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  • 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
  • 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
  • Breaking MYC metabolism

    The metabolic enzyme PRPS2 could represent a tractable way to modulate the notoriously undruggable oncogene MYC. The findings from a UCSF team are licensed to Effector Therapeutics and provide a clue as to how the …

    Published on 6/19/2014
  • Nav-i-gating antibodies for pain

    Nav1.7 entered the scene a decade ago as a pain target with wide-ranging analgesic effects, but it has been difficult to selectively target. Now, a team at Duke has succeeded with an antibody that blocks the channel in …

    Published on 6/12/2014
  • Table 1: Nav1.7 small molecule inhibitors and antibodies

    Cover Story: Nav-i-gating antibodies for pain Table 1. Nav1.7 small molecule inhibitors and antibodies. Specifically blocking the Nav1.7 (SCN9A) voltage-gated sodium channel subtype suppresses pain. At least six …

    Published on 6/12/2014
  • Alas, porphyria

    Treatment of acute porphyrias has long relied on hemin infusions, but the therapy has many drawbacks. A team led by Alnylam has created an siRNA therapeutic that reduces disease symptoms in mice. The company still needs…

    Published on 6/5/2014
  • Personalized medicine gets personal

    Epithelial cancers are the most common malignancies, but immunotherapy development has been stymied because the low mutation frequency of these tumors elicits muted mutation-specific T cell responses. An NCI team has …

    Published on 5/29/2014
  • Ob(nox)ious fibrosis

    Although the most advanced drug for idiopathic pulmonary fibrosis has shown a mortality benefit, the effects are modest and there remains room for improvement. New preclinical findings suggest inhibiting NOX4 could both…

    Published on 5/22/2014
  • Table 1: Select clinical-stage compounds that target epigenetic regulators

    Cover Story: Chromatin's rising tide Table 1. Select clinical-stage compounds that target epigenetic regulators. The availability of new research tools and data on epigenetic regulators has driven the development of …

    Published on 5/15/2014
  • Chromatin's rising tide

    At the SciBX Summit on Innovation in Drug Discovery, participants laid out a road map for how to capitalize on the full range of possible chromatin targets in and beyond oncology. They determined that industry and …

    Published on 5/15/2014
  • Figure 1: Blocking androgen signaling in castration-resistant prostate cancer

    Cover Story: BETting on OncoFusion Figure 1. Blocking androgen signaling in castration-resistant prostate cancer. Nearly all pathways associated with the transition from androgen-dependent to castration-resistant …

    Published on 5/8/2014
  • BETting on OncoFusion

    Medivation has licensed a portfolio of BET bromodomain inhibitors that could have better efficacy than the company's castration-resistant prostate cancer drug Xtandi enzalutamide. Medivation got the molecules from …

    Published on 5/8/2014
  • Peeking inside AMD

    Conflicting results from prior studies made it unclear whether IL-18 was a cause of or a potential therapy for age-related macular degeneration. A tiebreaker study suggests the latter, showing that the cytokine was at …

    Published on 5/1/2014
  • CRISPR in the liver

    Although the therapeutic utility of CRISPR-based approaches has yet to be demonstrated, venture dollars are flowing into new companies developing the platform. Proof of concept may come faster than expected as new …

    Published on 4/24/2014
  • Contaminating cancer genomes

    Two European teams have pinpointed a DNA-protective enzyme as a chemically tractable target whose inhibition kills cancer cells by accelerating DNA damage. Both groups have identified small molecule inhibitors of the …

    Published on 4/17/2014
  • Pre-EMP-tive strike against GBM

    UCLA researchers have treated glioblastoma in mice by inhibiting EMP2. The findings open up a new indication for spinout Paganini, which has a mAb against the target in development for triple-negative breast cancer.

    Published on 4/10/2014
  • Table 1: EMP2's downstream crowd

    Cover Story: Pre-EMP-tive strike against GBM Table 1. EMP2's downstream crowd. University of California, Los Angeles researchers have shown that in mouse models of glioblastoma multiforme (GBM) and breast cancer, …

    Published on 4/10/2014
  • Critical mass in diabetes

    A University of Bremen group has found a new target-STK4-that can increase beta cell mass and may provide an alternative to invasive islet cell transplants to treat diabetes. The team will need to rule out long-term …

    Published on 4/3/2014

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