NF-κB is identified as a family of highly conserved transcription factors, which are responsible for many cellular functions such as regulation of inflammatory responses, apoptosis, cellular growth, and others.
MF is a rare disease identified by the presence of excessive scar tissues in bone marrow that decreases the hematopoietic capacity of the bone marrow. The genetic mutation in the blood stem cells causes MF.
IL-6 is transcribed by IL-6 gene that encodes a cytokine, which functions in inflammation and maturation of B cells. The IL-6 protein can induce fever in patients suffering from autoimmune diseases, thereby acting as endogenous pyrogens.
JAK consists of four intracellular non-receptor tyrosine kinases that help in cytokine mediated signaling through the JAK-STAT pathway. It contains two almost identical phosphate transferring domains in which one domain shows kinase activity and the other regulates the activity of the first via negative inhibition.
Mitochondria are the organelles found in cytoplasm of most eukaryotic cells. They are also known as power-house of the cell. Various mitochondrial-related diseases have been discovered, in which the pathogenesis is not understood well.
Wnt or β-Catenin signaling pathway plays a key role in the development of pluripotent cells and embryonic development. The pathway regulates cell-to-cell interaction, activates blastocyst, accelerates trophoblast development, accelerate chorion-allantois fusion and implantation.
Galectins belong to the family of animal lectins and perform function by interacting with cytoplasmic and nuclear proteins, cell-surface and extracellular matrix glycoproteins and glycolipids to modulate signalling pathways.
Epidermal growth factor receptors (EGFR) are a group of receptor tyrosine kinase (RTK) that plays a major role in cell proliferation, survival and differentiation. In most of the cancer, EGFR are amplified and overexpressed, due to dysregulation, resulting in cancer development.
Endothelin antagonists are drug candidates that acts against endothelin receptors and generate pharmacological actions such as blocking the vasoconstriction and mediate vasodilatation. Endothelin-1 is a peptide which is comprised of 21 amino acids and formed by vascular endothelium.
Mitogen-activated protein kinase (MAPK) are ubiquitous regulators of many cellular functions including cell proliferation, cellular growth, inflammatory responses to stress signals and cell differentiation.
MDM2 protein are powerful oncogene which is overexpressed in various cancers, including breast cancer and sarcoma. There are many small molecule drug candidates that are being developed as MDM2 protein inhibitors as monotherapy or combination therapy for the treatment of various cancers.
Interleukin receptors are a group of cytokine receptors that are expressed by leukocytes. Interleukin receptors play an important role in the functioning of the immune system.
Interleukin 8 is a chemokine produced by various immune cells. They promote angiogenesis, proliferation and development of cancerous cells. The other diseases which are associated with interleukin 8 are depression, schizophrenia, bipolar disorder, migraine, Alzheimer’s disease, colitis, rheumatoid arthritis, multiple sclerosis, asthma, allergic rhinitis, psoriasis, eczema and others.
Hepatocyte growth factor (HGF) is secreted by human pancreatic stellate cells and present predominantly on cancer cells. Thus, HGF inhibitors has emerged as an effective therapy in preventing local tumor growth.
Glycogen synthase kinase 3 beta (GSK-3β) is an attractive target for the treatment of psychiatric disorders and neurodegenerative diseases. Many GSK-3β inhibitors have been developed for the treatment of different central nervous system disorders.
Spleen tyrosine kinase (Syk) is a protein that belongs to the family of tyrosine kinases with an ability to initiate the inflammatory responses, by connecting immune cell receptors to intracellular signaling pathways.
RNA interference (RNAi) is a molecule type that silences the gene and limits the transcription of the mutated gene. Gene silencing is a novel mechanism that inactivates the transcripts of mutated gene, by activating sequence specific RNA degradation process.
Rho kinase inhibitor, also known as ROCK inhibitor, inhibits the rho kinases, a family of small GTP-binding protein. Rho kinases are found to regulate cell motility, proliferation, shape, gene expression, apoptosis, and are also involved in the signalling pathway.
p53 is the tumor suppressor transcription factor that activates to various stimulus, including uncontrolled cell proliferation, oncogene over-expression, and DNA damage. p53 antigen modulators helps in preventing cancer development through regulation of cell cycle and apoptosis.
Cytotoxic T-lymphocyte antigen-4 (CTLA-4) plays important role in the T-cell regulation at an early stage of naive T-cell activation, primarily in the lymph nodes. CTLA-4 has emerged as an attractive cancer immunotherapy as a part of “immune checkpoint blockade”.
Cyclophilin befits in the group of protein that shows peptidyl-prolyl cis-trans isomerase activity, found in all types of cells. In humans, 16 cyclophilins have been identified till date. Cyclophilin A, a member of cyclophilin group, mediate the action of immunosuppressive drugs.
Cell cycle inhibitors include cyclin inhibitors and cyclin-dependent kinases (CDKs), which plays major role in developing new class of anti-cancer therapies. Also, cell cycle inhibitors in combination with chemotherapy, overcome drug resistance and improve cytotoxic efficacy. CDKs are rational targets for cancer treatment, that could restore cell-cycle checkpoints and may induce apoptosis.
Cathepsin K is a protease enzyme which is coded by CTSK gene. The function associated with the gene involves bone remodelling and resorption. Cathepsin K is a member of peptidase C1 protein family and expressed mainly in osteoclasts.
Aurora kinase are proteins that function as mitotic regulators and are aberrantly expressed in cancerous cells. Aurora kinase inhibitors target these aberrantly expressed regulators, provides genetic stability and prevents tumorigenesis.
