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Hematological neoplasms: Leukemias, Lymphomas and Myelomas

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LastUpdate Updated on 23/12/2025 [06:45:00]
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COMPOUND FOR DEGRADING USP7 PROTEIN BY MEANS OF TARGETED UBIQUITINATION, PHARMACEUTICAL COMPOSITION THEREOF AND USE THEREOF

Publication No.:  WO2025256654A1 18/12/2025
Applicant: 
SHANGHAI INST OF MATERIA MEDICA CHINESE ACADEMY OF SCIENCES [CN]
SHANDONG LABORATORY OF YANTAI DRUG DISCOVERY [CN]
ZHONGSHAN INST FOR DRUG DISCOVERY SIMM CAS [CN]
\u4E2D\u56FD\u79D1\u5B66\u9662\u4E0A\u6D77\u836F\u7269\u7814\u7A76\u6240,
\u70DF\u53F0\u65B0\u836F\u521B\u5236\u5C71\u4E1C\u7701\u5B9E\u9A8C\u5BA4,
\u4E2D\u79D1\u4E2D\u5C71\u836F\u7269\u521B\u65B0\u7814\u7A76\u9662
WO_2025256654_A1

Absstract of: WO2025256654A1

A compound for degrading USP7 protein by means of targeted ubiquitination, a pharmaceutical composition thereof and the use thereof. The structural formula thereof is shown as formula (I), wherein A is a specific protein ligand in an E3 ubiquitin ligase complex, and L is a divalent linker group between a small molecule ligand of the USP7 protein and the specific protein ligand in the E3 ubiquitin ligase complex. The protein degradation chimera has both USP7 protein inhibitory activity and USP7 protein-degrading activity, can effectively inhibit malignant proliferation of acute lymphoblastic leukemia cells, and thus can be used for diseases associated with abnormal USP7 protein expression.

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Publication No.:  WO2025257171A1 18/12/2025
Applicant: 
IDEOGEN A G [CH]
IDEOGEN A.G
WO_2025257171_A1

Absstract of: WO2025257171A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one nicotinamide phosphoribosyltransferase (NAMPT) inhibitor. Belinostat and the NAMPT inhibitor can either be administered separately or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one NAMPT inhibitor, wherein Belinostat and the NAMPT inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one NAMPT inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

METHODS FOR THE TREATMENT OF CHEMOTHERAPY-INDUCED CARDIOTOXICITY IN A SUBJECT IN NEED THEREOF

Publication No.:  WO2025257167A1 18/12/2025
Applicant: 
INSTITUT NATIONAL DE LA SANTE ET DE LA RECH MEDICALE [FR]
CENTRE HOSPITALIER UNIV DE TOULOUSE [FR]
UNIV PAUL SABATIER TOULOUSE III [FR]
INSTITUT NATIONAL DE LA SANT\u00C9 ET DE LA RECHERCHE M\u00C9DICALE,
CENTRE HOSPITALIER UNIVERSITAIRE DE TOULOUSE,
UNIVERSIT\u00C9 PAUL SABATIER TOULOUSE III
WO_2025257167_A1

Absstract of: WO2025257167A1

The evidences provided by the present patent application demonstrate the therapeutic effect of succinate dehydrogenase inhibition to counteract the cardiotoxicity of chemotherapies used in cancer, such as acute myeloid leukemia and breast cancer. This combination also remarkably exhibits a chemosensitizing capacity of cancer cells. The use of Malonate in patients affected by these pathologies would therefore protect the heart from the toxic side effects of chemotherapy and eliminate the most resistant cells for an ever better risk-benefit ratio for these patients. Accordingly, the present invention relates to a method for the treatment of chemotherapy-induced cardiotoxicity in a subject in need thereof comprising administering to said subject a therapeutically effective amount of a succinate dehydrogenase inhibitor.

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Publication No.:  WO2025257173A1 18/12/2025
Applicant: 
IDEOGEN A G [CH]
IDEOGEN A.G
WO_2025257173_A1

Absstract of: WO2025257173A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one Bcl-2 family protein inhibitor. Belinostat and the Bcl-2 family protein inhibitor can either be administered separately, or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one Bcl-2 family protein inhibitor, wherein Belinostat and the Bcl-2 family protein inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one Bcl-2 family protein inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

COMBINATION THERAPY FOR TREATING CANCER SUCH AS LYMPHOMA

Publication No.:  WO2025257176A1 18/12/2025
Applicant: 
IDEOGEN A G [CH]
IDEOGEN A.G
WO_2025257176_A1

