For nucleic-acid based therapeutics to deliver on their promise, they first will have to be delivered themselves - into the cells where they are meant to go. And that delivery issue, Gretchen Unger said, "has been the bane of nucleic acid commercialization, including RNAi." In the June 8, 2009, issue of the Journal of Clinical Investigation, Unger, who is president of Minneapolis-based start-up GeneSegues Inc., and colleagues from GeneSegues and the University of Minnesota, described a

The ultimate goal in HIV/AIDS work is a vaccine that can prevent HIV infection, yet the virus' wicked mutations and rapid variability have stymied progress at nearly every turn, so much that the AIDS Healthcare Foundation in 2008 sought to curtail federal spending on the vaccines effort. Yet UK firm PepTcell Ltd., unfazed by those past failures, is forging ahead with its own HIV vaccine approach designed to target specifically those regions of the virus that remain unchanged from variant to

With the so-called "patent cliff" looming on the horizon, the inhalable therapeutics market has become "one of the fastest growing spaces," said Rob Neville, executive chairman of 2007 start-up Savara Pharmaceuticals. "Companies are seeking new formulations and new routes of delivery," he said, and Savara's NanoCluster formulation technology, originally developed by company founder Cory Berkland at the University of Kansas, is designed as a next-generation pulmonary delivery platform aimed at

At a time when some small biotechs are having a harder time securing early stage partnerships, Adimab Inc. pulled off two major drug discovery deals in June 2009 and is in ongoing discussions with many of the top 15 drugmakers for possible future collaborations. Adimab founder and CEO Tillman Gerngross said, "We expect this momentum to continue for some time." Pretty good for a firm founded only in 2007 with a simple business model to deliver yeast-based antibodies with greater speed and in

After five years of operating in stealth mode, Tokai Pharmaceuticals Inc. stepped into the spotlight with preclinical prostate cancer data presented at AACR and a $22 million Series D financing. Cambridge, Mass.-based Tokai was founded in 2004 by venture firm Apple Tree Partners. Seth Harrison, managing general partner of Apple Tree, said his firm wanted to build an in-licensing company around the endocrinology network of Scott Chappel, former chief scientist at Serono Inc. The two founded

Versartis Inc. is off to a fast start since its founding, with enough money in hand to get at least one of its preclinical products for diabetes and growth hormone deficiency into the clinic in 2010. The Redwood City, Calif.-based company just closed a Series A financing that could bring in as much as $16 million, which will go toward the company's goal of moving one of its product candidates into the clinic. Versartis, a joint venture between Amunix Inc. and European venture capital firm

With a new take on a classic drug discovery technology, 2008 start-up MabCure Inc. is on a mission to improve cancer treatment by developing monoclonal antibodies against difficult-to-pinpoint tumor-specific antigens for the diagnostics market. "If caught early, cancer is 90 to 95 percent curable," said Amnon Gonenne, the company's president and CEO, adding that the challenge in detecting cancer early is the lack of clearly identified cancer markers. Take prostate cancer, for instance. "There

Jerry Colca, president and chief scientific officer at Metabolic Solutions Development Co., thinks diabetes drug developers are too "hung up" on targeting PPAR-gamma. The PPAR-gamma receptor is thought to be the primary target of the thiazolidinediones, a class of insulin-sensitizing diabetes drugs that includes the blockbusters Actos (pioglitazone, Takeda Pharmaceutical Co. Ltd.) and Avandia (rosiglitazone, GlaxoSmithKline plc). "Most of the drug industry believes the way these drugs actually

"When one door closes, another opens" could easily be the motto for the biotech industry; even as companies tighten their belts in response to economic challenges, those restructuring moves have created new opportunities in the space. One such venture is Zenobia Therapeutics Inc., which emerged last summer after ActiveSight, the San Diego-based division of Rigaku Americas Corp., ended some of its research work in a cost-cutting move. But only weeks before the doors closed on that research

