Sentences with phrase «small molecule therapies for»

Some research efforts are closer to clinical implementation, including developing improvements in clinical management of rare diseases and trials of small molecule therapies for inherited and mosaic genetic disorders.

Not exact matches

And Xenon, a Vancouver - based biotech, completed a US$ 31 - million private equity financing last April that will allow it to further develop its product pipeline, which consists of small - molecule therapies for select neurological, cardiovascular and metabolic diseases.

Initial results were discouraging: attempts to simply replace the protein therapies with small - molecule drugs that hit the same targets largely failed, says Saeed Fatenejad, a rheumatologist at Pfizer who is responsible for clinical development of tasocitinib.

Green explains that siRNAs must be encapsulated in particles that are different from those used to carry DNA because siRNAs are about 250 times smaller than the DNA molecules usually used for gene therapy.

A fruit fly model of Duchenne muscular dystrophy allowed Ruohola - Baker's lab to rapidly score small molecule therapy candidates for raising the level of sphingosine 1 - phosphate.

Small molecule drugs can be screened or designed to increase telomerase activity exclusively within stem cells for disease treatment as well as anti-aging therapies without increasing the risk of cancer.

Indeed, inhibiting CXCR4 function systemically with the small molecule antagonist plerixafor results in the peripheral mobilization of hematopoetic stem cells, thus mitigating the potential of such therapy for long - term anti-retroviral therapy.

«We are also developing novel small molecules to selectively block the receptor for FnEDA as a potential anti-fibrotic therapy in humans.»

We are currently developing small molecule Nav1.1 activators that increase Nav1.1 currents and interneuron - dependent gamma oscillations in vitro and in vivo to develop novel therapies for conditions with impaired interneuron function, including AD and Dravet syndrome.

The cells generated in the Zeng lab may not only provide a potential unlimited source for cell replacement therapy for Parkinson's disease, but also offer an unprecedented opportunity to develop screening models for assessing small molecule drugs and to clarify the mechanisms of disease.

Yet one thing is clear: Major pharmaceutical manufacturers, originally skeptical about the prospects for large - molecule therapies, have long since joined the race to develop the treatments, acquiring, merging or partnering with small companies working on mAbs and other biologics.

This will fast - track new immune - modulating therapies for cancer patients by discovering and developing small molecule product candidates against immuno - oncology targets.

The mission of the Institute for Applied Cancer Science (IACS) is to apply scientific knowledge of mechanisms driving tumor development and maintenance into the development of impactful small molecule cancer therapies.

Our research will contribute to a better mechanistic understanding of the microbes that live in our body, leading to the discovery of druggable small molecules, new targets for antibacterial therapy and beneficial bacterial strains that can be employed for intervention therapies.

To build upon the encouraging early discoveries, Helmsley renewed and expanded its Crohn's funding for the Institute in 2013 to begin new work with three major aims: 1) continue studies of individual genes to determine how genetic differences between Crohn's patients and healthy individuals contribute to the disease; 2) evaluate promising small molecules in disease - relevant studies and prioritize insights from genetics to help develop novel therapeutics; and 3) begin basic experimentation in animal models with Crohn's disease to provide the data necessary to begin testing new therapies in humans.

The company's lead candidate, GLN - 1001, is a first - in - class, orally available oncolytic small molecule, currently being developed as a potentially disease modifying therapy for glioblastoma by targeting specific vulnerabilities in tumor cells.

In melanoma, the MAPK and PI3K pathways are primary targets for therapy, but other pathways are also explored for inhibition by small molecule compounds.

The platform serves as a potential way to help researchers quickly discover new carriers for a broad range of treatments, from small molecules to gene therapies.

Figure 2: Comparing the patent landscapes for induced pluripotent stem cell (top) and a small - molecule drug candidate (MK - 1775, bottom) highlights need for intellectual property consideration in cell therapy DST — search and analysis conducted in Thomson Innovation and Thomson Data Analyzer (TDA) software from Thomson Reuters; themescape maps produced using TDA.

20 - 30 % of patients with acute myeloid leukemia have mutations of the FLT3 biomarker that make them eligible for several FLT3 - targeted small molecule therapies currently in late stage clinical trials.

Given our extensive experiences in neuronal differentiation of hESCs [6], [7], [8] and the potential application of hESC - derived neurons in cell replacement therapies for neurodegenerative diseases, we designed a set of experiments aimed at developing a hESC - based automated assay for screening small molecules that have differential toxicity to hESC - derived NSCs and their differentiated neural progenies.

Our research will contribute to a better mechanistic understanding of the microbes that live in our gut, leading to the discovery of druggable small molecules, new targets for antibacterial therapy and beneficial bacterial strains that can be employed for intervention therapies.