Vertex Pharmaceuticals is engaged in the business of discovering, developing and commercializing
small molecule drugs for the treatment of serious diseases.
ACADIA Pharmaceuticals Inc. is focused on the development and commercialization of
small molecule drugs for the treatment of central nervous system disorders.
It focuses on the development and commercialization of
small molecule drugs for the treatment of central nervous system disorders.
Developing
small molecule drugs for Type II diabetes and Osteoarthritis / inflammation based diseases.
«We could apply the strategy used in this study to quickly identify and design
small molecule drugs for other RNA - associated diseases,» explained study first author Sai Velagapudi, a research associate in the Disney lab.
Not exact matches
Shukla and colleagues discovered that a
small drug molecule called BX795, which is sold to labs
for use in experiments, helped clear HSV - 1 infection in cultured human corneal cells, in donated human corneas, and in the corneas of mice infected with HSV - 1.
«We are very excited about the prospect of using these more physiologically relevant cell - based models
for gene and
small molecule drug discoveries,» says Dr. Andrew Liu, Assistant Professor of Biology at the University of Memphis.
For instance, a team led by Sheng Ding of the Scripps Research Institute in La Jolla is screening for small - molecule drugs that can substitute for the reprogramming gen
For instance, a team led by Sheng Ding of the Scripps Research Institute in La Jolla is screening
for small - molecule drugs that can substitute for the reprogramming gen
for small -
molecule drugs that can substitute
for the reprogramming gen
for the reprogramming genes.
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.
But, says Michael Varney, head of the
small molecule drug discovery group at Genentech, those hiring trends are about to change: «
For us, over the next 3 years, we'll hire more medicinal chemists and more process and scale - up chemists than we will analytical chemists.»
Nanotechnology has unlocked new pathways
for targeted
drug delivery, including the use of nanocarriers, or capsules, that can transport cargoes of
small -
molecule therapeutics to specific locations in the body.
•
For small -
molecule drugs, excretion via the kidneys is the most common path of «decay».
This
small molecule is in clinical trials against cancer and has high specificity and good pharmacological properties, which could warrant its further development as antiviral
drug for treatment of pH1N1 virus infection,» Dr. Oxana Denisova says.
The researchers tested two anti-CK2
drugs for their ability to stimulate the production of new brown fat in mice: a new
small -
molecule CK2 - blocker called silmitasertib (CX - 4945), which is already in clinical trials as a cancer therapeutic; and a more precise next - generation antisense oligonucleotide (ASO)
drug developed in collaboration with Isis Pharmaceuticals, which eliminates CK2 by blocking the RNA instructions cells use to produce it.
In the early 2000s, Danishefsky — who is best known
for synthesizing Taxol (paclitaxel), a
small -
molecule cancer
drug derived from the Pacific yew tree — set his sights on synthesizing EPO that would be identical to that in the body.
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.
In principle, if further tests bear out the protective effects of the two antibodies, then optimized versions of them, or
small -
molecule drugs that hit the same target, could be developed as treatments
for rhinovirus infections.
According to Monk and Ackerman, possible future work includes using the zebrafish model system as a
drug - screening tool to search
for small molecules that may flip that switch.
«They also provide a promising strategy
for identifying
small molecule drugs designed to treat Alzheimer's disease and other neurological disorders.»
«Together, our findings indicate that these molecular regulators, TRIP - Br2 and GATA3, could be viable targets
for small drug molecules that could serve as potential therapeutic agents against obesity,» Liew said.
In early
drug development efforts, Longmore and his colleagues have done some preliminary work looking
for small molecules that may inhibit DDR2 binding to collagen.
«There has been a lot of work on Wnt signaling pathways,
for example, so there are a lot of
small -
molecule drugs that could be tested to trigger a stem cell to mature into a neuron.»
The researchers say the next step is to develop
small molecules or
drugs that regulate p75 NTR to reproduce this effect and potentially serve as a therapeutic intervention
for obesity and metabolic syndrome.
With that goal in mind, Xue and his team joined forces with Chen Zhao, MD, PhD, UI assistant professor of pathology, and used their understanding of CML genetics to look
for small molecules or
drug compounds that might be able to eradicate the leukemia stem cells.
