Gene editing CAR-T cell therapy has been recognized as an advanced therapy in regenerative medicine for common T-cell lymphoma

▎ CRISPR Therapeutics, editor of WuXi AppTec's content team, recently announced that the U.
S.
FDA has granted it the Allogeneic CAR-T Cell Therapy CTX130 Advanced Therapy for Regenerative Medicine (RMAT) designation for the treatment of granulomatous fungioids (MF) and Sézary syndrome (SS).


Mushroom granuloma and Sézary syndrome are two common types of cutaneous T-cell lymphomas
.
Although most patients with early MF/SS have a slow course, the prognosis for patients with advanced disease is poor, with median survival of less than 5 years
.
Early patients are treated primarily with skin-directed therapy to control skin lesions, such as topical therapy, phototherapy, and radiation therapy
.
For advanced patients, systemic treatments such as biological, targeted, and immunosuppressive chemotherapy can be tried to control the further development
of the disease.
In addition, allogeneic hematopoietic stem cell transplantation has the opportunity to provide eligible patients with the opportunity for a cure and a lasting complete remission
.

CRISPR Therapeutics is a gene-editing company
focused on using its proprietary CRISPR/Cas9 platform to develop transformative gene drugs for the treatment of serious diseases.
CTX130 is its wholly-owned health donor-derived CRISPR/Cas9 gene-edited allogene-alternating CAR-T cell therapy targeting CD70
.
CD70 is an antigen
expressed in a variety of solid tumors and hematologic malignancies.
CRISPR Therapeutics' official website points out that gene-edited CAR-T candidates can overcome the limitations of current patient-derived CAR-T therapies and can generate ready-to-use, efficient, and highly consistent allogeneic CAR-T cells
.
In addition, by eliminating or inserting genes by using CRISPR/Cas9, new classes of CAR-T products can be created to improve suitability
for solid tumors.

▲ CRISPR Therapeutics' Immuno-Oncology pipeline (image from CRISPR Therapeutics) Currently, CTX130 is undergoing two independent Phase 1 single-arm, multicenter, open-label clinical trials (COBALT-LYM and COBALT-RCC)
aimed at evaluating multiple dose levels of CTX130 in relapsed/refractory T/refractory T/ Safety and efficacy in adult patients with B-cell malignancy or relapsed/refractory renal cell carcinoma
.
The CTX130 has been granted orphan drug status
by the FDA.

"Obtaining RMAT recognition is an important milestone
for the CTX130 project.
The encouraging clinical data to date illustrate the transformative potential of our cell therapies in patients with T-cell lymphoma," said Dr.
Phuong Khanh (P.
K.
) Morrow, Chief Medical Officer of CRISPR Therapeutics, "We will bring our broad portfolio of allogeneic cell therapies to patients in
need as soon as possible.
" ”

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