Cholangiocarcinoma therapy Gavo-cel received FDA orphan drug designation

A few days ago, TCR²Therapeutics announced that the U.
S.
Food and Drug Administration (FDA) has granted Gavo-cel Orphan Drug Designation (ODD) for the treatment of cholangiocarcinoma
.

Gavo-cel is a new type of cell therapy consisting of autologous genetically engineered T cells expressing a single domain antibody that can recognize human mesothelin and fused with the CD3-ε subunit, which is integrated into the endogenous after expression Sex T cell receptor (TCR) complex
.

The purpose of this phase 1/2 trial is to determine the recommended phase 2 dose (RP2D) and the overall response rate of this therapy in mesothelin-expressing advanced cancer patients

Mesothelin is a cell surface glycoprotein that is highly expressed in a variety of solid tumors, including malignant pleural/peritoneal mesothelioma, ovarian cancer, cholangiocarcinoma, breast cancer, and pancreatic cancer
.

Because it can promote the proliferation, invasion and metastasis of cancer cells, mesothelin plays an active role in malignant transformation and tumor invasion

TCR² proprietary T cell receptor (TCR) Fusion Construct T cells (TRuC-T cells) can specifically recognize and kill cancer cells by using signals from the entire TCR, independent of human leukocyte antigen (HLA)
.

In preclinical studies, compared with chimeric antigen receptor T cells (CAR-T cells), TRuC-T cells show superior anti-tumor activity, while secreting and releasing fewer cytokines

The company's TRuC-T cell candidate product Gavo-cel, which targets solid tumors, is currently being studied in phase 1/2 clinical trials to treat mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, and malignant pleura/peritoneum Mesothelioma and cholangiocarcinoma
.

The company's other TRuC-T cell candidate product for hematological malignancies, TC-110, is currently being studied in a phase 1/2 clinical trial to treat CD19-positive adult acute lymphoblastic leukemia (aALL) and aggressive or indolent non- Patients with Hodgkin's Lymphoma (NHL)

According to the previously published phase 1 trial data, Gavo-cel is safe and well tolerated.
No patients had neurotoxicity or target toxicity in normal tissues, and 2 patients had cytokine release syndrome greater than grade 3.
(CRS), and used touzumab and glucocorticoid therapy
.

All 8 patients underwent at least one disease remission assessment, the disease control rate (DCR) was 100%, all patients experienced tumor regression, and the median diameter of the target lesions dropped to 43% (Range: 5%-75) %)

Part of the data from the Phase 1/2 dose-escalation clinical trial of Gavo-cel (TC-210) for the treatment of patients with refractory mesothelin-expressing solid tumors will be highlighted as part of the oral report of the European Society of Medical Oncology on September 17 , Including the efficacy of gavo-cel in malignant mesothelioma, ovarian cancer and cholangiocarcinoma