Non-steroidal anti-inflammatory drugs (NSAIDs) have long been used to treat a variety of inflammatory conditions. Many of these conditions, such as cancer or arthritis, require long term use of the NSAIDs due to the chronic nature of the disease. However, the NSAIDs in current use have toxicities associated with their long-term use that hinder their use for these chronic conditions.
Since its emergence in 2019, COVID-19 infected over 600 million people and over 6 million people have died from the disease. COVID-19 is an infectious disease caused by the SARS-CoV-2 virus. Neutralizing antibodies have been developed to bind to the receptor binding domain (RBD) on the spike (S) protein. Blocking the interaction of the RBD and the ACE2 receptor, is critical in neutralizing the virus. However, the S2 subunit, is also critical for viral infection and entry into human cells.
Albinism (also called achromia, achromasia, or achromatosis) is a congenital disorder characterized by the complete or partial absence of pigment in the skin, hair and eyes due to absence or defect in any one of a number of proteins involved in the production of melanin. Certain forms of albinism are known to be due to mutations in tyrosine metabolism. In oculocutaneous albinism (OCA), pigment is lacking in the eyes, skin and hair. In ocular albinism, only the eyes lack pigment. Patients with albinism experience varying degrees of vision loss associated with foveal h
Retinal degeneration is a deteriorative condition of the human retina caused by the progressive and eventual death of photoreceptor cells. To date, no effective treatment for genetically inherited or age-associated retinal degeneration, which includes a large patient population worldwide, is available.
Epidermal growth factor receptor variant III (EGFRvIII) is a variant of EGFR that is an excellent target for immunotherapy because of its expression in cancer cells and not in normal cells.
Castrate-resistant prostate cancer (CRPC) is characterized by androgen-independent cancer cells that have adapted to the depletion of hormones and continue to grow. Abnormal androgen receptor signaling is known to drive advanced castrate-resistant prostate cancer.
Papillomavirus pseudoviruses that comprise the L1/L2 capsid proteins can package a wide variety of non-viral DNA plasmids and deliver the packaged genetic material to cells. This function makes them attractive candidates as targeted gene delivery vehicles.
Vascular Endothelial Growth Factor-A (VEGF-A) is an angiogenic agent that drives blood vessel formation in solid tumors and other diseases, such as macular degeneration and diabetic retinopathy. Several therapies that target the ability of VEGF to stimulate angiogenesis have been approved. These therapies regulate VEGF-A activity by binding VEGF-A, thereby blocking VEGF-A from binding to its receptor on target cells. This technology utilizes a different approach to regulating VEGF-A activity by providing a VEGF-A protein antagonist that is produced by engineering native VEGF-A protein.
T cell immunotherapy is used in the treatment of various pathologies – including cancers and infections. Current therapies employ chimeric antigen receptors (CARs) consisting of the intracellular fragment of CD3-zeta as the signaling domain with varied combinations of co-stimulatory, transmembrane, spacer/hinge, and extracellular targeting domains. While effective in treating hematological malignancies, CAR T cells need to be activated through T cell receptor (TCR) activation.
Mutation of amino acid 61of the neuroblastoma rat sarcoma viral oncogene homologue (NRAS) is a known driver of oncogenesis in melanoma. Glutamine (Q) to lysine (K) mutation at this position of NRAS is prevalent in approximately 10% of all melanoma cases and associated with aggressive tumors and low patient survival. Therefore, Q61K mutated NRAS is an important candidate for targeted therapies, including cellular immunotherapy.