Absstract of: WO2025257176A1

The present disclosure relates to a combination therapy for treating cancer comprising belinostat or a pharmaceutically acceptable salt, tautomer, stereoisomer, mixture of stereoisomers, prodrug, or deuterated analog thereof and at least one glucose transporters (GLUT) inhibitor. Belinostat and the GLUT inhibitor can either be administered separately or in the form of a single composition. Thus, the disclosure relates to a kit comprising Belinostat and at least one GLUT inhibitor, wherein Belinostat and the GLUT inhibitor are in separate formulations and, alternatively, to a composition comprising belinostat and at least one GLUT inhibitor, such as a pharmaceutical composition. It also relates to such kit or such composition for use in the treatment of cancer, preferably for the treatment of lymphoma(s).

NON-INVASIVE MYELODYSPLASTIC SYNDROME DIAGNOSTICS

Publication No.:  WO2025257825A1 18/12/2025
Applicant: 
YEDA RES AND DEVELOPMENT CO LTD [IL]
YEDA RESEARCH AND DEVELOPMENT CO. LTD
WO_2025257825_A1

Absstract of: WO2025257825A1

Non-invasive methods of diagnosing myelodysplastic syndrome (MDS) in a subject comprising applying a trained machine learning model to a set of parameters from the subject, wherein the machine learning model outputs an MDS score and wherein an MDS score beyond a predetermined threshold indicates the subject suffers from MDS is provided.

CAR T-CELLS COMPRISING AN ANTI CD33, AN ANTI CLL1 AND AT LEAST ONE FURTHER CAR ANTI CD123 AND/OR FTL3

Publication No.:  US2025381224A1 18/12/2025
Applicant: 
AUTOLUS LTD [GB]
Autolus Limited
US_2025381224_PA

Absstract of: US2025381224A1

The present disclosure provides a cell comprising: an anti-CD33 chimeric antigen receptor (CAR); an anti-CLL 1 CAR; and an anti-CD123 and/or anti-CAR FLT3 CAR. The cell can be used in the treatment of a disease such as acute myeloid leukemia (AML).

AAV-Mediated Gene Transfer for Retinopathy

Publication No.:  US2025381236A1 18/12/2025
Applicant: 
OCULOGENEX INC [US]
OCULOGENEX INC
US_2025381236_A1

Absstract of: US2025381236A1

The present invention relates generally to gene therapy for treating ailments that can affect vision such as retinal degeneration, retinal dystrophy, macular degeneration, macular dystrophy, ischemic retinopathies, and glaucoma. Embodiments include systems and treatments that use AAV-mediated gene therapy or non AAV-mediated DNA, mRNA, or protein therapy to target all retinal cells. An AAV virion can be introduced (e.g., via intravitreal or subretinal injection) into an eye of an individual, or systemically, to express a heterologous gene product such as BMI1 protein (B lymphoma Mo-MLV insertion region 1 homolog).

IMMUNOTHERAPEUTIC COMPOSITIONS FOR TREATMENT OF GLIOBLASTOMA MULTIFORME

Publication No.:  US2025381266A1 18/12/2025
Applicant: 
VARIATION BIOTECHNOLOGIES INC [CA]
Variation Biotechnologies Inc
US_2025381266_A1

Absstract of: US2025381266A1

The present disclosure provides compositions and methods useful for treating Glioblastoma Multiforme (GBM), e.g., compositions comprising virus-like particles (VLPs) comprising Moloney Murine leukemia virus (MMLV) core proteins and the human cytomegalovirus epitopes, gB and pp65, formulated with GM-CSF, which, at dose of at least 10 μg gB/pp65Gag, reverse dysregulation of anti-HCMV immunity in GBM patients.

TREATMENT METHODS FOR SECOND LINE THERAPY OF CD19-TARGETED CAR T CELLS

Publication No.:  US2025381272A1 18/12/2025
Applicant: 
JUNO THERAPEUTICS INC [US]
Juno Therapeutics, Inc
US_2025381272_PA

Absstract of: US2025381272A1

Provided are adoptive cell therapy involving the administration of doses of cells for treating subjects with certain B cell malignancies, and related methods, compositions, uses and articles of manufacture. The cells generally express recombinant receptors such as chimeric antigen receptors (CARs). In some embodiments, the disease or condition is a large B cell lymphoma (LBCL) relapsed or refractory to first-line chemoimmunotherapy.

