Last week, Newton, MA-based AesRx announced that it has begun human trials of its lead drug, Aes-103, to treat sickle cell disease. To biotech observers, this may not have seemed like a big deal—sickle cell is a rare disease that affects only about 75,000 people in the U.S.—but for AesRx CEO Stephen Seiler, just getting this far is a major achievement. Seiler founded the company in 2008 and tried to raise $10 million in venture financing to advance the drug through “pre-clinical” (animal) trials. But he had to give up. “Everyone said, ‘This is sexy technology, but we don’t fund pre-clinical drugs anymore,'” Seiler says.
So Seiler, who bought the drug from a New Jersey company that had gone bankrupt, nurtured its early development with about $1.5 million in angel funding, money from his own pocket, and a $750,000 loan from the Massachusetts Life Sciences Center. Then, in November 2010, AesRx (pronounced ES-er-ex) formed a partnership with the National Institutes of Health, which committed an undisclosed amount of money to help advance the drug through pre-clinical studies and the first human trials. “It was a huge step towards getting us through the Valley of Death,” says Seiler, quoting terminology that CEOs often use to describe the chronic lack of venture funding for early-stage biotechs.
Seiler says Aes-103 is the only drug in development that targets the underlying cause of sickle cell disease. The condition, which is inherited, is caused by abnormal hemoglobin, the oxygen-carrying protein in red blood cells. The red blood cells become distorted into a sickle-like shape, causing pain, organ damage, infections, and other complications that can turn fatal. AesRx’s drug, called 5-hydroxymethyl-2-furfural (5HMF), is derived from sugar and designed to prevent blood cells from sickling.
There is one anti-sickling drug on the market already, but it has such harsh side effects it can’t be used in children—a huge proportion of the patient population. If Aes-103 proves safe in the healthy adult volunteers participating in the first trial, the company will design trials in children, Seiler says. “It’s important to get this into
