Current Clinical Research

CRISPR Therapeutics is focused on the development of novel therapies to treat hemoglobinopathies, cancer, cardiovascular diseases, diabetes, and other diseases.

Learn about our current clinical research and trials below.

Sickle Cell Disease and β-thalassemia

Sickle cell disease and β-thalassemia are inherited blood diseases that result from mutations in the gene that makes hemoglobin. Hemoglobin is a molecule that is responsible for carrying oxygen within red blood cells as they travel in the bloodstream.1,2

People who have sickle cell disease have red blood cells that take on a crescent or sickle shape under certain circumstances. The most common symptoms patients with sickle cell disease have are anemia, frequent infections, and pain.2

People who have β-thalassemia have less hemoglobin. This can result in anemia, or fewer red blood cells than normal, for which patients may require frequent red blood cell transfusions that can have long-term negative consequences on the heart, liver, and other organs.1

Some symptoms of anemia include:1,3
  • Dizziness
  • A fast heartbeat
  • Shortness of breath
  • Pale skin

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Get the Facts on Hemoglobinopathies: Sickle Cell Disease (SCD) and β-thalassemia

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CRISPR Approach for Hemoglobinopathies

Our approach to treat SCD and β-thalassemia is designed to switch back on expression of a different form of hemoglobin called fetal hemoglobin (HbF), which is naturally present in all people before birth. Increased levels of HbF are intended to substitute for the diseased adult hemoglobin in patients with SCD and β-thalassemia, with the goal of reducing or eliminating symptoms.


CRISPR Therapeutics’ cancer programs are investigating blood cancers such as B- and T-cell lymphomas and renal cell carcinoma, a type of solid tumor.

B cells and T cells are types of white blood cells called lymphocytes. They are part of the immune system, which helps your body fight infections and other disease.

If genes that control the growth of lymphocytes no longer work properly, these cells can multiply out of control or live longer than they normally would. This condition is called lymphoma.4

Symptoms of B-cell and T-cell lymphoma can vary in severity, and include swollen lymph nodes that may be painful, fever, night sweats, weight loss, chills, tiredness, and itching.5

Renal cell carcinoma, or RCC, is a type of kidney cancer. In renal cell carcinoma, cells in the kidney multiply and create tumors. In its advanced form, RCC cells have spread to other parts of the body.6 Symptoms of RCC include blood in the urine, pain or pressure in the side or back, swelling in the ankles and legs, high blood pressure, anemia, tiredness, weight loss, and fever.6

What is CAR T Cell Therapy?

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Understanding CAR T Cell Therapy

CRISPR Therapeutics’ gene-editing approach to cancer uses allogeneic CAR T cells. Allogeneic CAR T cells are T cells that have been modified to include a chimeric antigen receptor (CAR) on their surface. With this receptor, allogeneic CAR T cells are able to find and kill cancer cells.

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CRISPR Therapeutics’ gene-editing approach in oncology is still being investigated in clinical trials, and its safety and efficacy have not been established. It is not approved for use in patients in the United States or in any other countries.

What are Clinical Trials?

A clinical trial is a study used to learn more about a treatment for a disease or condition. People with the condition being studied are enrolled as volunteers. The purpose of a trial is to determine the safety of a treatment and how well it works.

Clinical trials follow strict guidelines laid out by federal regulatory agencies, such as the U.S. Food and Drug Administration.
For more information, visit the U.S. Food and Drug Administration’s website.
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Current Clinical Trials

The therapies that CRISPR Therapeutics is investigating in ongoing clinical trials are not approved by the FDA or any other countries’ Health Authority. This means that their safety and efficacy are still being studied in clinical trials. You can find more information about our current trials at or use the links below:

You can also talk to your health care provider about any of these conditions or you are interested in learning more about clinical trials and if enrolling in a trial may be right for you.


1. Beta thalassemia: Genetics Home Reference, U.S. National Library of Medicine. 17, 2020.

2. Sickle cell disease: Genetics Home Reference, U.S. National Library of Medicine. 17, 2020.

3. Mayo Clinic. Anemia: Symptoms & causes. Mayo Clinic Website. Published August 16, 2019.

4. What is the difference between B-cell lymphoma and T-cell lymphoma? Dana Farber Cancer Institute. June 18, 2019.

5. What is lymphoma? Lymphoma Research Foundation.

6. Kidney cancer - introduction. November 25, 2019.

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