HUMANIZED ANTIBODY MOLECULES TO CD138 AND USES THEREOF

Publication No.:  US2025382379A1 18/12/2025
Applicant: 
VISTERRA INC [US]
VISTERRA, INC
US_2025382379_PA

Absstract of: US2025382379A1

Humanized antibody molecules that specifically bind to CD138 are disclosed. The humanized antibody molecules can be used to treat, prevent, and/or diagnose disorders, such as multiple myeloma.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Publication No.:  US2025382301A1 18/12/2025
Applicant: 
ARIZONA BOARD OF REGENTS ON BEHALF OF THE UNIV OF ARIZONA [US]
UNIV OF DUNDEE [GB]
Arizona Board of Regents on Behalf of the University of Arizona,
University of Dundee
US_2025382301_A1

Absstract of: US2025382301A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

Compositions and Methods for Retrieving Tumor-related Antibodies and Antigens

Publication No.:  US2025382349A1 18/12/2025
Applicant: 
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA [US]
The Trustees of the University of Pennsylvania
US_2025382349_PA

Absstract of: US2025382349A1

The present invention includes compositions and methods for retrieving tumor-related antibodies and antigens. In one aspect, the invention includes a method for Sequential Tumor-related Antibody and antigen Retrieving (STAR) which directly and efficiently identifies potent antibodies that can specifically bind to tumor-related antigens on the tumor cell surface. In another aspect, the invention includes a CAR comprising a nanobody, a transmembrane domain, and an intracellular domain, wherein the nanobody is retrieved by a STAR method. In another aspect, the invention includes a CAR T system that targets CD13 and treats acute myeloid leukemia. In another aspect, the invention includes a CAR T system and ADC that targets CDH17 and treats NETs and other types of tumors expressing this antigen, with tolerable toxicities.

SMALL MOLECULE INHIBITORS OF DYRK/CLK AND USES THEREOF

Publication No.:  US2025382277A1 18/12/2025
Applicant: 
ARIZONA BOARD OF REGENTS ON BEHALF OF THE UNIV OF ARIZONA [US]
UNIV OF DUNDEE [GB]
Arizona Board of Regents on Behalf of the University of Arizona,
University of Dundee
US_2025382277_A1

Absstract of: US2025382277A1

This invention is in the field of medicinal chemistry. In particular, the invention relates to a new class of small-molecule compounds having a 6,6-heterocyclic structure (e.g., compounds having a naphthyridine, pyrido-pyridazine, pyrido-pyrazine, quinoline, pyrazino-pyridazine, pyrimido-pyrimidine, quinazoline, quinoxaline or cinnoline ring system) which function as inhibitors of DYRK1A, DYRK1B, DYRK2, DYRK3, CLK1, CLK2, CLK3, CLK4, CDK7, CDK8/19, PI3K, PDGFrA/B, mTOR, WNT, homeodomain-interacting kinases (HIPKs), and/or CMGC kinases leading to inhibition of WNT signaling, and their use as therapeutics for the treatment of Alzheimer's disease, down syndrome, Parkinson's disease, Huntington's disease, diabetes, autoimmune diseases, inflammatory disorders (e.g., airway inflammation, osteoarthritis (e.g., knee related osteoarthritis)), cancer (e.g., glioblastoma, prostate cancer, metastatic breast cancer, metastatic lung cancer, multiple myeloma, secondary metastatic tumors of the brain, colorectal cancer and metastatic colorectal cancer (e.g., metastatic colorectal cancer in the liver)), and other diseases.

MOLECULES THAT ARE VITAMIN D RECEPTOR AGONISTS AND HISTONE DEACETYLASE INHIBITORS

Publication No.:  WO2025255667A1 18/12/2025
Applicant: 
THE ROYAL INSTITUTION FOR THE ADVANCEMENT OF LEARNING/MCGILL UNIV [CA]
THE SIR MORTIMER B DAVIS JEWISH GENERAL HOSPITAL [CA]
THE ROYAL INSTITUTION FOR THE ADVANCEMENT OF LEARNING/MCGILL UNIVERSITY,
THE SIR MORTIMER B. DAVIS JEWISH GENERAL HOSPITAL
WO_2025255667_A1

Absstract of: WO2025255667A1

There is provided a compound of formula I or a pharmaceutically acceptable salt or solvate thereof, (Formula I) wherein W is a 5 or 6 membered heteroaryl group comprising at least one nitrogen atom; X1 is CO or O; X2, is NH or CH2; Y is O or CH2, and n is an integer of from 2 to 5. The compound is particularly useful for the treatment of proliferative diseases such as psoriasis or a cancer selected from the group consisting of leukemia, non-small cell lung cancer, colon cancer, central nervous system cancer, melanoma, ovarian cancer, renal cancer, prostate cancer, and breast cancer.

