Current Clinical Research

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

Learn about our current clinical research and trials in hemoglobinopathies and oncology 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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Both diseases require lifetime treatment. They can result in painful symptoms, the need for regular transfusions, and frequent hospitalizations. Complications from both diseases can cause someone to die earlier than would be expected.1,2

Get the Facts on Gene Editing for Hemoglobinopathies

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CRISPR in Hemoglobinopathies

CRISPR Therapeutics is researching a gene-editing approach designed to edit blood cells to increase hemoglobin.

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CRISPR Therapeutics’ gene-editing approach 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.

CRISPR/Cas9 is a gene editing technology that works by making precise, directed changes to DNA.

Learn more in our video


Oncology

CRISPR Therapeutics’ cancer programs are investigating blood cancers such as B- and T-cell lymphomas and multiple myeloma as well as 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

Multiple myeloma is a type of cancer that develops in the bone marrow, the spongy material found in the center of most bones. Bone marrow is one of the places the body makes blood cells, including plasma cells. Plasma cells make antibodies, which the body uses to attack and kill germs.6

Multiple myeloma causes plasma cells in the marrow to multiply out of control. As a result, the marrow produces fewer:

  • Red blood cells (anemia), which can lead to fatigue and weakness
  • White blood cells (leukopenia), which weakens the immune system and increases the risk of infection
  • Platelets (thrombocytopenia), which can lead to abnormal bleeding and bruising6

Multiple myeloma can cause tumors in the bones that can lead to pain, fractures and loss of bone mass in the spine, skull, and ribs.6

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.7 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.7

What is Car T Cell Therapy?

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

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CRISPR Therapeutics’ gene-editing approach 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.8
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 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 clinicaltrials.gov or use the links below:

You can also talk to your health care provider about clinical trials and if enrolling in a trial may be right for you.

References

1. Beta thalassemia. Genetics Home Reference, U.S. National Library of Medicine. https://ghr.nlm.nih.gov/condition/beta-thalassemia. August 17, 2020. Accessed: September 18, 2020.

2. Sickle cell disease. Genetics Home Reference, U.S. National Library of Medicine. https://ghr.nlm.nih.gov/condition/sickle-cell-disease. August 17, 2020. Accessed: September 18, 2020.

3. Mayo Clinic. Anemia: Symptoms & causes. Mayo Clinic Website. Published August 16, 2019. Accessed December 15, 2020. https://www.mayoclinic.org/diseases-conditions/anemia/symptoms-causes/syc-20351360

4. What is the difference between B-cell lymphoma and T-cell lymphoma? Dana Farber Cancer Institute. https://blog.dana-farber.org/insight/2019/06/what-is-the-difference-between-b-cell-lymphoma-and-t-cell-lymphoma/. June 18, 2019. Accessed: September 18, 2020.

5. What is lymphoma? Lymphoma Research Foundation. https://lymphoma.org/aboutlymphoma/. Accessed: September 18,2020.

6. Multiple myeloma. Genetics Home Reference, U.S. National Library of Medicine. https://ghr.nlm.nih.gov/condition/multiple-myeloma. August 17, 2020. Accessed: September 18, 2020.

7. Kidney cancer - introduction. https://www.cancer.net/cancer-types/kidney-cancer/introduction. November 25, 2019. Accessed: November 11, 2020.

8. ClinicalTrials.gov. Learn About Clinical Studies. ClinicalTrials.gov. Updated March 2019. Accessed December 04, 2020. https://clinicaltrials.gov/ct2/about-studies/learn

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