If you enjoy listening to loud music, you may be glad of the efforts of Frequency Therapeutics later in life. The company has just raised $32 million in a financing round it hopes will help it bring its therapy for noise-induced hearing loss into the clinic.
The Massachusetts biotech is focusing on using small-molecule drugs to stimulate the regrowth of the sensory hair cells in the inner ear or cochlea that can be damaged by sustained exposure to loud noise. If effective, it could provide the first treatment option for an estimated 360 million people with established hearing loss as well as the 1.1 billion people worldwide at risk from exposure to "recreational noise."
We start with around 15,000 such cells per ear, but that population is gradually whittled away by loud noise and cannot be replaced, eventually resulting in hearing loss.
Frequency's small-molecule approach sets it apart from other groups developing more complex therapies for hearing loss based on genetic manipulation, said Marc Cohen, who is co-founder of CoBro Ventures—which led the Series A round—as well as the chairman of Frequency's board, in a statement.
The aim is to "gain much of the same effect as gene therapy and CRISPR by using small molecules, which we believe are safer and allow for easier delivery," according to Cohen. "Our data is very compelling and we are excited to be moving to the clinic in the next 12 to 18 months."
The company's progenitor cell activation (PCA) platform has grown out of MIT, and specifically the labs of Robert Langer, Sc.D., and Jeffrey Karp, Ph.D., who have been working on using drugs to stimulate progenitor cells to create a range of functionally active offspring.
In preclinical studies reported earlier this year, the team showed that compounds interacting with the Lgr5 receptor could stimulate cochlear progenitor cells into large colonies of cells with the capacity to develop into hair cells. The ability to regenerate hair cells is absent in humans, but is seen in some species of birds and amphibians.
Frequency isn't alone in following this small-molecule path. Boehringer Ingelheim also has a drug discovery program in play, stemming from a collaboration forged last year with Professor Renjie Chai, a researcher at China Southeast University Institute of Life Sciences, focusing on other cellular targets such as Shh, Hippo, and Foxg1.
Given the sheer number of potential patients, it's unsurprising that several big pharma companies—including Pfizer, Roche, Sanofi, Novartis (thanks to a deal with gene therapy specialist GenVec) and GlaxoSmithKline—as well as smaller players including Auris Medical and Decibel Therapeutics, also have active hearing loss programs.
The new cash reserves will help Frequency expand its R&D pipeline beyond hearing loss, however, according to CEO David Lucchino.
"We believe there is even broader potential in indications beyond hearing loss with the further development of the PCA platform," he said, pointing to potential applications in "skin disorders, muscle regeneration and gastrointestinal diseases."