Machine Learning and/or Neural Networks to Validate Stem Cells and Their Derivatives for Use in Cell Therapy, Drug Delivery, and Diagnostics

Many biological and clinical procedures require functional validation of a desired cell type. Current techniques to validate rely on various assays and methods, such as staining with dyes, antibodies, and nucleic acid probes, to assess stem cell health, death, proliferation, and functionality. These techniques potentially destroy stem cells and risk contaminating cells and cultures by exposing them to the environment; they are low-throughput and difficult to scale-up.

Novel Human Immunogenic Epitopes of the Human Endogenous Retrovirus ERVMER34-1

Summary:

The National Cancer Institute (NCI) seeks research co-development partners and/or licensees for the clinical translation of novel peptide-based therapeutic cancer vaccines derived from ERVMER34-1, a human endogenous retrovirus (HERV) antigen, offering a unique opportunity to address a significant unmet need in the treatment of various carcinomas.

Enhancing Activity of Bispecific Antibodies in Combination with Ibrutinib for the Treatment of Cancer

This technology includes the combination of a kinase inhibitor (specifically ibrutinib) with a bispecific antibody (specifically a CD19/CD3 bispecific antibody) to be used to treat cancer. CD19/CD3 bispecific antibodies (bsAbs) can be used to recruit endogenous T cells against CD19+ tumor cells via the formation of cytolytic synapses. lbrutinib, a BTK inhibitor, has been shown to normalize T cell dysfunction characteristic of CLL.

Antibody Targeting of Cell Surface Deposited Complement Protein C3d as a Treatment for Cancer

This technology includes monoclonal antibodies (mAb) that specifically and with high affinity bind the final complement components C3dg and C3d (subsequently referred to as C3d), which can be used to kill tumor cells that carry C3d on their cell surface. We show that tumor cells of patients treated with the therapeutic anti-CD20 mAb ofatumumab carry C3d on the cell surface and can bind and be killed by addition of anti-C3 mAbs. In contrast, further addition of more ofatumumab has only minimal effects.

Functions and Targets of Therapeutic MicroRNAs to Treat and Diagnose Cancer

This technology includes a method to identify potentially therapeutic microRNAs in cancer, particularly squamous cell carcinoma of the head and neck (HNSCC). This approach first utilizes a large and publicly available expression dataset, which is then validated by a smaller independent dataset to determine deregulated microRNAs expression. These results are then intersected with in vitro functional anti-proliferative screening data to select for microRNAs that play a functional tumor suppressive role and likely serve as therapeutic targets.

A New Molecular Scaffold for Targeting hRpn13 as a Treatment for Cancer

This technology includes a new chemical scaffold (with lead compound XL5) against hRpn13 that induces apoptosis, which may have clinical efficacy against cancer. The structure of XL5-conjugated hRpn13 guided the design of XL5-PROTAC degrader compounds that exhibit greater efficacy than previous hRpn13-targeting compounds, as evaluated by selectivity for hRpn13, induction of apoptosis, and loss of cell viability. In cells, XL5-PROTACs revealed the presence of a truncated hRpn13 product that binds to proteasomes and is selectively degraded by XL5-PROTACs.

Systemic CRISPR Therapy for the Treatment of Inherited Diseases

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.
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