Disease: Relapsed or Refractory T or B cell malignancies, (NCT04502446)

Disease info:

Lymphoma is a type of blood cancer that develops when white blood cells called lymphocytes grow out of control. Lymphocytes are part of our immune system. They travel around our body in our lymphatic system, helping us fight infections. There are two types of lymphocyte: T lymphocytes (T cells) and B lymphocytes (B cells).

Lymphomas can be grouped as Hodgkin lymphomas or non-Hodgkin lymphomas, depending on the types of cell they contain. T-cell lymphomas are non-Hodgkin lymphomas that develop from T lymphocytes.

Most T-cell lymphomas develop from mature T cells. They usually affect older adults, typically people in their mid-60s. They are more common in men than in women. Occasionally, T-cell lymphoma can develop from immature T cells. This is known as T-cell lymphoblastic lymphoma. It tends to affect children and young adults.

In most cases, it is not known what causes T-cell lymphomas. In a few types of T-cell lymphoma, research has shown that certain viral infections or medical conditions can increase the risk of developing lymphoma. 

Human T-lymphotropic virus type 1 (HTLV-1) is linked with development of adult T-cell leukaemia/lymphoma (ATL).
Past infection with Epstein-Barr virus (EBV) is linked to the development of a range of lymphomas, including angioimmunoblastic T-cell lymphoma (AITL).
Enteropathy-associated T-cell lymphoma (EATL) is linked with coeliac disease.

Remember that T-cell lymphomas are very rare and most people with these conditions do not develop lymphoma.

T-cell lymphomas are rare. They account for around 1 in 10 cases of non-Hodgkin lymphoma. Around 700 people are diagnosed with T-cell lymphomas in the UK each year.
Official title:
A Phase 1, Open-Label, Multicenter, Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD70 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Relapsed or Refractory T or B Cell Malignancies

United States, Texas

Study start:
Jul. 31, 2020
45 participants
Gene editing method:
Insert anti-CD70 CAR, MCH-I knock-out, TCR knock-out
Delivery method:
No information - Ex-vivo
CTX130 allogeneic CAR-T cells targets CD70, an antigen expressed on both hematologic cancers, including certain lymphomas, and solid tumors, including renal cell carcinoma.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and efficacy of CTX130 in subjects with relapsed or refractory T or B cell malignancies.

CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components. Three modifications to healthy donor T cells are made to allow CAR-T cells to be used off-the-shelf:

CAR: The chimeric antigen receptor (CAR) allows CAR-T cells to target and kill cancer cells. A CAR has two key domains: one that binds to the surface of cancer cells and another that activates the T cell. In CTX130 CRISPR-Cas9 is uded to insert the CAR construct precisely into the TCR alpha constant (TRAC) locus, which it is expected to result in a safer, more consistent product.

TCR: T cells use the T cell receptor (TCR) to recognize and kill cells presenting foreign antigens (a sign of infection), thereby providing immunity from disease. Donor T cells could also recognize a patient’s cells as foreign through this receptor, leading to an unwanted side effect known as graft versus host disease (GvHD). CTX130 uses CRISPR-Cas9 to eliminate the TCR with high efficiency, which reduces the risk of GvHD occurring during off-the-shelf use.

MHC I: To improve CAR-T cell persistence and increase the chance for durable remissions, CTX130 uses CRISPR-Cas9 to eliminate the class I major histocompatibility complex (MHC I) expressed on the surface of the CAR-T product candidate. If present, MHC I could lead to rejection of the CAR-T product by the patient’s own T cells. Eliminating this molecule should mitigate that effect.

Last updated: Aug. 31, 2020
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