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Hepatocellular Carcinoma (HCC) is one of the most common cancers worldwide with largely unfavorable outcomes due to a lack of effective treatment options for patients in the later state of disease. The gold standard of care for HCC patients with intermediate to locally advanced tumors is transcatheter arterial chemoembolization (TACE), a procedure whereby the tumor is targeted both with local chemotherapy and restriction of local blood supply. TACE procedures are often not effective however, and a need exists to identify patients that will respond to TACE.

Problem: Cryopreservation is a process where living biological materials like cells, tissues, and cell therapies (which are susceptible to damage caused by unregulated chemical kinetics) are preserved by cooling to very low temperatures in the presence of specific cryopreservation media that protects the biological material from damage. In order to be used, the biological material ideally should be thawed in a controlled manner that minimizes damage and desirably brings the material back to a viable state.

T cells with T cell receptors (TCRs) for cancer-specific antigens are used for adoptive cell therapy (ACT), wherein a patient’s T cells are redirected against their own cancer. However, these isolated T cells may require further ex vivo manipulation to enhance their anti-tumor activity. The ex vivo manipulation of these T cells, or the selection of less functionally inert T cells, and genetic insertion of tumor specific TCRs may circumvent these limitations.

The HIV-positive population continues to rise despite a worldwide decline in the rates of infection caused by human immunodeficiency virus (HIV).  The HIV virus continues to spread due to a lack of effective vaccines and pre-exposure prophylaxis methods, even though the availability and effectiveness of antiretroviral therapy has helped reduce acquired immunodeficiency syndrome (AIDS)-related deaths. 

The technology is directed to compositions and methods of designing nucleic acid nanoparticles (NANPs) composed entirely of DNA, RNA, or DNA and RNA to achieve desirable immunostimulation and decrease undesirable effects on the immune system by changing the composition of the NANP. Benefits of the invention include the desirable activation of the immune system by these particles to increase the efficacy of vaccines and immunotherapies.

Scientists at NIH have identified a process to select highly tumor-reactive T cells from a patient tumor sample based on the expression of four specific T cell surface markers: programmed cell death protein 1 (PD-1; CD279), 4-1BB (CD137), T cell lg-and mucin-domain-containing molecule-3 (TIM-3), and/or lymphocyte activation gene 3 (LAG-3). After this enriched population of tumor fighting T cells, primarily tumor infiltrating lymphocytes (TIL), is selected and expanded to large quantities, it gets re-infused into the patient via an adoptive cell transfer (ACT) regimen.

Several malignancies associated with a poor prognosis such as lung, pancreatic and colorectal cancers frequently harbor constitutively active KRAS mutants, which play a pivotal role in oncogenesis.  Currently, there are no potentially curative treatments against most mutant KRAS harboring cancers once they become metastatic and unresectable.  Despite intensive efforts to develop potent mutant KRAS inhibitors, none have shown a significant improvement to patients.

The National Institute on Aging's (NIA) Cellular Biophysics Section is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize biological pacemakers.

A common symptom of many heart diseases is an abnormal heart rhythm or arrhythmia. While effectively improving the lives of many patients, implantable pacemakers have significant limitations such as limited power sources, risk of infections, potential for interference from other devices, and absence of autonomic rate modulation.

Adoptive cell transfer (ACT) and T-cell receptor (TCR) therapies use lymphocytes that target somatic mutations expressed by tumors cells to treat cancer patients. One of the challenges of these therapies is the identification and isolation of mutation-specific cells and TCRs. While neoantigen specific cells are relatively abundant in the tumor, they are far less common in peripheral blood, a more accessible source of T cells. 

Retinal Degenerations (RD) are the leading cause of blindness in the United States. The degeneration of the Retinal Pigment Epithelium (RPE) is associated with various types of RD such as Stargardt’s disease, retinitis pigmentosa, choroideremia, Late-Onset Retinal Degeneration (L-ORD), and Age-related Macular Degeneration (AMD). The RPE as a layer of cells in the back of the eye. Therefore, it is essential to maintain the health and integrity of retinal photoreceptors.