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.
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.
Not exact matches
Researchers at the Center
for Engineering MechanoBiology (CEMB), an NSF Science and Technology Center at the University of Pennsylvania, study plants like this Arabidopsis thaliana to learn how
molecules, cells and tissues integrate mechanics within plant and animal biology, with the aim of creating new materials, biomedical
therapies and agricultural technologies.
Dr. Sharp and project co-leader Joshua Nosanchuk, M.D., professor of medicine at Einstein and attending physician, infectious diseases at Montefiore Medical Center, developed a wound - healing
therapy that uses
molecules of silencing RNA (siRNAs) specific
for FL2.
Professor Ali Tavassoli, who led the study with colleague Dr. Ishna Mistry, explains: «In an effort to better understand the role of HIF - 1 in cancer, and to demonstrate the potential
for inhibiting this protein in cancer
therapy, we engineered a human cell line with an additional genetic circuit that produces the HIF - 1 inhibiting
molecule when placed in a hypoxic environment.
The Mast Cells and Basophiles: Targets
for Innovative
Therapies project, funded by COST funding BM1007 of the EU, brings together European experts focusing on the identification of new target
molecules for drug development, including allergy drugs.
Treatment
for advanced melanoma has seen success with targeted
therapies — drugs that interfere with division and growth of cancer cells by targeting key
molecules — especially when multiple drugs are used in combination.
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.
Dr. Lazaridis says this
molecule therefore should be further examined as a target of
therapy for the disease.
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.
While this is hopeful news
for some patients, more research is needed to further understand not only IL13RA2, but other
molecules in breast cancers that may guide diagnosis, prognosis, and ultimately drug development and
therapy.
«
Molecule common in some cancers, rheumatoid arthritis leads to potential
therapy for both.»
And because their surfaces can be easily tailored to facilitate the attachment of various medicinal
molecules, nanodiamonds have tremendous potential
for the delivery of a vast range of
therapies.
But rather than delivering the entire gene
for the clotting - factor proteins to cells, as most gene
therapies do, the researchers used the viruses to engineer immune - regulating B cells to express a fragment of the clotting factor fused to an immune
molecule called an immunoglobulin.
Advances in understanding the cells and
molecules that transmit pain signals are providing new targets
for drugs that could relieve various kinds of pain — including those poorly controlled by existing
therapies
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.
«But we still have a far way to go: To reliably use this type of photoswitchable
molecules for a photodynamic
therapy of patients, numerous other studies have to be carried out in cooperation with our partners in Kiev.»
Gain an understanding of the specific
molecules and pathways that are the targets
for rationally designed
therapies
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.
She's also created artificial bone materials, targets
for tuberculosis
therapy, cell microarray platforms, and a cell nanoinjector, a device used to inject
molecules into living cells.
For all the advantages of these extended - life
molecules, the researchers predict that they will be supplanted in perhaps a decade by advances in gene
therapy, which will enable people with haemophilia to produce their own clotting factors.
A study investigating the function of the recently discovered enhancer RNA
molecules may open new avenues
for gene
therapy.
The new, extended
therapy combines recombinant factor VIII with a fusion protein that allows the
molecule to remain in the circulation longer — translating into a need
for less frequent treatment.
The
therapy has been in development
for six years and involves a first - in - class
molecule designed by the team.
Recently, scientists have uncovered many of the
molecules in the pathway that control the switch from fetal to adult hemoglobin, opening the door to new
therapies; if you could prevent the switch from happening, or reverse it, and let people with sickle cell disease use fetal hemoglobin
for life, that should reduce symptoms.
However, Professor Auguste's team, discovered the overexpression of intercellular adhesion
molecule - 1 (ICAM - 1) in human TNBC cell lines and tissues, and demonstrated that it is a potential molecular target and biomarker
for TNBC
therapy and diagnosis.
To devise a potential new
therapy, the investigators engineered a population of neural stem cells to express a potent version of a gene called TRAIL, which codes
for a
molecule that activates cell - death - inducing receptors found only on the surface of cancer cells.
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.»
«Given the alarming trend of resistance to our current antimalarial
therapies, this is really an exciting finding,» says Dr. Mota, the senior author of the study, «and we are already working to develop Torin
molecules suitable
for clinical trials of antimalarial activity in humans.»
Neoantigens are
molecules on cell's surfaces that are produced by DNA mutations that are present in cancer cells but not in normal cells, making neoantigens ideal targets
for immune
therapy against cancer, say the scientists.
«Being able to target a specific site in the genome is essential
for the next generation of rationally designed
therapies, and the lessons we've learned have changed the way we design
molecules to target individual genomes,» says Ansari.
Our methodology enables the discovery of unique biomarkers at a single
molecule level
for personalized
therapy approaches.
«It is amazing that we can make one genetic adjustment and positively impact on lifespan and intestinal health, understanding more about the underlying
molecules at work here promises new strategies
for anti-ageing
therapies,» says Dr Jennifer Tullet from the University of Kent.
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.
Our collaborative approach and deep scientific and regulatory understanding of each
molecule type helps us shape the best strategy
for bringing a novel
therapy to market and design custom studies and programs that reduce risk and accelerate timelines.
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.
A biopsy of each patient's cancer will be screened
for 600 genes as well as protein expression to uncover
molecules and pathways that are driving the cancer and to predict a patient's response to chemotherapy or other tailored
therapy.
The SMDC has allowed Gladstone scientists to design
molecules to modulate the functions of proteins or specific cellular phenotypes and, most significantly, screen
for drugs that could be used as
therapies for disease.
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.
We are interested in the mechanistic function and structure of antimicrobial / antifungal proteins and peptides and the identification of target
molecules for the development of new antimicrobial / antifungal
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.
Xavier and his team will also devote time to advancing their research by continuing to develop assays to screen
molecules for potential
therapies, study the microbiome, and compose a map of each cell in the gut in both healthy individuals and IBD patients.