A small molecule approach to regeneration of hearing cells and other damaged tissue.
Frequency Therapeutics is an early-stage developer of small molecule drugs to activate “progenitor cells” and restore healthy tissue. Its lead program is in treating hearing loss by regenerating sensory cells in the inner ear, for which it is planning a Phase 1 trial to launch in mid-2018.
WHAT’S AT STAKE
Frequency is yet another industry reverberation from the inventive activities of Dr. Robert Langer and his lab at MIT. My conversation with the company’s cofounders, David Lucchino, president and CEO, and Chris Loose, Ph.D., chief scientific officer, starts out in 2006, after Loose, top of his class at Princeton, joined the Ph.D. program at Langer’s lab and teamed up with Lucchino to win the lab’s business competition. Their business plan? Start a company, Semprus BioSciences, that would develop and gain FDA clearance for a new biomedical technology by 2012. They met their goals, winning the clearance and selling Semprus, then counseled with Langer about what to do next.
“Dr. Langer was very passionate about finding a way to get the disease-modifying benefits of gene therapy or CRISPR — but without drug delivery complexities or permanently changing the genetics of the body,” says Loose. “He asked, what if we could just leverage the stem or progenitor cells that are already in your body, and just give them a simple cue with small molecule drugs that activate them in place to restore healthy tissue and function? That led to what we now call Progenitor Cell Activation or PCA technology. He was passionate about PCA being a whole new mode of medical therapy.”
Lucchino and Loose sensed the business potential of Langer’s concept once the team found ways to control cells called Lgr5 progenitors, present in the ear, with small molecule drugs to restore the hearing cells. “It is when we are in our mother’s womb that the hearing function fully develops, and it is turned on even before you were born. So the hearing cells you’re born with are the same ones you die with,” Lucchino says. “Dr. Langer and the Frequency team figured out how to take advantage of this biological anomaly and hotwire it temporarily using small molecules to get it to regenerate itself.”
Two factors ensure the action is temporary and precise: The drugs only need to be present a short period of time, and the activated cells are contained only in their proper place in the ear, the cochlea, after local infusion. “We actually will do a simple injection across the eardrum, which is a procedure done all the time for steroids or antibiotics, and then the small molecules can easily diffuse where they get together to get to the progenitors to reawaken cochlear cells,” says Lucchino.
With hearing loss now almost as common as farsightedness, hearing aids remain the sole solution available and have become considerably cheaper, but simply amplifying sounds in the ear cannot solve the biological problem and may only add to the harm over time. One advantage of having a large potential market will hopefully be an almost limitless patient pool for clinical trials. Another, if investors continue to find the company’s concept plausible, will be in funding the actual trials.
But the ultimate advantage of success would be the practical logic behind the company’s primary approach. “In the history of regenerative medicine, many have taken a really challenging approach: removing cells from the body, manipulating them, and then trying to put them back into the right spots, to do the right job,” Lucchino says. “That was one of Langer’s insights — just use the progenitor cells that are already in place and programmed to do the right job — and that is the heart of our technology.”
Employees: 16 Headquarters: Woburn, MA
2017 Series A Funding Round: $32M
Lead Investor: CoBro Ventures
April 2017: Announced $32 million Series A financing to support clinical development of a first-in-class hearing restoration therapeutic. February 2017: Announced novel small molecule approach to restore hearing, published in Cell Reports. 2017 Board Members: Tim Barberich, Marc Cohen (chairman), Marc Kozin, Dr. Robert Langer, David Lucchino