Current Clinical Research
CRISPR Therapeutics is focused on the development of novel therapies to treat hemoglobinopathies (inherited blood disorders), cancer, autoimmune diseases, cardiovascular diseases, diabetes, and other diseases.
Hemoglobinopathies
The most common symptoms people with sickle cell disease have are anemia, frequent infections, and pain.1
Our approach to treat sickle cell disease 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 people living with sickle cell disease, with the goal of reducing or eliminating symptoms.
This can result in anemia, or fewer red blood cells than normal, for which people may require frequent red blood cell transfusions that can have long-term negative consequences on the heart, liver, and other organs.2
Our approach to treat beta 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 people living with beta thalassemia, with the goal of reducing or eliminating symptoms.
Oncology (Cancer)
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.4
Our investigational approach to treat B-cell and T-cell lymphoma 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.
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.5
Our investigational approach to treat renal cell carcinoma 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.
Autoimmune Diseases
SLE can affect many parts of a person’s body including joints, skin, kidneys, brain, and other organs. Common symptoms include joint pain, swelling, fatigue, hair loss, weight loss, sun sensitivity, and rashes on the skin. People living with SLE can also have other symptoms depending on which tissues in the body are affected.8
Cardiovascular Diseases
Plaque buildup can make blood vessels too narrow for blood to flow freely. This condition is called atherosclerosis and increases the risk of heart attack, stroke, and other cardiovascular problems. Lp(a) can also lead to inflammation, causing thickening or hardening of arteries or stiffening of valves that control blood flow in the heart, which can lead to aortic valve disease.10
High levels of Lp(a) are mostly determined by your genes that you inherit from your parents. Unlike other types of bad cholesterol, Lp(a) cannot be lowered much by diet and lifestyle changes.11
People with HoFH are not able to rid their bodies of excess cholesterol and it builds up in the blood. This can increase the risk of developing a type of heart disease called coronary artery disease where the cholesterol forms clumps that can clog arteries and limit blood flow to the heart. Coronary artery disease increases the risk of a heart attack.13 In people with homozygous familial hypercholesterolemia, cholesterol can also build up in body tissues other than the heart and blood vessels.13
HoFH happens when a child inherits a genetic mutation that causes homozygous familial hypercholesterolemia from both of their parents.12
Symptoms of HoFH can include angina (a type of chest pain). Some visible signs of HoFH can include growths on tendons called tendon xanthomas, yellowish color of the skin of the eyelids, and a grey ring around the cornea in the front part of the eye.13
Coronary artery disease increases the risk of a heart attack.11 In people with heterozygous familial hypercholesterolemia, cholesterol can also build up in body tissues other than the heart and blood vessels.13
People with heterozygous familial hypercholesterolemia have inherited a genetic mutation that causes HeFH from one of their parents.12
Symptoms of HeFH can include angina (a type of chest pain). Some visible signs of HeFH can include growths on tendons called tendon xanthomas, yellowish color of the skin of the eyelids, and a grey ring around the cornea in the front part of the eye.13
SHTG can raise a person’s risk of heart disease and of developing acute pancreatitis, which is a sudden swelling of the pancreas.14
Severe Hypertriglyceridemia can be caused by genetic variations combined with factors in a person’s environment. The condition can be made worse by certain dietary factors.14
Mixed Dyslipidemia can increase a person’s risk of developing coronary heart disease and other cardiovascular problems.16
Mixed Dyslipidemia can be caused by genetic variations inherited from a person’s parent.16
Diabetes
If not treated, high blood glucose levels can cause damage to blood vessels, nerves, and organs throughout the body.17
Symptoms of T1D include frequent thirst, frequent urination, frequent hunger, tiredness, weight loss, dry, itchy skin, sores that heal slowly, tingling in the feet, and blurred vision.17
About Clinical Trials
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 a countries’ health or regulatory authority, such as the U.S. Food and Drug Administration. For more information, visit the U.S. Food and Drug Administration’s website.
Current Clinical Trials
Product candidatesbeing investigated in ongoing clinical trials generally are not approved by the FDA or any other countries’ health or regulatory authority for the population or indication being investigated. This means that the safety and efficacy of product candidates are still being studied in clinical trials. You can find more information about our current trials in the United States at clinicaltrials.gov.
You can also speak with your health care provider if you are interested in learning more about clinical trials and whether enrolling in a clinical trial may be right for you.
1. Sickle cell disease: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/genetics/condition/sickle-cell-disease/. Updated March 14, 2024.
2. Beta thalassemia: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/genetics/condition/beta-thalassemia/. Updated May 1, 2023.
3. 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/ Updated June 18, 2019.
4. Understanding lymphoma. Lymphoma Research Foundation. https://lymphoma.org/understanding-lymphoma/aboutlymphoma/ Accessed July 25, 2024.
5. American Cancer Society. What is Kidney Cancer? https://www.cancer.org/cancer/types/kidney-cancer/about/what-is-kidney-cancer.html. Updated May 1, 2024.
8. Systemic lupus erythematosus: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/ency/article/000435.htm. Updated January 25, 2023.
9. Lipoprotein (a): Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/ency/article/007262.htm. Updated May 8, 2022.
10. Atherosclerosis: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/atherosclerosis.html. Updated March 6, 2024.
11. Blood cholesterol diagnosis: National Heart, Lung, and Blood Institute. https://www.nhlbi.nih.gov/health/blood-cholesterol/diagnosis. Updated April 18, 2024.
12. Familial Hypercholesterolemia: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/genetics/condition/familial-hypercholesterolemia/. Updated January 1, 2020.
13. American Heart Association. Understanding homozygous familial hypercholesterolemia. https://www.heart.org/en/health-topics/cholesterol/genetic-conditions/homozygous-fh. Updated October 30, 2023.
14. Hypertriglyceridemia. Cleveland Clinic Health Library. https://my.clevelandclinic.org/health/diseases/23942-hypertriglyceridemia. Updated: July 29, 2022.
15. Triglycerides. Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/triglycerides.html. Updated October 7, 2020.
16. Familial Combined Hyperlipidemia. Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/ency/a.... Updated May 8, 2024.
17. Diabetes Type 1: Medline Plus, U.S. National Library of Medicine. https://medlineplus.gov/diabetestype1.html. Updated June 29, 2020.
