With its development program for pleuromutilins, a new class of antibiotics, Nabriva Therapeutics AG has joined the ranks of innovators determined to find new cures to combat diseases caused by life-threatening pathogens. The company, which was spun out in 2006 from Holzkirchen, Germany-based Sandoz GmbH, part of Novartis AG, currently is developing BC-3205 and BC-3781, which are first-generation systemic pleuromutilins, and BC-7013, a topical compound that belongs to a subclass of

It's a story familiar to the biotech industry: Promising-sounding candidates get through Phase I testing sometimes even Phase II proof of concept and then implode, leaving researchers and investors alike scratching their heads. "The question we ask ourselves is 'Why do bad things happen to good drugs?'" said Robert Shorr, CEO at Cornerstone Pharmaceuticals Inc. He and a group of other serial entrepreneurs had a few ideas when they established the Cranbury, N.J.-based company in 2002 "with our

Curemark LLC, a company focused on complex neurological diseases, has managed to move from a Series A financing round to Phase III studies in just two months without having to tap any venture capital money. The Rye, N.Y-based company has begun enrolling pediatric patients in Phase III studies of its proprietary compound for autism, using funds from its recently completed $6.5 million Series A round. Founded in 2004, Curemark had planned to raise $5M in that initial round, which was

Using artificial intelligence, BioVista Inc. created a drug profiling platform that can identify new uses for existing compounds, providing the start-up with dozens of options for internal development as well as partnerships and financing. Charlottesville, Va.-based BioVista began as a research project run by brothers Aris and Andreas Persidis. Aris boasted a biotech background that included co-founding Anadys Pharmaceuticals Inc. and Cellzome Inc., while Andreas brought artificial intelligence

Far from the biotech hubs on the East and West coasts, small start-up KemPharm Inc., based in North Liberty, Iowa, has worked steadily - and with modest funding - over the past three years to move its first product into the clinic. With the recent closing of a $3.8 million Series B round, which brings the company's total investments to $7.6 million, KemPharm is set to submit an investigational new drug application for its first program, KP106 for attention deficit hyperactivity disorder, by

Novation Pharmaceuticals Inc. is hitting the fundraising trail in search of a $10 million Series A round to support partnerships for its mRNA-based drug discovery platform and preclinical work with its first two product candidates. Novation's scientific founders, Tania Kastelic and Dominique Cheneval, discovered while working at Sandoz in the mid-1990s that small molecules could be used to target mRNA and increase or decrease the amount of protein being produced. But their discovery languished

A strep infection may be worrisome for more than just the sore throat that is its calling card. Some researchers believe that in children, strep infection and infectious disease more generally also can cause tics, obsessive-compulsive disorders and a group of disorders collectively known as "pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection" or PANDAS. Strep, so the theory goes, can trigger an autoimmune response that leads to antibodies against brain

The traditional view of acute inflammation is that it ends, more or less, by petering out. But Charles Serhan, professor at Harvard Medical School and the director of its Center for Experimental Therapeutics and Reperfusion Injury, said that a better way to think of it is as a process that is in some ways analogous to apoptosis or programmed cell death: an active cellular process. Serhan has been studying the molecular mechanisms underlying that active process for a decade now. Chief among his

In the U.S., HIV has gone from death sentence to chronic illness, at least for those with access to care. But globally, the disease is anything but contained: The World Health Organization's Joint United Nations Program on HIV/AIDS estimates that in 2007, 2.7 million people were infected with the disease - about 7,000 every day. Given that an HIV infection is, and remains for the foreseeable future, incurable, the best weapon against HIV would be an effective vaccine. The quest for that vaccine

In medical school, students are usually told that half of what they will learn over the next four years is wrong - but their teachers don't know which half. Hepatitis C treatment is, in a way, similar: Roughly half of treated patients respond to the standard treatment of Pegylated interferon alfa plus ribavirin but doctors currently can't predict which half will show a sustained virological response, or SVR, to the often grueling year-long regimen, and which half will end up with nothing to

A study published in the Dec. 3, 2009, issue of Sciencexpress reported that treating chimpanzees with a drug that sequesters miR122 - a microRNA that regulates the expression of liver genes, and which the hepatitis C virus hijacks for its own replication in infected individuals - led to a decrease in viral load of several orders of magnitude that far outlasted the three-month treatment. Though the treatment did not enable the animals to clear hepatitis infection, senior author Henrik Oerum said

Exosomes small vesicles that are shed from cells and contain circulating nucleic acids, or CNAs have been known for some time to play a role in cell-to-cell communication in plants, Brian Durie said. But in animals, they are still "an underappreciated biologic mechanism." But Durie, who is medical director of the International Myeloma Foundation, is working with researchers from the U.S/Germany firm Chronix Biomedical to harness that mechanism for both diagnosis and prognosis of disease. And

Three papers in the July 2, 2009, issue of Nature reported new findings on the genetics of schizophrenia. Together, those studies - from the International Schizophrenia Consortium, the European S-Gene consortium and the Molecular Genetics of Schizophrenia group - investigated the genetic makeup of nearly 15,000 schizophrenic volunteers and nearly 50,000 controls and were a "spectacular success" in identifying risk factors, David Collier, from the S-Gene consortium, told reporters at a press

Even though only about 1 percent of the human genome actually codes for proteins, over the past few years the notion that much of the genome is so-called junk DNA has increasingly been replaced by an understanding that noncoding DNA has important regulatory roles. Accompanying that is a belief that even regulatory DNA, whose roles are not immediately obvious, has roles over longer time scales. But in the Aug. 16, 2009, advance online edition of Nature, researchers from the University of

Despite the fact that hearing loss is a common problem, and one that can lead to enormous social isolation in those affected, research on hearing, both normal and impaired, is something of a stepchild to vision research. But two papers have identified genes that are involved in hearing loss, with one of the newly identified genes, the loxhd1 gene, joining only two other genes to date that are linked to progressive rather than congenital hearing loss. Beyond the specifics of the genes themselves

BCR-ABL otherwise known as the Philadelphia chromosome, the fusion of two kinases that is the root cause of chronic myelogenous leukemia is the most famous fusion gene in cancer. But it is by no means the only one: Gene fusions occur in other hematological cancers, as well as sarcomes and solid tumors including lung and prostate cancers. In a brief report in the Oct. 30, 2009, edition of Science, senior author Arul Chinnaiyan, who is director of the Michigan Center for Translational Pathology

Coaxing a stem cell into the desired adult cell type can be reminiscent of helicopter parenting. Just the right ingredients, at just the right time, are necessary to steer the cell down exactly the path desired by the researchers - whatever the path might be. Two papers out this week describe a method to coax highly pluripotent cells into more specialized blood-forming stem cells that is also popular in certain parts of the helicopter parent world: exposing it to a heartbeat. In parenting

Basic research continues to show just how much remains to be learned about stem cells. A few years down the line, a paper on the factors controlling stem cells in particular is likely to be very different from the one just published. In just June of 2009 at least three papers have turned current wisdom on stem cells and the transcription factors controlling their differentiation on its ear. A paper in the June 25, 2009, advance online edition of Nature showed two transcription factors that are

In an advance that has implications for both cell replacement therapy and cord blood banking, two papers in the Oct. 2, 2009, issue of Cell Stem Cell reported generating induced pluripotent stem cells, or iPS cells, from cord blood. The findings add an additional source of iPS cells to researchers' and maybe, ultimately clinician's arsenals. Like stem cells in general, iPS cells are rapidly being generated from ever-new sources. The 2008 generation of iPS cells from elderly patients with