Since its founding last spring, Archer Pharmaceuticals Inc., a company focused on Alzheimer's products, had one financing round, the amount of which was not disclosed. But it was enough to launch the firm. And while the company believes it can carry its lead Alzheimer's products through an investigational new drug application, Archer Pharma will need $25 million to get from Phase I to the end of Phase II in U.S. patients, Michael Mullan, CEO and chief scientific officer of Archer, said. The

GliaMed Inc. is getting a makeover, complete with a new CEO, new company headquarters in the Boston area, a new website that recently went live and the development that the company perhaps is most proud of a stem cell-like product for wound healing that is attracting attention from potential investors. Founded in 2001, the company is focused on developing compounds called regenerative immunophilin ligands, or RILs, that have been shown to regenerate skin and other tissue in animal models

Varinel Inc. is advancing through preclinical studies in a variety of neurodegenerative disease models with a pipeline of multifunctional small molecules built on an iron chelator core. Chelators are small molecules that bind to metal ions. Iron chelators such as Novartis AG's Exjade (deferasirox) bind and remove excess iron that can accumulate after blood transplants in patients with sickle cell disease, anemia, myelodysplastic syndromes and other conditions. But the iron chelators available

With $30 million of Series A financing in its coffers, Calixa Therapeutics Inc. is gearing up for a Phase II trial of CXA-101, a broad-spectrum cephalosporin antibiotic that has shown potent anti-pseudomonal activity. San Diego-based Calixa was founded in late 2007 by antibiotic expert James Ge and serial entrepreneur Eckard Weber. The two have a long history of working together at antibiotic firms funded by Domain Associates, where Weber serves as a partner. In the early part of the decade

While navigating the curves of La Jolla, Calif.'s Coast Boulevard, Jay Lichter, managing director of Avalon Ventures, experienced an intense episode of vertigo. After a trip to the emergency room, he found himself in the office of Jeffrey Harris, a practicing physician and chief of the division of otolaryngology at the University of California, San Diego. Harris diagnosed Lichter with Meniere's disease, a disorder of the inner ear that affects 500,000 to 700,000 Americans and causes vertigo

For several decades, chemists have used a process known as deuteration to understand how a drug candidate is metabolized or eliminated from the body. But scientists at Auspex Pharmaceuticals have taken that process to a whole new level. Vista, Calif.-based Auspex is working to develop drugs that last longer in the bloodstream using traditional drug development as well as technology adapted from a decades-old analytical tool. The company, which began focusing on deuteration about five years ago

Biokine Therapeutics Ltd. received a $1.2 million research grant and initiated its first clinical trial with lead compound BKT140, a selective antagonist of the chemokine receptor CXCR4. The grant which was awarded by the Office of the Chief Scientist of the Ministry of Industry, Trade and Labor of Israel brings Biokine's fundraising total to slightly more than $5 million to date. That total includes seed financing provided by the company's founders and the Weizmann Institute of Science

Dynamix Pharmaceuticals, a newly founded biotech still in the discovery stage of research, has secured a corporate partner for up to two drug candidates and closed a $10 million financing round - proof, it said, that some funding still is flowing to early stage projects even in this economy. "It's difficult but it's doable," said Oren Becker, president and CEO of Dynamix. Becker, who founded Rehovot, Israel-based Dynamix in February 2009, said that the cash needs of a start-up tend not to be

Since its founding less than a year ago, San-Diego-based Avineuro Pharmaceuticals Inc. has moved quickly to advance two major programs into the clinic. The privately owned company, which is focused on neurology and psychiatry, announced Tuesday it has initiated Phase I studies of a potential treatment for schizophrenia (AVN-211) and is about to move its molecule for Alzheimer's disease (AVN-101) into the clinic as early as next week. Avineuro licensed its two main compounds from ChemDiv Inc

Less than a year after spinning out of Verus Pharmaceuticals Inc., Meritage Pharma Inc. advanced its oral viscous formulation of the corticosteroid budesonide into a Phase IIb trial for pediatric eosinophilic esophagitis. Budesonide is the active ingredient in AstraZeneca plc's Pulmicort, an inhaled powder or suspension for asthma, and Rhinocort, a nasal spray for allergies. Together the products earned $1.8 billion in 2008, although a licensed generic of Pulmicort is set to be launched by Teva