Clinical Trial

Disease: Relapsed or Refractory B-cell Malignancies, (2018-003916-38)

Disease info:

B-cell malignancies are cancers that arise from abnormalities in B cells, and include B-cell lymphomas and leukaemias. While leukaemias typically originate in the bone marrow and spread through the bloodstream, lymphomas usually originate in the lymph nodes or the spleen and spread through the lymphatic system.

There are more than 70 known types of B-cell lymphoma, and these make up approximately 85 % of all cases of non-Hodgkin lymphomas in the United States. NHL is one of the most common cancers in the United States, accounting for about 4% of all cancers. B-cell lymphomas may grow and spread slowly with few symptoms (also known as indolent lymphoma) or may be very aggressive with severe symptoms. Other common types of B-cell lymphoma include:

  • Diffuse large B-cell lymphoma (DLBCL)
  • Follicular lymphoma
  • Chronic lymphocytic leukaemia (CLL) /small lymphocytic lymphoma (SLL)
  • Mantle cell lymphoma (MCL)
  • Marginal zone lymphomas
  • Burkitt lymphoma

In leukaemia, the bone marrow produces abnormal levels of white blood cells. B-cell acute lymphoblastic leukaemia (B-ALL) is an aggressive leukaemia in which too many B-cell lymphoblasts (immature white blood cells) are found in the bone marrow and blood. B-ALL is the most common type of ALL. Note that B-ALL is also called B-cell acute lymphocytic leukaemia and precursor B-lymphoblastic leukaemia. 

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.



NHL accounts for about 4% of all cancers in the U.S. The American Cancer Society estimates 80,550 people will be diagnosed with NHL in 2023. ALL accounts for less than 1% of all cancers in the U.S., with 6,540 new cases estimated in the U.S. in 2023.
Official title:
A Phase 1/2 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9–Engineered T Cells (CTX110) in Subjects with Relapsed or Refractory B-Cell Malignancies

Head of Regulatory Affairs: Steve Caffé M.D. CRISPR Therapeutics AG

Phone: +1847347 0018






United States

Study start:
Dec. 30, 2019
95 participants
Gene editing method:
Type of edit:
Gene insertion, gene knock-out
T Cell Receptor (TCR); Class I major histocompatibility complex (MHC I)
Delivery method:
Non-viral - Ex-vivo
CRISPR-Cas9 to insert a chimeric antigen receptor (CAR) construct with CD19 and disrupt two genes: The T cell receptor (TCR) and the class I major histocompatibility complex (MHC I) respectively.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Ongoing


To assess the safety of escalating doses of CTX110 in subjects with relapsed or refractory B-cell malignancies to determine the recommended Phase 2 dose (RP2D).

Phase 2 (cohort expansion): To assess the efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies, as measured by objective response rate (ORR).

CTX110 CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components. CTX110 is an allogeneic CRISPR-Cas9 gene-edited CAR-T cell therapy targeting CD19 for the treatment of CD19+ malignancies.

Last updated: Dec. 28, 2023
Search CRISPR Medicine