Angiopoietin (ANG) receptor plays a crucial role in regulation of vascular permeability and pathological vascular remodelling during tumour angiogenesis, metastasis and inflammation. Thus, it has emerged as a new clinical approach for the development of therapeutics which treat cancer and ophthalmological diseases.
Adenosine 5-monophosphate activated protein kinase (AMPK) plays an important role in regulation of homeostasis. Dysregulation of AMPK can result in many diseases which includes obesity and type 2 diabetes. Several studies have also suggested that AMPK are also associated with the development of neurological diseases and cancer.
Androgens are responsible for the development of male characteristics by binding to androgen receptors. Androgen receptor plays an important role in the development and progression of prostate cancer. Thus, it has been an attractive target for the clinical interventions and treatment of prostate cancer.
Alpha-synuclein is a protein that is abundantly found in the brain. The minimal amount of alpha-synuclein is also found in the heart and other tissues. The protein is prominently found at the tips of the nerve cells. It helps in the maintaining supply of the synaptic vesicle in presynaptic terminals. In addition, the alpha-synuclein protein also helps in release of dopamine.
Tagraxofusp, sold under the brand name of Elzonris, is an Interleukin 3 (IL-3) conjugated truncated diphtheria toxin. It is composed of catalytic and translocation domains of diphtheria toxin fused via Met-His linker to a full-length human IL-3.
Cabozantinib, sold under the brand name of Cabometyx and Cometriq, is used for the treatment of medullary thyroid cancer and a second line treatment for renal cell carcinoma. It is a small molecule inhibitor of the tyrosine kinases c-Met and VEGFR2, and also inhibits AXL and RET.
Myeloidysplastic syndromes (MDS) can be described as a group of hematologic malignancies, that occurr due to abnormality in blood-forming cells in the bone marrow. It is a rare blood cancer and can be mild, moderate or severe, in terms of severity.
Akt, also known as PKB, is generally activated in several types of cancers such as breast, gastric, lung, ovary, and pancreatic cancer. Akt inhibitor prevent the functioning of activated form of Akt kinases. Akt kinases are also involved in promoting growth factor-mediated cells that mediates cell proliferation and inhibition of apoptosis through the inactivation of pro-apoptotic proteins, such as mouse double minute 2 homolog (MDM2) and Bcl-2 antagonist of cell death.
Uveal melanoma is the most common primary malignant tumour of intraocular malignancy, arises from melanocytes in the iris, and ciliary body. The most common symptoms of uveal carcinoma include blurred vision, photopsia, visual field loss, visible tumour, pain, and metamorphopsia.
Tuberous sclerosis, also known as tuberous sclerosis complex, is a genetic disorder characterized by the growth of numerous noncancerous (benign) tumors in many parts of the body. It can occur in the brain, skin, kidneys, and other organs, in some cases leading to significant health problems.
Thymic carcinoma, also known as thymomas, is a rare type of cancer that forms in the cells present in the outside surface of the thymus. Thymus is a small organ that lies in the upper chest under the breastbone.
Sézary syndrome, a type of cutaneous T-cell lymphoma, affects Sézary cells present in the skin, lymph nodes and blood. It accounts approximately 3 to 5 percent of cases of cutaneous T-cell lymphoma. Sézary syndrome is more prevalent in males compared to females.
Pontine glioma is a malignant tumour which develops from the brain stem cells. Based on the grade of growth, it is categorized into anaplastic astrocytomas and glioblastoma multiforme (GBM). Pontine glioma is more prevalent in children than adults.
Juvenile myelomonocytic leukemia is a rare disease that occurs mostly in the children who are under four years of age. The abnormal growth of the monocytes results in the accumulation of monocytes in the bone marrow.
Hypopharyngeal cancer, a type of head and neck cancer, develops in hypopharynx. Pharynx is a hollow tube about five inches long, and the bottom part of the pharynx called hypopharynx. Most of the hypopharyngeal malignancy occurs in squamous cells.
Breast cancer generally occurs when breast cells grow uncontrollably. Mostly, breast cancer development starts in the duct that carries milk to the nipples. Some breast cancer cells develop in milk producing glands. As per the National Institutes of Health (NIH), in the U.S., 14.6% of all new cancer cases diagnosed every year are of breast cancer.
Gorlin syndrome, also known as basal cell nevus syndrome, is an inherited genetic disorder characterized by multiple basal cell skin cancer. The common symptoms observed during Gorlin syndrome are calcium deposition in brain, jaw cysts, skeletal changes and developmental disability.
Bone metastases is a common form of cancer of the skeleton that inhabits in bone marrow and stimulates the local bone activity. In this condition, the normal bone homeostasis disrupts which leads to the development of the tumour. Some of the major symptoms observed in patients with bone metastases are bone pain, spinal cord compression, and high blood calcium levels.
Angioimmunoblastic T-cell lymphoma is a rare type of peripheral T-cell lymphoma which is characterized by lymphadenopathy, fever, and arthritis. The signs and symptoms associated with the disease include pleural effusion, arthritis, edema, ascites and pruritic skin rash.
Acquired immunodeficiency syndrome (AIDS) related cancer include those types of cancer which occurs in patients suffering from AIDS. People with AIDS are reported to be highly suspected to the occurrence of lymphomas.
Adrenocortical carcinoma, also known as adrenal cortex carcinoma, is a rare form of cancer that occurs at the cortex layer of the adrenal gland. There are two adrenal glands present in the human body: one at the top of each kidney.