The process of
drug discovery has been dominated in recent years by the screening of large libraries of structurally diverse
small molecules for effects on target proteins associated with disease.
A division of preclinical innovation will house a $ 44 million set of mostly intramural programs, including
small -
molecule screening and
drug development
for rare diseases.
Interestingly, the dominant model
for the mechanism by which a
small molecule like a
drug fits into the active site of a disease - promoting enzyme and modulates its activity is that of a «lock and key».
Arguably, the most economical and efficient strategy
for development of
small molecules with regenerative medicine applications is to use non-mammalian animal models
for both target - and phenotype - based
drug discovery.
Hopefully, this knowledge, coupled to utility of the mAGES system, will spark the search
for small molecule drug combinations which can safely and efficiently modulate the involved pathways and promote astrocyte to NSC conversion.
The consolidation of these efforts in an independent initiative will allow faculty from the CMI to lead a focused investigation in
small molecule drug discovery
for multiple therapeutic applications.
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.
The facility maintains a
small molecule library of ~ 100,000
drug - like
small molecules for high - throughput screening and provides access to liquid handling robotics and a multilabel plate reader.
UniChem supports the integration of high - throughput screening data on
small molecules with chemical libraries and chemical resources
for drug discovery and optimization.
Scientists can screen collections of
small organic
molecules — the type of
molecules needed
for pill - based
drugs — to find any that compete with the peptides by binding tightly to Gα proteins and preventing them from transmitting signals.
In 1994, Schreiber and co-workers discovered that the proteins FKBP12 and mTOR are the simultaneous targets of the
small molecule rapamycin, now approved
for use as an immunosuppressant
drug given to patients after organ transplantations to help prevent rejection.
I wish them well on developing (1) an antibody to detect and image glucosepane in animals (2) a
small molecule drug or enzyme (catabody) to remove the glucosepane (3) luck in finding a development partner
for this hypothetical glucosepane breaker.
Orders of magnitude more sensitive than conventional Raman microscopy, they allow mapping of 3D distributions of
small molecules, such as metabolites and
drugs, as well as tumor identification in tissues, which open many exciting new possibilities
for biology and medicine.
An emerging strategy in early
drug discovery entails using fragments, i.e.
molecules smaller than conventional
drug candidates, as starting points
for design of novel therapeutic compounds.
Because LINGO1 has be shown to have a negative impact on neuronal development and survival, inibiting it with a
small molecule drug may provide an effective approach
for developing a treatment
for the severe forms of essential tremor.
In another sign of growing pharmaceutical industry interest in targeted protein degradation, Celgene will pay Vividion Therapeutics $ 101 million in a four - year pact to develop
small -
molecule drugs for cancer, inflammation, and neurodegenerative diseases.
Because SLC6A14 is essential
for tumour growth yet dispensable
for normal development and tissue maintenance,
small molecules that block amino acid import through this transporter could be effective and selective anti-cancer agents, particularly as components of rational
drug combinations.
Then
for postdoctoral research I moved to Yale University, where I worked in a lab that looked at all kinds of different disease areas using the approaches of a field called chemical biology, which is using
small molecules — chemicals — to probe biological systems in the hopes of developing compounds that could be new
drugs.
These alternate strategies include using
small -
molecule drugs that affect RNA metabolism or protein stability, as well as administering modified viruses
for therapeutic gene delivery (see «Getting a fix on SMA»).
By default, that's already the case
for small -
molecule drugs such as the ones under development at Repligen and Roche.
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.
Reykjavik, ICELAND, January 13, 2005 — deCODE genetics (Nasdaq: DCGN) has submitted an investigational new
drug application (IND) to the U.S. Food and Drug Administration (FDA) for DG041, a novel, first - in - class, orally - administered small molecule for the treatment o
drug application (IND) to the U.S. Food and
Drug Administration (FDA) for DG041, a novel, first - in - class, orally - administered small molecule for the treatment o
Drug Administration (FDA)
for DG041, a novel, first - in - class, orally - administered
small molecule for the treatment of...
The ability to encapsulate
small -
molecule therapeutic compounds into vaults is critical to their development
for drug delivery.
