Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics Pipeline Analysis 2018

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a progressive disorder that primarily affects brain and spinal cord (central nervous system). People with MPS III generally do not display features at birth, but they begin to show signs and symptoms of this disorder during early childhood. Affected children often initially have delayed speech and behaviour problems.

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In later stages of this disorder, people with MPS III may develop seizures and movement disorders. Patients with MPS III are also known to have short stature, joint stiffness, or mild dysostosis multiplex. Affected individuals often experience chronic diarrhoea and recurrent upper respiratory and ear infections. People with MPS III may also have hearing loss and vision problems. MPS III is divided into four types; IIIA, IIIB, IIIC, and IIID, which are distinguished by their genetic cause.

Get the detailed analysis @ https://www.pharmaproff.com/report/mps-iiitherapeutics-pipeline-analysis The drug candidates in MPS III pipeline include, but are not limited to, EGT-101, MPS IIIA (Sanfilippo A) Program and LYS-SAF302. Some companies having drugs in the MPS III pipeline are Esteve Pharmaceuticals S.A., Orchard Therapeutics Limited, Lysogene S.A. among others.

p53 Antigen Modulators Therapeutics Pipeline Analysis 2018

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.

Thus, new opportunities in cancer immunotherapy are expected to develop better drug candidates targeting p53, with complete understanding of interactions between p53 and the immune system, to avoid various adverse events. The major challenge is prevention of tissue damage by selectively modulating p53 activity, under pathophysiological conditions that generate redox stress.Thus, new opportunities in cancer immunotherapy are expected to develop better drug candidates targeting p53, with complete understanding of interactions between p53 and the immune system, to avoid various adverse events.

p53 antigen modulator therapies are being widely studied for the development of various target specific cancer therapies. Dendritic cell-derived vaccines, adenoviral p53 vectors, MDM2 inhibitors and small-molecules to reinstate the DNA binding activity of p53 are some commercial approaches as p53 antigen modulator therapies, for the treatment of various indications. Also, research studies have demonstrated that novel therapeutic strategies are being developed to overcome the challenges related to in-depth knowledge of p53 and associated pathways.

Quark Pharmaceuticals Inc. is developing QPI-1002 as a nuclease-resistant, synthetic double-stranded RNA oligonucleotide designed to temporarily inhibit the expression of the pro-apoptotic gene p53, for the treatment of delayed graft function and acute kidney injury. Some of the companies having a therapeutic pipeline of p53 antigen modulators include Innovation Pharmaceuticals Inc., Aprea Therapeutics, and Actavalon Inc.

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The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licencing, grants, technology and others.

Septicaemia Therapeutics Pipeline Analysis 2018

Septicemia, also known as bacteremia or blood poisoning, is characterized by bacterial blood infection. Septicemia occurs as a result of bacterial infection elsewhere in the body, which enters in the bloodstream.

Some common infections that lead to septicemia are urinary tract infections, lung infections, infections in the abdominal area, and kidney infections. The symptoms associated with this disease are fever, nausea and vomiting, reduced urine volume, shock rapid heart rate, very fast respiration, and chills.

Septicemia can lead to severe complications like sepsis, septic shock, and acute respiratory distress syndrome. ContraFect Corporation is in the process of developing Exebacase as a protein which acts as a cell wall modulator for the treatment of septicemia.

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Myelodysplastic Syndromes (MDS) Epidemiology Insights

Myelodysplastic 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.

This syndrome can be classified into three main types: MDS with single lineage dysplasia wherein a single type of blood cell appears abnormal; multilineage dysplasia causing two or more cell types to appear abnormal; and MDS with excess blasts, wherein the patients have a shortage of either red blood cells, white blood cells or platelets. Some prominent risk factors that increase the risk of myelodysplastic syndromes are aging, exposure to industrial chemicals, chemo- or radiation therapy, and smoking.

MDS is an uncommon syndrome with an incidence of 4–5 patients per 100,000 populations. It increases with age and can reach up to 20–50 per 100,000 population, in patients aged over 60 years. According to a 2019 study, the annual incidence of pediatric MDS is approximately 1–4 cases/million, which accounts for less than 5% of childhood hematological malignancies.

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Pipeline Analysis of Pontine Glioma Therapeutics

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. The common signs and symptoms of pontine glioma are squints, swallowing problems, slurred speech, facial weakness, abnormal gait, difficulty in writing and changes in personality and behaviour.

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The only efficient treatment for pontine glioma is radiotherapy; as surgery is not possible due to the tumor’s position in the brain stem. Company like Y-mAbs Therapeutics Inc. is in the process of developing omburtamab for the treatment of pontine glioma.