BCMA-targeted CAR-t cell therapy for multiple myeloma

Publication No.:  EE202500046A 15/12/2025
Applicant: 
LEGEND BIOTECH USA INC [US]
JANSSEN BIOTECH INC [US]
LEGEND BIOTECH USA INC.,
Janssen Biotech, Inc.
DK_202530747_A1

Absstract of: EE202500046A

Provided herein are methods of treating a subject who has multiple myeloma and has received one to three prior treatment(s). Infusions of chimeric antigen receptor (CAR)-T cells comprising a CAR capable of specifically binding to an epitope of BCMA are administered to the subject.

CHIMERIC CHECKPOINT RECEPTOR FOR THE USE IN TREATMENT OF MALIGNANT B-CELL DISEASES

Publication No.:  US2025375522A1 11/12/2025
Applicant: 
UNIV ZU KOELN [DE]
Universit\u00E4t zu K\u00F6ln
US_2025375522_PA

Absstract of: US2025375522A1

The invention is related to a chimeric checkpoint receptor (CCR) fusion protein, a nucleic acid molecule encoding said fusion protein, a vector comprising said nucleic acid molecule, a host cell comprising said nucleic acid molecule and/or expressing the fusion protein, a method for providing said host cell, a pharmaceutical composition comprising said fusion protein, nucleic acid molecule or host cell, and said products for use as a medicament and in the treatment of B cell lymphoma.

COMBINATION THERAPY WITH CLK/DYRK INHIBITORS AND BCL2 INHIBITORS TO TREAT LEUKEMIA

Publication No.:  US2025375442A1 11/12/2025
Applicant: 
MEMORIAL SLOAN KETTERING CANCER CENTER [US]
MEMORIAL HOSPITAL FOR CANCER AND ALLIED DISEASES [US]
SLOAN KETTERING INSTITUTE FOR CANCER RES [US]
BIOSPLICE THERAPEUTICS INC [US]
Memorial Sloan-Kettering Cancer Center,
Memorial Hospital for Cancer and Allied Diseases,
Sloan-Kettering Institute for Cancer Research,
Biosplice Therapeutics, Inc
US_2025375442_PA

Absstract of: US2025375442A1

The present disclosure provides methods for treating leukemia (e.g., AML) using a CLK/DYRK inhibitor and a B-cell lymphoma 2 (BCL2) inhibitor (e.g., venetoclax). Kits for use in practicing the methods are also provided.

SMARCA2/4 INHIBITION AS A STRATEGY TO TREAT TUMORS THAT HARBOR ABERRANT BAF ASSEMBLIES

Publication No.:  US2025375450A1 11/12/2025
Applicant: 
BOARD OF REGENTS THE UNIV OF TEXAS SYSTEM [US]
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
US_2025375450_PA

Absstract of: US2025375450A1

The present disclosure is concerned with substituted quinazoline-2,4-diamines and compositions for the treatment of disorders associated with altered expression of SMARCA2 and/or SMARCA4 such as, for example, cancer (e.g., sarcomas, carcinomas, hematological cancers, solid tumors, breast cancer, cervical cancer, gastrointestinal cancer, colorectal cancer, brain cancer, skin cancer, prostate cancer, ovarian cancer, thyroid cancer, testicular cancer, pancreatic cancer, liver cancer, endometrial cancer, melanoma, gliomas, leukemia, lymphoma, chronic myeloproliferative disorders, myelodysplastic syndrome, myeloproliferative neoplasm, non-small cell lung carcinoma, plasma cell neoplasm (myeloma)). This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

METHODS AND COMPOSITIONS FOR INHIBITION OF IRF4

Publication No.:  US2025376679A1 11/12/2025
Applicant: 
THE UNIV OF NORTH CAROLINA AT CHAPEL HILL [US]
The University of North Carolina at Chapel Hill
US_2025376679_PA

Absstract of: US2025376679A1

The invention relates to the inhibition of expression of interferon regulatory factor-4 (IRF4) using RNA interference, chemically-modified oligonucleotides, and/or chimeric siRNA multivalent combinations. The invention further relates to methods of treating IRF4 related conditions such as multiple myeloma.

