway, not taking new volunteers

Underway, not taking new volunteersPhase 3Updated a few months agoNCT05329649

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Tests CTX001

For Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies and Hematological Diseases

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).

Ages 2 Years - 11 Years7 locations
View full study
Underway, not taking new volunteersPhase 3Updated a few months agoNCT05356195

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Tests CTX001

For Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases and Hemoglobinopathies

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).

Ages 2 Years - 11 Years6 locations
View full study
Ask about CRISPR Therapeutics AG's trials
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More about CRISPR Therapeutics AG

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 a Phase 1 trial; and CTX321, a lipoprotein(a) program not yet in human trials.

Where CRISPR Therapeutics AG trials take place

The trials above run at 67 study sites in 8 countries. Tap a country to see its states, cities and hospitals.

United States
29▼
Germany
10▼
Australia
9▼
Spain
9▼
United Kingdom
5▼
New Zealand
2▼
Italy
2▼
Canada
1▼

Rare diseases CRISPR Therapeutics AG works on

Each disease page lists every open trial, not only CRISPR Therapeutics AG's, plus approved treatments.

Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin.

How common: 1 in 1,500 to 3,500 people of European ancestry; about 100,000 people in the U.S

Dermatomyositis

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints.

How common: About 9.6 new cases per million people a year, according to NORD; juvenile-onset incidence about 0.4 cases per 100,000 children

Homozygous Familial Hypercholesterolemia

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...

How common: Approximately 1 in 250,000 to 1 in 360,000 people globally

Immune Thrombocytopenia

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.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Lupus Nephritis

Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.

How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)

Mantle Cell Lymphoma

Mantle cell lymphoma is a rare type of non-Hodgkin lymphoma that starts in B cells, a kind of white blood cell.

How common: About 1 in 200,000 people (Cleveland Clinic)

Multiple Sclerosis

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.

How common: Approximately 1 million people in the U.S.; about 1 in 300

Neuromyelitis Optica

Neuromyelitis Optica (NMO) is a rare inflammatory autoimmune disorder of the central nervous system characterized by recurrent attacks of optic neuritis (vision loss) and acute myelitis (spinal cord...

How common: About 1 to 2 per 100,000 people worldwide, according to MedlinePlus Genetics

Sickle Cell Disease

Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion.

How common: About 100,000 Americans with SCD; about 1 in 365 Black or African American births and 1 in 16,300 Hispanic American births

Stiff Person Syndrome

Stiff Person Syndrome (SPS) is a rare neurological disorder characterized by progressive stiffness and rigidity of the trunk and limbs, along with painful muscle spasms.

How common: Extremely rare with approximately 1-2 cases per million people; estimated 300-500 affected individuals in North America

Systemic Sclerosis

Systemic sclerosis (scleroderma) is a rare autoimmune disease in which the immune system triggers scarring (fibrosis) of the skin and internal organs while also damaging small blood vessels.

How common: About 276 per million U.S. adults in a large Detroit-area study; about 19 new cases per million adults each year

Thalassemia

Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes anemia, from mild or none in carriers to severe in the major forms, where it can...

How common: About 1 in 100,000 people have symptoms of beta thalassemia, according to NORD, most often people with Mediterranean, Middle Eastern, African or Asian roots

Warm Autoimmune Hemolytic Anemia

Warm autoimmune hemolytic anemia (wAIHA) is a rare autoimmune disease in which the immune system makes antibodies, usually of the IgG type, that stick to red blood cells at normal body temperature...

How common: About 1 to 3 new cases per 100,000 people each year, according to the FDA and NORD

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. CRISPR Therapeutics AG's own site is crisprtx.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about CRISPR Therapeutics AG