Pipeline Analysis of Hormone Sensitive Breast Cancer Therapeutics

Breast cancer generally occurs when breast cells grow uncontrollably. Mostly, breast cancer development starts in the duct that carries milk to the nipples. 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. Also, as per the American Cancer Society (ACS), 1 in every 5 woman has cancer cell in their breast tumour with several growth promoting protein receptors on their surface. HER2 (human epidermal growth factor receptor 2) gene plays significant role in the development of breast cancer in most of the cases. Alpelisib is one of the key pipeline drug candidates for hormone sensitive breast cancer pipeline.

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Pfizer Inc., and Novartis AG are major players involved in the development of drugs for hormone sensitive breast cancer. Get the Clinical Trials & Results Report of Hormone Sensitive Breast Cancer Therapeutics at https://www.pharmaproff.com/request-sample/1114

Clinical Studies on Interleukin 8 Inhibitors Therapeutics

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.

Interleukin 8 inhibitors therapies have shown improvements as anti-CXCL8 signalling inhibitors for the treatment of various diseases, including cancer and inflammatory diseases. Studies demonstrated that interleukin 8 can be indirectly targeted by mitogen activated protein kinase (MAPK) inhibitors, phosphatidylinositol-3-kinase (PI3K)/AKT inhibitors, and NF-κB inhibitors while direct targeting can be done by CXCL8 neutralizing antibodies, and other drug therapies such as Reparixin. Potent effects of interleukin 8 inhibitors therapeutics as chemo-modulators and anti-angiogenics are expected to treat various tumors

Bristol-Myers Squibb Company is in the process of developing HuMax-IL8 as a biologic which acts as an interleukin 8 inhibitor for the treatment of solid tumors. Dompé farmaceutici S.p.A. is another key player has interleukin 8 inhibitor pipeline therapeutics.

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Mitogen-Activated Protein Kinase (MAPK) Inhibitors Therapeutics Pipeline analysis

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. MAPK pathway, involves three main kinases, Raf, MEK and ERK, has emerged as a novel target for the development of new cancer therapies with reduced side-effects.

MAPK inhibitors are being developed as small molecules, for the treatment of various types of cancer. They have also showed additional benefits with high effectiveness at lower concentrations in comparison to the ATP-competitive inhibitors, for the cancer therapy.

Mereo BioPharma Group plc is in the process of developing Acumapimod as an oral p38 MAPK inhibitor for the treatment of acute exacerbations of chronic obstructive pulmonary disease (AECOPD). eFFECTOR Therapeutics Inc., Genentech Inc., and Kura Oncology Inc. are some other companies having pipeline of MAPK inhibitors.

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Choroidal Neovascularization Therapeutics Pipeline Analysis 2018

Choroidal neovascularization is a type of medical condition in which new blood vessels are formed from choroid and extend into the subretinal space, or subretinal pigment epithelium, or a combination of both.

The disease can be symptomatized by a painless loss of vision, paracentral or central scotoma, and metamorphopsia. Choroidal neovascularization can be diagnosed by indocyanine green angiography, spectral domain optical coherence tomography, and fluorescein angiography.

People having choroidal neovascularization suffers from loss of vision due to the accumulation of subretinal fluid. Choroidal neovascularization can be treated by angiogenesis inhibitors or anti-VEGF (vascular endothelial growth factors) drugs.

Promedior Inc. is in the process of developing PRM-151 as a cell differentiation modulator for the treatment of choroidal neovascularization. Similarly, Graybug Vision Inc. is also developing GB-102 as a platelet-derived growth factor inhibitor for the treatment of this medical condition. Ohr Pharmaceutical Inc., and Ocugen Inc. are some other key players involved in the treatment of choroidal neovascularization.

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Cell Cycle Inhibitors Therapeutics Pipeline Analysis

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.

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Tolero Pharmaceuticals, Inc., a clinical-stage company focused on developing novel therapeutics for hematologic and oncologic disorders, developing alvocidib – a potent CDK9 inhibitor in combination with cytarabine and daunorubicin. In April 2018, the company presented preclinical data supporting the apoptosis-inducing activity of alvocidib at the American Association for Cancer Research (AACR) Annual Meeting in the U.S., Chicago.

Boehringer Ingelheim International GmbH is in the process of developing volasertib, a cell-cycle kinase inhibitor by acting as a polo-like kinase 1 (PLK1) antagonist for the treatment of AML. ImmunoGen Inc., Pharma Mar S.A., and Eli Lilly and Company are some other major companies involved in the development of cell cycle inhibitor therapeutics for the management of several diseases.