With $2.3 million in seed funding in hand, Arginetix Inc. is conducting preclinical studies to advance its small-molecule arginase inhibitors in the crowded fields of pulmonary arterial hypertension (PAH) and erectile dysfunction (ED). The Baltimore-based start-up owes its beginnings to scientific co-founders David Christianson of the University of Pennsylvania and Dan Berkowitz of Johns Hopkins University. Their labs established preclinical proof of concept for arginase inhibition in PAH, ED

With several investigator-initiated studies already in the works, start-up Asphelia Pharmaceuticals Inc. is preparing to begin its own clinical trials to evaluate parasite eggs as a treatment for autoimmune diseases. The technology has its roots in the "hygiene hypothesis," which suggests that the increase in autoimmune diseases, allergies and asthma observed over the past half century - particularly in cold, clean, urban environments - may be attributable to the corresponding decline in early

Their company may be young and, for the time being, mainly virtual, but the folks at Carolus Therapeutics do not shy away from thinking big. It's named after Carolus Magnus, or Charlemagne, who in the first century A.D. founded an empire that eventually encompassed much of Europe. Carolus' scientific co-founders are located in Aachen, Germany, the main residence of the original Carolus. The company's business operations are in the New World, and headquartered in San Diego. Carolus was founded

Alzheimer's disease is "really the toughest nut to crack" in the neuroscience field, said Jeffrey Ives, the recently named CEO of Satori Pharmaceuticals Inc., a 2005 start-up aimed at developing disease-modifying therapies for neurodegenerative disorders, starting with Alzheimer's. A former Pfizer Inc. executive, Ives first began working with Satori co-founders - industry veteran and chemist Mark Findeis and Daphne Zohar, of PureTech Ventures, along with a team of neurodegenerative disease

Building on the work of leading researchers from the Broad Institute of Harvard and MIT, Forma Therapeutics Inc. hopes to discover new ways of targeting cancer and has secured substantial funding in recent days. The Cambridge, Mass.-based company has gotten a good start on that goal, signing a deal worth a potential $200 million with the Novartis Option Fund, one of its investors, to develop compounds against an undisclosed protein-protein interaction target in the field of oncology. Founded

Anaphore Inc. raised $25 million in Series A financing to support preclinical development of its fully human trimeric proteins dubbed Atrimers for immune-mediated diseases and oncology. Atrimers represent a "new product class in the protein therapeutics space," Anaphore's chief business officer, Bruce Steel, said. Katherine Bowdish, CEO of the La Jolla, Calif.-based start-up, explained that trimeric protein-based drugs have three binding domains, offering stronger and longer-lasting binding

An early stage German company developing allosteric inhibitors of myosin is the latest addition to the portfolio of the technology transfer and IP asset management firm Ascenion GmbH. Kinaris Biomedical GmbH, an early stage company established by scientists from Hanover Medical School and the Technical University of Dresden, aims to convert intimate knowledge of the structure and function of myosins well-known molecular motors into new, small-molecule drugs for a range of indications, including

Since transitioning to a drug development company three years ago, Knopp Neurosciences Inc. has been moving at a brisk pace to advance its drug candidate for Lou Gehrig's disease through the clinic. If all goes well with the remaining studies, the Pittsburgh-based company also could see the compound move rapidly through the regulatory process. The FDA has granted fast-track status for the development of KNS-760704 in amyotrophic lateral sclerosis (ALS). The company wasted little time in

Less than two years after spinning out of Korean pharmaceutical company CrystalGenomics Inc., San Diego-based Palkion Inc. is poised to complete preclinical toxicology and move into the clinic with an oral anemia drug. "I had been following CrystalGenomics for a number of years," said Wendy Johnson, president and CEO of Palkion and a venture partner with ProQuest Investments. "I was impressed with the fact that they were doing discovery research - something a lot of companies aren't doing any

Aragon Pharmaceuticals Inc.'s name says it all: The company is targeting the androgen receptor - abbreviated in scientific circles as "AR" - and wants it gone. The technology behind Aragon came from Charles Sawyers, chairman of human oncology at Memorial Sloan-Kettering Cancer Center, and Michael Jung, chemistry professor at the University of California, Los Angeles. Sawyers wanted to understand why men with prostate cancer eventually become resistant to hormone therapy. He traced the problem

Two years after spinning out of the University of Wisconsin, Colby Pharmaceutical Co. is "emerging from underneath the radar," according to CEO David Zarling. The start-up is actively raising a Series A financing that will be used to advance its preclinical pipeline of small-molecule oxidative stress inhibitors for cancer. "We've never really asked anyone for money because we haven't needed it until now," Zarling said. Colby has thus far survived on about $15 million in grant money and a small

Sorrento Therapeutics Inc., with a patented platform for generating fully human monoclonal antibodies, is hoping to fill the void created over the last few years by big pharma's antibody acquisition spree. There are a "limited number of gateway technologies" for the creation of human antibodies, Sorrento president and CEO Antonius "Toni" Schuh explained, adding that most of those technologies have been "locked up." Cambridge Antibody Technology Group plc was bought by AstraZeneca plc for $1.3

With a $7.25 million Series A financing in hand, Acetylon Pharmaceuticals Inc. is advancing toward the clinic with isoform-selective histone deacetylase (HDAC) inhibitors for cancer and other diseases. Histone deacetylases are a hot target in the cancer field, but their potency has thus far come hand in hand with serious side effects. Merck & Co. Inc.'s HDAC inhibitor Zolinza (vorinostat) is approved for recurrent cutaneous T-cell lymphoma (CTCL) but has warnings for pulmonary embolism

What began when serial entrepreneur Paul Schimmel founded Angiosyn Inc. - later sold to Pfizer Inc. for $527 million - is advancing to the next level in the hands of San Diego start-up aTyr Pharma Inc. Schimmel, a scientist with The Scripps Research Institute (TSRI), had a hand in founding Alkermes Inc., Cubist Pharmaceuticals Inc., Repligen Corp., Alnylam Pharmaceuticals Inc. and Sirtris Pharmaceuticals Inc. (now part of GlaxoSmithKline plc), just to name a few. Working with fellow TSRI

As molecules go down biochemical pathways, they need to pass through transition states - normally unstable, high-energy conformations. This is where enzymes come in. They catalyze chemical reactions that are unlikely to occur on their own by binding to targets in the transition state, essentially cheating thermodynamics. Transition state analogues - molecules that resemble transition states, but are more stable - can be very effective enzyme inhibitors, as marketed drugs like oseltamivir