LAFAYETTE, NY Inside a laboratory tucked in the LaFayette hills south of Syracuse, a small biotech company is quietly developing drugs that may show promise to treat, prevent and possibly even reverse macular degeneration, a disease that’s a leading cause of vision loss.
The new drug therapies being developed and tested are designed to work on two varieties of the eye disease: age-related macular degeneration and juvenile-onset macular degeneration, said Kelsey Moody, who founded the biotech company Ichor Therapeutics in 2013.
The 30-year-old started working out of his Tipperary Hill living room while he was a SUNY Upstate Medical University student in 2013. He then moved his office/lab – and living quarters – to a former tanning salon in LaFayette in 2014. Ichor this year moved again to larger offices just down the road in LaFayette.
Moody was a second-year medical student when he started his company with a $540,000 grant from Life Extension Foundation, a Florida-based non-profit that funds research in aging, age-related diseases and ways to extend human lifespan.
Ichor has so far attracted more than $3.2 million from investors, foundations and local government.
Why settle in tiny rural LaFayette? Moody said he was able to set up his lab there for much less than what it would have cost him to rent space at a university, and the community has been supportive of his work.
Moody, a tall, lanky man who grew up in Clinton County, is easygoing and yet driven, according to colleagues. They describe him as focused, hard-working, and a problem-solver.
But he also believes in a casual work environment where employees can relax and have fun as well as work. At work, catered meals are common and the staff plays volleyball together several nights a week. Employees compete for company swag, such as T-shirts, personalized lab coats, and NERF guns to use in company games.
Outside of work, Moody plays on several local basketball leagues, and loves to go hiking with his girlfriend Danique Wortel and their dog, Calvin. Wortel, also works at Ichor as the company’s husbandry director.
Moody said he was happy to move to the larger building so he and his 10 employees and 10 interns can work in what is now a U.S. Food and Drug Administration-compliant lab. The 10 employees include scientists, researchers and lab assistants. He also hires interns from several upstate colleges who work in the lab and assist with research.
The new drug therapies that Ichor is developing are enzymes that target both age-related and juvenile onset macular degeneration. The juvenile disease shares many of the same features as the age-related type, Moody said.
Moody says he’s particularly interested in age-related diseases because he said they don’t get as much attention and visibility as other types.
If the treatments work, the enzymes, derived from several sources, will be available as an injection. Moody is in what he calls “stealth mode” right now as he works to test the validity of the treatments, so he’s not giving away too many details about them.
He plans to publish his findings once he, in conjunction with Syracuse University, secures patents to protect his work.
The company’s research holds a lot of promise if it works as Moody hopes, said Dr. Szilard Kiss, an ophthalmologist and an associate professor of ophthalmology at Weil Cornell Medical College. Kiss, who runs the retina division at Cornell’s medical college in New York City, treats patients with macular degeneration and other retinal disorders.
Age-related macular degeneration is the leading cause of vision loss and blindness in people older than 50, according to the American Macular Degeneration Foundation. It affects more than 10 million Americans – more than cataracts and glaucoma combined – and is considered incurable.
The disease results in the deterioration of the central portion of the retina, the inside back layer of the eye that records the images people see and sends them via the optic nerve from the eye to the brain.
Moody said his team believes macular degeneration is caused in part by “junk that accumulates in the eye” over time. Just so much debris can build up in the eye before it begins to cause problems, he said. There are multiple types of this substance that accumulate, and Moody said it may cause the eye disease or it could be a side effect of it.
He said Ichor has developed “therapeutic” enzymes that break down that “junk” or particles.
“Either we hit a home run and cure the disease or, if that fails, we have the answer to the important question of what happens when we get rid of the junk,” Moody said.
Cornell’s Kiss said it’s that buildup of toxic material called lipofuscin under the retina in the “padding” behind it that leads to the types of macular degeneration Moody is targeting.
Kiss, who is familiar with research into this condition and read up on Moody’s work, said Moody is aiming at the A2E molecule which is one of the components of that toxic buildup.
Other researchers are working on this as well, but without success to date, Kiss said.
“It’s a tough nut to crack, as it’s difficult to soak up that A2E,” Kiss said. “If what Ichor is doing works and can treat the dry form and prevent the wet form of macular degeneration, it would be really great.”
“He’s not the first one to try,” Kiss said, but Moody could be the first to succeed.
The wet form of the eye condition is the most severe and most likely to cause vision loss. The dry form can lead to the wet form.