PTC's GEMS technology exploits the regulatory mechanisms found in the untranslated regions of messenger RNA
for the identification of
small -
molecule drugs that can treat diseases by selectively increasing or decreasing the expression of key proteins by modulating post-transcriptional mechanisms.
Abbreviations: Aβ, amyloid β - peptide; AD, Alzheimer's disease; ALS, amyotrophic lateral sclerosis; Ambra1, activating
molecule in Beclin -1-regulated autophagy; AMPK, AMP - activated protein kinase; APP, amyloid precursor protein; AR, androgen receptor; Atg, autophagy - related; AV, autophagic vacuole; Bcl, B - cell lymphoma; BH3, Bcl - 2 homology 3; CaMKKβ, Ca2 + - dependent protein kinase kinase β; CHMP2B, charged multivesicular body protein 2B; CMA, chaperone - mediated autophagy; 2 ′ 5 ′ ddA, 2 ′, 5 ′ - dideoxyadenosine; deptor, DEP - domain containing mTOR - interacting protein; DRPLA, dentatorubral pallidoluysian atrophy; 4E - BP1, translation initiation factor 4E - binding protein - 1; Epac, exchange protein directly activated by cAMP; ER, endoplasmic reticulum; ERK1 / 2, extracellular - signal - regulated kinase 1/2; ESCRT, endosomal sorting complex required
for transport; FAD, familial AD; FDA, U.S. Food and
Drug Administration; FIP200, focal adhesion kinase family - interacting protein of 200 kDa; FoxO3, forkhead box O3; FTD, frontotemporal dementia; FTD3, FTD linked to chromosome 3; GAP, GTPase - activating protein; GR, guanidine retinoid; GSK3, glycogen synthase kinase 3; HD, Huntington's disease; hiPSC, human induced pluripotent stem cell; hVps, mammalian vacuolar protein sorting homologue; IKK, inhibitor of nuclear factor κB kinase; IMPase, inositol monophosphatase; IP3R, Ins (1,4,5) P3 receptor; I1R, imidazoline - 1 receptor; JNK1, c - Jun N - terminal kinase 1; LC3, light chain 3; LD, Lafora disease; L - NAME, NG - nitro - L - arginine methyl ester; LRRK2, leucine - rich repeat kinase 2; MIPS, myo - inositol -1-phosphate synthase; mLST8, mammalian lethal with SEC13 protein 8; MND, motor neuron disease; mTOR, mammalian target of rapamycin; mTORC, mTOR complex; MVB, multivesicular body; NAC, N - acetylcysteine; NBR1, neighbour of BRCA1 gene 1; NOS, nitric oxide synthase; p70S6K, ribosomal protein S6 kinase - 1; PD, Parkinson's disease; PDK1, phosphoinositide - dependent kinase 1; PE, phosphatidylethanolamine; PI3K, phosphoinositide 3 - kinase; PI3KC1a, class Ia PI3K; PI3KC3, class III PI3K; PI3KK, PI3K - related protein kinase; PINK1, PTEN - induced kinase 1; PKA, protein kinase A; PLC, phospholipase C; polyQ, polyglutamine; PS, presenilin; PTEN, phosphatase and tensin homologue deleted from chromosome 10; Rag, Ras - related GTP - binding protein; raptor, regulatory - associated protein of mTOR; Rheb, Ras homologue enriched in brain; rictor, rapamycin - insensitive companion of mTOR; SBMA, spinobulbar muscular atrophy; SCA, spinocerebellar ataxia; SLC, solute carrier; SMER,
small -
molecule enhancer of rapamycin; SMIR,
small -
molecule inhibitor of rapamycin; SNARE, N - ethylmaleimide - sensitive factor - attachment protein receptor; SOD1, copper / zinc superoxide dismutase 1; TFEB, transcription factor EB; TOR, target of rapamycin; TSC, tuberous sclerosis complex; ULK1, UNC -51-like kinase 1; UVRAG, UV irradiation resistance - associated gene; VAMP, vesicle - associated membrane protein; v - ATPase, vacuolar H + - ATPase; Vps, vacuolar protein sorting
Further the Meiler laboratory develops a structure - based
drug design module
for ROSETTA and docking protocols particularly suited
for docking
small molecules into comparative models.