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  • Box 1: eIF4A profiled

    Cover Story: Kinase convergence on eIF4F Box 1. eIF4A profiled. A team at the Memorial Sloan-Kettering Cancer Center has published data in Nature showing that inhibition of the RNA helicase eukaryotic translation …

    Published on 9/11/2014
  • Selectively splicing SMN2

    A three-way collaboration between PTC, Roche and the Spinal Muscular Atrophy Foundation has resulted in orally available, blood brain barrier-penetrant, selective small molecule modulators of SMN2 that are advancing in …

    Published on 9/4/2014
  • Figure 1: Modulating SMN2 splicing

    Cover Story: Selectively splicing SMN2 Figure 1. Modulating SMN2 splicing. Spinal muscular atrophy (SMA) is caused by insufficient expression of functional and stable survival of motor neuron (SMN) protein, leading …

    Published on 9/4/2014
  • Overcoming ibrutinib resistance

    The CDK4 inhibitor palbociclib might help overcome ibrutinib resistance in mantle cell lymphoma by restoring sensitivity to inhibitors of BTK or PI3K.

    Published on 8/28/2014
  • Figure 1: Blocking signaling to overcome Imbruvica resistance in MCL cells

    Cover Story: Overcoming ibrutinib resistance Figure 1. Blocking signaling to overcome Imbruvica resistance in MCL cells. [a] B cell receptor (BCR) stimulation by an extracellular antigen induces Bruton's tyrosine …

    Published on 8/28/2014
  • Table 1: CDK4 and CDK6 or BTK inhibitors in development for cancer

    Cover Story: Overcoming ibrutinib resistance Table 1. CDK4 and CDK6 or BTK inhibitors in development for cancer. At least five cyclin dependent kinase 4 (CDK4) and CDK6 inhibitors and five Bruton's tyrosine kinase (…

    Published on 8/28/2014
  • Fat chance for cancer cachexia

    With no drugs on the market and a pair of recent Phase III failures in cancer-associated cachexia, companies may consider targeting the cause of the wasting disease rather than its symptoms. Two independent academic …

    Published on 8/21/2014
  • Cancer-associated cachexia products in clinical development

    Cover Story: Fat chance for cancer cachexia Table 1. Cancer-associated cachexia products in clinical development. Although two compounds failed in Phase III trials for cancer cachexia, the landscape is still full of…

    Published on 8/21/2014
  • Navigating the new road in psychiatry

    The lack of predictive animal models for neuropsychiatric diseases is arguably the biggest single factor stifling early drug development in the field. To kick-start discovery for diseases such as autism, schizophrenia …

    Published on 8/14/2014
  • Box 1. Once more into the misfoldome

    Cover Story: Dyeing to diagnose preeclampsia Box 1. Once more into the misfoldome. In addition to identifying a method for diagnosing preeclampsia, the report in Science Translational Medicine suggests that the …

    Published on 8/7/2014
  • Dyeing to diagnose preeclampsia

    A Congo red-based urine test that could rapidly confirm a diagnosis of preeclampsia might avoid treatment delays. The method will be tested in an international trial next year and has been licensed by Yale spinout …

    Published on 8/7/2014
  • Fresh air for COPD

    An undisclosed company has picked up an integrin-targeting antibody from UCSF that could address the underlying pathology in COPD by improving airway remodeling via suppression of TGFB1.

    Published on 7/31/2014
  • Box 2: Considerations for compelling target and therapeutic validation

    Cover Story: It is an RNA world Box 2. Considerations for compelling target and therapeutic validation. · Empirical validation that the lncRNA target is expressed in the right place at the right time to impact the …

    Published on 7/24/2014
  • It is an RNA world

    RNA-based drugs have redefined the universe of tractable targets by putting virtually anything that is gene encoded within reach of a disease-modifying agent, turning RNA into the biotech industry's de facto third drug …

    Published on 7/24/2014
  • Table 1: 2014 first-quarter industry activity in RNA therapeutics

    Cover Story: It is an RNA world Table 1. 2014 first-quarter industry activity in RNA therapeutics. Deals involving Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) headline a barrage of activity totaling over $1 billion in…

    Published on 7/24/2014
  • Table 2: Accessible human tissues

    Cover Story: It is an RNA world Table 2. Accessible human tissues. Tissues that are accessible to single-stranded antisense and double-stranded RNAi therapeutics in human patients with exemplary therapeutics and …

    Published on 7/24/2014
  • Table 3: Orphan drug designations

    Cover Story: It is an RNA world Table 3. Orphan drug designations. Companies with RNA therapeutics programs receiving orphan drug designations from the FDA. Source: http://www.fda.gov/; BCIQ: BioCentury Online …

    Published on 7/24/2014
  • Table 4: Delivery platforms

    Cover Story: It is an RNA world Table 4. Delivery platforms. Platforms in clinical development to deliver RNA therapeutics in vivo. Platform Company Program Description Stage Encapsulation platforms Lipid …

    Published on 7/24/2014
  • Box 1: Attributes of the GalNAc-ASGR1 system

    Cover Story: It is an RNA world Box 1. Attributes of the GalNAc-ASGR1 system. · Ligand with nanomolar affinity for receptor · Tissue- or cell type-restricted expression of the receptor · High copy number of …

    Published on 7/24/2014
  • Fixing mitophagy in Parkinson's disease

    Genentech has pulled back the curtain on its efforts in Parkinson's disease and described a new and potentially disease-modifying target involved in the clearance of damaged mitochondria. The Roche unit now needs to …

    Published on 7/17/2014
  • Going immune on beige fat

    Two independent teams at UCSF and Harvard have found an immune-regulated pathway for beige fat formation and a hormone, METRNL, that activates it. Ember Therapeutics has an option to license the molecule, which could …

    Published on 7/10/2014
  • Figure 1: Pathway for driving beige fat formation

    Cover Story: Going immune on beige fat Figure 1. Pathway for driving beige fat formation. Cold stimulus promotes beige fat formation and thermogenesis, but the pathways involved in such processes were unclear. The …

    Published on 7/10/2014
  • Keys to the CAR

    Although companies, investors and academics are all racing to develop chimeric antigen receptor -- based T cells for cancer, numerous scientific challenges still constrain the technology. The way forward will likely …

    Published on 6/26/2014
  • Figure 1: Building a better T cell

    Cover Story: Keys to the CAR Figure 1. Building a better T cell. Various engineering strategies can be used to design T cells to engage tumor-associated antigens and kill tumor tissue while sparing normal tissue.

    Published on 6/26/2014
  • Figure 2: Receptor expression and engagement of antigen

    Cover Story: Keys to the CAR Figure 2. Receptor expression and engagement of antigen. (I) T cell receptors (TCRs) consist of a heterodimer of a- and b-chains that associate with the g-, d-, e- and z-chains of the CD3…

    Published on 6/26/2014

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