Robert P. Doyle, a Syracuse University chemistry and biology professor and an associate professor of medicine at SUNY Upstate Medical University, agrees with Kiss.
“The science behind what Ichor is doing is very sound,” said Doyle, who is working with Moody to patent the drug treatments. “The technology is very new, and there’s no reason it won’t work, but it will of course have to be validated.”
Doyle, who says he and Moody will publish a paper soon, said Moody excels both as a research scientist and as a businessperson.
“He’s done an awful lot of scientific research in his career before starting his own company, but he also has an MBA and is good at business,” Doyle said. “He has good ideas and he is willing to have them challenged. He’s fearless.”
Doyle said Moody also looks for help and advice from a scientific advisory board that Moody created.
Ichor also is developing compounds to treat acute myeloid leukemia, a cancer of the myeloid lining of blood cells. Macular degeneration and the leukemia are not related, but Moody said work on the two involved the same lab equipment and analytical chemistry.
The compounds Ichor is developing to treat the leukemia appear to hold the most promise when combined with existing drugs and would be aimed at lessening the side effects of traditional chemotherapy and radiation, Moody said.
Moody expects the macular degeneration treatment to be a bigger hit because there is no effective treatment for early stages of the disease.
With more than $3 million from investors and grants, Moody said he is now able to move to the next phase – from preclinical testing to more rigorous animal testing of the compounds in his new lab.
CenterState Corporation for Economic Opportunities, a Syracuse-based economic development group, gave Ichor $75,000 in 2015 as part of its state-funded Grants for Growth program. Ichor received a “concept to marketplace” grant which is designed for companies in the later stages of bringing a product to the marketplace, said Eleanor Hanna, a spokeswoman for CenterState.
“We look for companies with a strong business model and growth potential, and also companies that demonstrate innovation,” Hanna said.
Ichor is ready to move into what’s called pivotal efficacy studies – which could help prove the drugs work. When those are complete, Ichor will work to raise another $5 million to $10 million to complete the final animal trials and move into testing on people.
“We think we could have our (animal trial) data in 12 months, and we could be doing human trials in two to three years, Moody said.
With mice trials about to begin, Moody said once it’s determined the compounds break down the substances that gather in the eye, the next step is to test the vision of the mice. How? Working with other collaborators, tiny equipment will be used to test the mice’s vision using electrodes that track the signals from their eyes.
Moody, who is originally from Beekmantown in Clinton County, received his bachelor’s degree in biochemistry at the State University College at Plattsburgh in 2010 and a master’s in business administration from Concordia University in Portland in January 2013.
As an undergraduate at Plattsburgh, Moody helped found the SENS Foundation Academic Initiative, an international student research and development program which supports students who are looking into careers in regenerative medicine and biomedical gerontology.
Moody served as chief technology officer at ImmunePath, a start-up stem cell therapeutics company in Menlo Park, Calif., where he was in charge of laboratory automation and analytics. The company successful tested its work in mice but could not raise enough investment capital to move into human trials, according to an Upstate Medical University article on Moody.
In 2012, Moody started to study medicine. In his second year there, he was featured in an Upstate Medical Alumni journal article that talked about his research and how he started Ichor.
Moody said after securing the $540,000 grant, he withdrew from Upstate to focus his efforts on building his company.
Another group helping to fund Moody’s work is The Methusaleh Foundation, a non-profit based in Springfield, Va., dedicated to advancing tissue engineering and regenerative medicine. It gave the company an $80,000 grant.
“I bet on people as opposed to companies,” said David Gobel, chief executive officer of the foundation. “Kelsey is someone who gets results at low cost.”
Aubrey de Gray, who is now the foundation’s chief science officer and who consults with Moody, said Ichor’s work is “extremely impressive and promising.” If the drugs work as designed “it will be completely revolutionary,” he said.
Roger Bagg, founder of UK biotech company BioSenex Ltd., said he gave Ichor a $1 million grant because he’s likes Moody’s innovation and growth.”His (Moody’s) ability to grow Ichor from a small set-up in his living room, to the current team and facility, has impressed me greatly,” Bagg said.
Moody said most other R&D work has focused on treating the later stages of the macular degeneration while his focus is on the early stages.
“Basically, my job is not to get too excited, because you have to be very disciplined in your research,” Moody said. “You have to be able to replicate your findings over and over to be sure your drug works the way you want.”