GT-002 

GT-002 is a partial positive allosteric modulator targeting the α3 subtype of GABAA receptors, as well as a specific binder of tumor-associated TF-glycosylated LYPD3. GT-002 mildly enhances GABA-induced chloride currents by binding to the benzodiazepine site of GABAA receptors, thereby alleviating prefrontal hypofunction and improving cognitive, memory and social interaction abilities. GT-002 can be used in research related to schizophrenia spectrum disorders, various squamous cell carcinomas, colorectal cancer, non-small cell lung cancer, and aromatic L-amino acid decarboxylase deficiency^[1][2][3][4].

Human

GT-002 binds with high affinity to the GABA_A receptor benzodiazepine site (K_i = 0.57 nM, IC₅₀ = 0.68 nM), shows minimal off-target binding to the dopamine transporter, and acts as a partial positive allosteric modulator inducing only 10-20% of the Cl⁻ current activation of diazepam^[1].
GT-002 specifically binds the tumor-associated protein/carbohydrate combined epitope on LYPD3 in ELISA, with no binding to non-glycosylated LYPD3 or the target carbohydrate on unrelated proteins^[2].
GT-002 (10^-3-10⁴ ng/mL; 1.5 h) specifically binds to a combined TF-LYPD3 glycopeptide epitope, with an EC₅₀ of 5.5 ng/mL for TF-carrying recombinant LYPD3, and does not bind non-glycosylated LYPD3, Tn-carrying LYPD3, sialylated LYPD3, or irrelevant TF-carrying glycoproteins^[3].
GT-002 (10 μg/mL; 30 min at 4°C) binds specifically to cell surface-expressed TF-glycosylated LYPD3 on LYPD3-F9 recombinant cells and neuraminidase-treated LYPD3-positive CaOV-3 and ZR-75-1 tumor cells, but not to non-glycosylated LYPD3, LYPD3-negative tumor cells, or untreated LYPD3-positive tumor cells^[3].
GT-002 (10 μg/mL; overnight/incubation) binds to TF-glycosylated LYPD3 in a range of human SCC tumor tissues, and shows highly restricted binding to normal human tissues (only skin epidermis), with normal tissue binding re-established by neuraminidase treatment due to sialic acid masking of its epitope^[3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

GT-002 (10 μg/mL; immunohistochemical staining) binds to CaOV-3 ovarian cancer xenograft tumors grown in mice, with 20% of tumor cells showing weak to moderate membranous staining^[3].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

27076  [NCBI]

O95274

LYPD3/C4.4A

ELISA, FACS, Functional assay

Unconjugated

The product can be reconstituted/diluted with sterile PBS or saline.

Please refer to the lot-specific COA for specific buffer information.

Please store the product under the recommended conditions in the Certificate of Analysis.

• [1]. Siebner TH, et al. Acute effects of partial positive allosteric GABA_A receptor modulation by GT-002 on psychophysiological and cognitive measures: protocol for the TOTEMS phase II trial targeting cognitive impairment associated with schizophrenia. Front Psychiatry. 2025;16:1656792. Published 2025 Nov 25.

  [2]. Kehler P, et al. 1347 Targeting of a cancer-associated LYPD3 glycoform for tumor therapy[J]. 2022.

  [3]. Neumann T, et al. Targeting a cancer-specific LYPD3 glycoform for tumor therapy[J]. Frontiers in Drug Discovery, 2023, 3: 1298916.

  [4]. Curry DJ, et al. Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using an MR-Compatible Cannula: 48 Weeks of Follow-Up. J Inherit Metab Dis. 2026;49(2):e70151.