Biopharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery
The March 2014 issue of Biopharma Dealmakers showcases companies with partnering opportunities. This week, find out about how you can collaborate with Sorbent Therapeutics Inc.
CRISPR in the liver Amy Donner doi:10.1038/scibx.2014.447 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 findings show that a CRISPR-based therapeutic can correct a mutation in adult mice with genetic liver disease. Full Text | PDF
Reversing (heart) failure in Friedreich's ataxia Michael J. Haas doi:10.1038/scibx.2014.448 Heart failure accounts for over half the deaths in patients with Friedreich's ataxia, but there are no therapies for the neurodegenerative disease. Now, a team of French researchers has shown that i.v. frataxin gene therapy could prevent or reverse heart failure in a mouse model of the disease. Full Text | PDF
Leveling cancer through IL-15 Kai-Jye Lou doi:10.1038/scibx.2014.449 Findings that link high IL-15 levels in tumors with increased local T cell proliferation and favorable patient outcomes position the cytokine as a robust prognostic and complement ongoing efforts to develop IL-15 as a therapeutic in colorectal cancer and other malignancies. Full Text | PDF
Mired in miR-25 Lauren Martz doi:10.1038/scibx.2014.450 New evidence that miR-25 inhibition can help treat heart failure contradicts earlier results. Although the current study shows that blocking miR-25 restores SERCA2A—and thus improves cardiac contractility—discrepancies between the studies need to be resolved before a therapy can be developed. Full Text | PDF
Homeobox C10 (HOXC10) doi:10.1038/scibx.2014.451 In vitro and mouse studies suggest decreasing HOXC10 methylation could help treat breast cancers resistant to aromatase inhibitors and other estrogen-depleting therapies. Full Text | PDF
X-box binding protein 1 (XBP1) doi:10.1038/scibx.2014.452 Studies in mice and patient samples suggest inhibiting the XBP1 branch of the unfolded protein response could help treat triple-negative breast cancer (TNBC). Full Text | PDF
7,8-Dihydro-8-oxoguanine triphosphatase (NUDT1; MTH1) doi:10.1038/scibx.2014.453 In vitro, cell culture and mouse studies have identified inhibitors of MTH1 that could help treat cancer. Full Text | PDF
CD248 endosialin (TEM1) doi:10.1038/scibx.2014.454 Mouse studies suggest a DNA plasmid encoding a TEM1–tetanus toxoid (TT) fusion peptide could be used as a cancer vaccine. Full Text | PDF
G protein–coupled receptor 39 (GPR39); smoothened (SMO) doi:10.1038/scibx.2014.455 In vitro studies suggest agonizing GPR39 could inhibit the hedgehog pathway to help treat cancer. Full Text | PDF
IL-15 doi:10.1038/scibx.2014.456 Patient studies suggest IL-15 could be useful for treating colorectal cancer. Full Text | PDF
PTK7 protein tyrosine kinase 7 (PTK7; CCK4) doi:10.1038/scibx.2014.457 Studies in mice and patient samples suggest inhibiting PTK7 could be useful for treating lung adenocarcinomas. Full Text | PDF
Glycogen dependent kinase 3 (GSK3) doi:10.1038/scibx.2014.458 Zebrafish and cell culture studies suggest GSK3 inhibition could help treat embryonal rhabdomyosarcoma (ERMS). Full Text | PDF
Adenosine A1 receptor (ADORA1) doi:10.1038/scibx.2014.459 In vitro studies identified a biased ADORA1 agonist that could help treat cardiovascular diseases without common side effects. Full Text | PDF
Dipeptidyl peptidase-4 (DPP-4; CD26) doi:10.1038/scibx.2014.460 Mouse studies suggest a DPP-4 peptide vaccine could help treat type 2 diabetes. Full Text | PDF
Glucosidase-β bile acid 2 (GBA2) doi:10.1038/scibx.2014.461 Mouse studies suggest inhibiting GBA2 could help treat type 1 Gaucher's disease. Full Text | PDF
Iroquois homeobox 3 (IRX3) doi:10.1038/scibx.2014.462 In vitro and mouse studies suggest decreasing IRX3 expression could help treat obesity. Full Text | PDF
Ubiquitin D (UBD; FAT10) doi:10.1038/scibx.2014.463 Mouse studies suggest inhibiting FAT10 could help treat obesity. Full Text | PDF
Nav1.6 (PN4; SCN8A) doi:10.1038/scibx.2014.464 In vitro and mouse studies identified a Nav1.6 inhibitor that could help treat optic neuritis, which is often an initial sign of multiple sclerosis (MS). Full Text | PDF
Hypoxia-inducible factor 1α (HIF1A; HIF1α); notch 1 (NOTCH1) doi:10.1038/scibx.2014.465 Mouse studies suggest stimulating expansion of a vascular endothelial vessel subtype in bone could help treat fractures or age-dependent osteoporosis. Full Text | PDF
Frataxin (FXN; FRDA) doi:10.1038/scibx.2014.466 Mouse studies suggest FXN gene therapy could help prevent or treat heart failure in patients with Friedreich's ataxia. Full Text | PDF
Cystathionase (CTH) doi:10.1038/scibx.2014.467 Studies in mice and human samples suggest dietary supplementation with cysteine could help prevent neurotoxicity in patients with HD. Full Text | PDF
MicroRNA let-7b (MIRLET7B; LET-7B); toll-like receptor 7 (TLR7); transient receptor potential A1 (TrpA1) doi:10.1038/scibx.2014.468 Cell culture and mouse studies suggest inhibiting extracellular MIRLET7B could help treat pain. Full Text | PDF
Detection of Plasmodium falciparum or Trypanosome infection with ultrasound-based separation of red blood cells (RBCs) doi:10.1038/scibx.2014.469 An ultrasound-based separation technique could help detect low levels of parasitemia. Full Text | PDF
Ex vivo model of HIV latency to screen latency-reversing agents doi:10.1038/scibx.2014.470 An ex vivo model of latent HIV in patient T cells could help predict clinical efficacy of latency-reversing agents. Full Text | PDF
Four-gene recombination proficiency score to guide the use of DNA-damaging cancer therapies doi:10.1038/scibx.2014.471 Studies in patient tumor samples have identified a four-gene expression signature that could help determine prognosis and guide treatment for various cancers. Full Text | PDF
Humanized mice to model chronic HBV infection doi:10.1038/scibx.2014.472 Mice with humanized immune and liver cells could be used to model chronic HBV infection. Full Text | PDF
Mice with directed integration of human immunoglobulin transgenes to produce chimeric antibodies with fully human variable domains doi:10.1038/scibx.2014.473 Transgenic mice that produce chimeric antibodies with fully human variable domains could be used to discover therapeutic antibodies. Full Text | PDF
Blood-based DNA methylation signature for progressive supranuclear palsy (PSP) and frontotemporal dementia (FTD) doi:10.1038/scibx.2014.474 Studies in human samples have identified a blood-based methylation signature that could be useful for diagnosing PSP and FTD. Full Text | PDF
Loss-of-function SWI/SNF-related matrix-associated actin-dependent regulator of chromatin subfamily a member 4 (SMARCA4; BRG1) mutations associated with small-cell carcinoma of the ovary, hypercalcemia type (SCCOHT) doi:10.1038/scibx.2014.475 Genetic studies suggest loss-of-function mutations in SMARCA4 could help diagnose SCCOHT. Full Text | PDF
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