PROTEASOME INHIBITOR COMPRISING LACTONE STRUCTURE

Publication No.:  WO2025254458A1 11/12/2025
Applicant: 
VAXCELL BIO CO LTD [KR]
\uC8FC\uC2DD\uD68C\uC0AC \uBC15\uC140\uBC14\uC774\uC624
WO_2025254458_PA

Absstract of: WO2025254458A1

The present invention relates to a novel proteasome inhibitor comprising a digoxigenin-derived lactone ring-based compound as an active ingredient, and can simultaneously exhibit high 20S proteasome inhibitory activity and low normal cell toxicity. The compound has excellent cancer cell selectivity, has pharmacological properties with improved anticancer effects and therapeutic indices, and can be useful for various carcinomas such as multiple myeloma, breast cancer, and hepatocellular carcinoma.

COMBINATION OF ENGINEERED NATURAL KILLER (NK) CELLS AND ANTIBODY THERAPY AND RELATED METHODS

Publication No.:  US2025375524A1 11/12/2025
Applicant: 
INDAPTA THERAPEUTICS INC [US]
Indapta Therapeutics, Inc
US_2025375524_A1

Absstract of: US2025375524A1

Provided herein are methods for treatment and uses involving dosing of compositions containing NK cells deficient in expression of FcRγ chain (g-NK cells) engineered with a recombinant chimeric antigen receptor (CAR) in combination with a monoclonal antibody. Among the provided methods and uses are methods and uses for treating cancer, such as multiple myeloma or lymphoma.

ANTI-HLA-DR IGM ISOTYPE MONOCLONAL ANTIBODY POTENTIATING THE ACTIVITY OF ANTI-CD20 IGG1 OR IGM ISOTYPE ANTIBODIES

Publication No.:  WO2025253079A1 11/12/2025
Applicant: 
CENTRE NATIONAL DE LA RECHERCHE SCIENT [FR]
INSTITUT NATIONAL DE LA SANTE ET DE LA RECH MEDICALE [FR]
UNIV DE MONTPELLIER [FR]
UNIV BREST BRETAGNE OCCIDENTALE [FR]
CENTRE HOSPITALIER REGIONAL ET UNIV DE BREST [FR]
ASSOCIATION POUR LA RECH EN HEMATOLOGIE ONCOLOGIE PEDIATRIQUE HEMOSTASE [FR]
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE,
INSTITUT NATIONAL DE LA SANT\u00C9 ET DE LA RECHERCHE M\u00C9DICALE,
UNIVERSIT\u00C9 DE MONTPELLIER,
UNIVERSIT\u00C9 BREST BRETAGNE OCCIDENTALE,
CENTRE HOSPITALIER REGIONAL ET UNIVERSITAIRE DE BREST,
ASSOCIATION POUR LA RECHERCHE EN HEMATOLOGIE ONCOLOGIE PEDIATRIQUE HEMOSTASE
WO_2025253079_PA

Absstract of: WO2025253079A1

The present invention relates to a novel anti-HLA-DR IgM isotype monoclonal antibody and to the use thereof alone or in addition to anti-CD20 IgG1 or IgM isotype antibodies, in the treatment of B-phenotype lymphoid hemopathies, and in particular of non-Hodgkin lymphoma B, chronic lymphocytic leukemia (CLL) and B-cell malignant lymphoid hemopathies in general.

INDUCING APOPTOSIS

Publication No.:  WO2025255419A1 11/12/2025
Applicant: 
ROSWELL PARK CANCER INST CORPORATION [US]
ROSWELL PARK CANCER INSTITUTE CORPORATION
WO_2025255419_A1

Absstract of: WO2025255419A1

Provided are methods for inducing apoptosis of cells. The cells may be cancer cells (e.g., lymphoma cells). The cancer cells may be resistant to other known therapies, such as, for example, therapy with rituximab, a chimeric anti-CD20 monoclonal antibody. A method can include contacting the cells with a compound having the following structure: (MMRi36).

ANTI-HUMAN TIGIT ANTIBODIES FOR IN VITRO DIAGNOSTICS

Nº publicación: WO2025255297A1 11/12/2025

Applicant:

AGILENT TECH INC [US]
AGILENT TECHNOLOGIES, INC

WO_2025255297_PA

Absstract of: WO2025255297A1

The disclosure provides binding agents (e.g., antibodies) against human TIGIT, as well as kits and methods for using the same (e.g., immunoassays) as part of a companion diagnostic and for other applications. In some aspects, the binding agents described herein may be used in assays for detecting ovarian cancer, squamous cell carcinoma of the head and/or neck, non-small cell lung cancer, cervical cancer, esophageal cancer, melanoma, breast cancer, hepatocellular cancer, colorectal cancer, gastric cancer, renal cell carcinoma, prostate cancer, pancreatic cancer, bladder cancer, lymphoma, metastatic cancer, and/or a solid tumor in, on, or derived from any human tissue or organ.

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