CRISPR Therapeutics is at the forefront of gene-editing technology, specifically utilizing CRISPR/Cas9 to develop innovative therapies for serious diseases. Their flagship product, Exagamglogene autotemcel (exa-cel), represents the first approved CRISPR-based therapy aimed at treating sickle cell disease and transfusion-dependent beta thalassemia. This autologous, ex vivo gene-edited therapy works by modifying a patient's own hematopoietic stem cells to produce fetal hemoglobin, thereby alleviating the symptoms associated with these hemoglobinopathies. The company is also exploring in vivo editing techniques, which involve delivering CRISPR/Cas9 directly to hematopoietic stem cells within the body, potentially streamlining treatment processes and improving patient outcomes.

In addition to hemoglobinopathies, CRISPR Therapeutics is expanding its research into oncology and autoimmune diseases through the development of allogeneic CAR T cell therapies. These therapies leverage gene editing to enhance the potency of T cells, enabling them to target and eliminate cancer cells more effectively. The company is investigating various applications, including treatments for B- and T-cell malignancies and solid tumors. Furthermore, CRISPR Therapeutics is exploring regenerative medicine approaches, such as gene-edited stem cell therapies for conditions like Type 1 diabetes. Their diverse pipeline reflects a commitment to harnessing the transformative potential of CRISPR technology across multiple disease areas.

United States

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Cancer

Cardiovascular Diseases

Endocrine System Diseases

Genetic Diseases

Hematologic Diseases

Immune System Diseases

Lung Diseases

Musculoskeletal Diseases

Various

CRISPR Therapeutics

Sickle cell disease

CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease
Gene editing
CAR T

CASGEVY™ (Exagamglogene autotemcel) (CTX001) (autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy) (Exa-cel)

Beta thalessemia

CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease
Our approach to treat β-thalassemia and SCD is designed to increase &nbsp;levels of fetal hemoglobin (HbF), a naturally-occurring form of &nbsp;hemoglobin present in all people before birth. HbF can substitute for &nbsp;the diseased hemoglobin in β-thalassemia and SCD patients, reducing or &nbsp;eliminating symptoms.
Our lead therapeutic program, CTX001, aims to increase fetal &nbsp;hemoglobin expression by editing patients' own blood stem cells with &nbsp;CRISPR/Cas9
As a therapy, CTX001 involves isolating a patient’s own blood stem &nbsp;cells, editing them with CRISPR/Cas9 to increase HbF expression, and &nbsp;then returning the edited cells to the patient. We believe that over &nbsp;time these edited blood stem cells will generate red blood cells that &nbsp;have increased levels of HbF, which may reduce or eliminate patients’ &nbsp;symptoms. In 2017, we signed an agreement to co-develop and &nbsp;co-commercialize this program with our partner Vertex Pharmaceuticals.
<a href="http://www.crisprtx.com/programs/hemoglobinopathies">Source</a>
Gene editing
CAR T

CASGEVY™ (Exagamglogene autotemcel) (CTX001) (autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy) 

B-cell malignancies

Blood cancer

CD19+ malignancies

CTX110 is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy targeting CD19 in development for the treatment of CD19+ malignancies
Gene editing
CAR T

CTX110 (CD19)

T-cell lymphoma

CTX130, a wholly-owned program of CRISPR Therapeutics, &nbsp;is a healthy donor-derived gene-edited allogeneic CAR-T investigational &nbsp;therapy targeting cluster of differentiation 70, or CD70, an antigen &nbsp;expressed on various solid tumors and hematologic malignancies.
CTX130, marks our transition from hematologic to solid tumors. CTX130 &nbsp;targets CD70, an antigen expressed on both hematologic cancers, &nbsp;including certain lymphomas, and solid tumors, including renal cell &nbsp;carcinoma. Following on these programs, we plan to use both novel &nbsp;targeting and advanced editing to develop gene-edited cell therapies to &nbsp;treat numerous other cancer
<a href="http://www.crisprtx.com/programs/immuno-oncology">Source</a>
Gene editing
CAR T

CTX130 (CD70)

CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease
Our approach to treat β-thalassemia and SCD is designed to increase &nbsp;levels of fetal hemoglobin (HbF), a naturally-occurring form of &nbsp;hemoglobin present in all people before birth. HbF can substitute for &nbsp;the diseased hemoglobin in β-thalassemia and SCD patients, reducing or &nbsp;eliminating symptoms.
Our lead therapeutic program, CTX001, aims to increase fetal &nbsp;hemoglobin expression by editing patients' own blood stem cells with &nbsp;CRISPR/Cas9
As a therapy, CTX001 involves isolating a patient’s own blood stem &nbsp;cells, editing them with CRISPR/Cas9 to increase HbF expression, and &nbsp;then returning the edited cells to the patient. We believe that over &nbsp;time these edited blood stem cells will generate red blood cells that &nbsp;have increased levels of HbF, which may reduce or eliminate patients’ &nbsp;symptoms. In 2017, we signed an agreement to co-develop and &nbsp;co-commercialize this program with our partner Vertex Pharmaceuticals.
<a href="http://www.crisprtx.com/programs/hemoglobinopathies">Source</a>
Gene editing

Exagamglogene autotemcel (CTX001) (autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy)

Type 1 diabetes

VCTX210 is an investigational, allogeneic, gene-edited, stem cell-derived product candidate for Type 1 Diabetes (T1D) being developed by CRISPR Therapeutics and ViaCyte.
Gene editing
CAR T

VCTX210

CTX112 incorporates the edits in CTX110 plus additional edits to the genes encoding Regnase-1 and transforming growth factor-beta (TGF-β) receptor type 2 (TGFBR2), which have been shown to increase CAR T potency and reduce CAR T exhaustion in pre-clinical studies.

CTX112

Solid tumor/s

CTX131 incorporates the edits in CTX130 plus additional edits to the genes encoding Regnase-1 and TGFBR2.
<a href="http://www.crisprtx.com/programs/immuno-oncology">Source</a>
Gene editing
CAR T

CTX131 (CD70) 

VCTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of T1D, which incorporates additional gene edits that aim to further enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose.

CTX211 (formerly VCTX211)

Atherosclerotic cardiovascular disease

In vivo gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of ANGPTL3.

CTX310 (ANGPTL3)

CTX320, In vivo investigational gene-editing therapy using lipid nanoparticle (LNP) delivery of Cas9 mRNA and gRNA to the liver to reduce expression of Lp(a)

CTX320 Lp(a)

Systemic lupus erythematosus

Multiple myeloma

CTX120 is an allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapy targeting BCMA in development for the treatment of multiple myeloma.
Our lead immuno-oncology cell therapy programs, CTX110 and CTX120, bring a potentially best-in-class allogeneic approach to validated tumor &nbsp;targets. CTX120 targets BCMA, an antigen expressed in &nbsp;multiple myeloma.
For our initial allogeneic gene-edited CAR-T product candidates, such &nbsp;as our lead immuno-oncology program CTX110, we make three modifications &nbsp;to healthy donor T cells to allow our CAR-T cells to be used &nbsp;off-the-shelf: CAR, TCR, and MHC I.&nbsp;
 
CAR: The chimeric antigen receptor (CAR) &nbsp;allows CAR-T cells to target and kill cancer cells. A CAR has two key &nbsp;domains: one that binds to the surface of cancer cells and another that &nbsp;activates the T cell. The current generation of CAR-T products uses &nbsp;randomly-integrating viruses to deliver the CAR construct to the DNA of T &nbsp;cells. In contrast, we use CRISPR/Cas9 to insert the CAR construct &nbsp;precisely into the TCR alpha constant (TRAC) locus, which we expect to &nbsp;result in a safer, more consistent product
 
TCR: T cells use the T cell receptor (TCR) &nbsp;to recognize and kill cells presenting foreign antigens (a sign of &nbsp;infection), thereby providing immunity from disease. Donor T cells could &nbsp;also recognize a patient’s cells as foreign through this receptor, &nbsp;leading to an unwanted side effect known as graft versus host disease &nbsp;(GvHD). We use CRISPR/Cas9 to eliminate the TCR with high efficiency, &nbsp;which reduces the risk of GvHD occurring during off-the-shelf use
 
MHC I: To improve CAR-T cell persistence &nbsp;and increase the chance for durable remissions, we use CRISPR/Cas9 to &nbsp;eliminate the class I major histocompatibility complex (MHC I) expressed &nbsp;on the surface of our CAR-T product candidates. If present, MHC I could &nbsp;lead to rejection of the CAR-T product by the patient’s own T cells. &nbsp;Eliminating this molecule should mitigate that effect
 
<a href="http://www.crisprtx.com/programs/immuno-oncology">Source</a>
Gene editing
CAR T

CRISPR Therapeutics

Company Details