PCA Regeneration Opportunities
Naturally reversing degenerative damage and disease
Frequency is developing progenitor cell activation (PCA) therapies; innovative disease-modifying molecules that temporarily activate cellular regeneration at specific sites in the body for unprecedented healing benefits. Frequency’s most advanced program is in chronic noise induced hearing loss.
Hearing loss caused by prolonged exposure to excessive noise, including noise pollution, can be attributed to many professional environments, such as heavy construction sites or military training. According to the World Health Organization, over 5% of the world’s population suffers from disabling hearing loss, and not just the elderly. Untreated hearing loss in working-age adults costs the U.S. more than $56 billion in annual productivity. Chronic noise induced hearing loss, the focus of our lead program, affects around 48 million people in the U.S. alone.
The human hearing organ is incapable of spontaneously restoring lost or damaged hair cells, making hearing loss a permanent condition. Our therapy uses a proprietary combination of small-molecule drugs to cause inner ear progenitor cells to multiply and create new hair cells using an innovative approach we call PCA Regenerative Medicine. Because we are utilizing small molecules, our therapy can be easily administered by direct injection into the middle ear where it diffuses into the cochlea, a common delivery method for other inner ear medicines. Unlike hearing aids or cochlear implants, our therapy has the potential to restore natural hearing function by restoring lost sensory cells. And unlike cochlear implants, our therapy does not require an invasive surgery.
Hearing Loss Affects Nearly 1 Billion People Worldwide
Local company testing revolutionary hearing treatment
Using research developed at MIT and Harvard Medical, Frequency Therapeutics is trying to rebuild the tiny hair cells in the inner ear, helping to restore hearing. Listen as CEO David Lucchino discusses. - May 2017
Our PCA Regeneration approach could address a host of other disease indications beyond reversing hearing loss, and many common conditions would benefit from this simple therapeutic approach. Skin is one area where we are applying our knowledge of using small molecules to facilitate the body’s innate regenerative capacity to reverse biological deficits to restore healthy tissue and function.
One of those opportunities is in alopecia, a common condition that causes hair loss. Androgenic alopecia affects as much as 50% of men and 25% of women over 50 years of age. We are studying the application of a PCA regenerative approach to address this condition, as well as exploring how PCA could be applied to other skin disorders that could benefit from stimulating cells to naturally drive repair.
We believe our PCA regenerative medicine approach could have applications in wound-induced forms of muscle atrophy, as well as for more severe muscle diseases such as spinal muscular atrophy (SMA), a fatal genetic disease.
Muscle atrophy is defined as a decrease in muscle mass, and its effects range from partial to complete wasting away of muscle tissue. This condition causes abnormal weakness that severely impacts patients’ lives. Muscle atrophy generally has a quick onset, and reversing its effects could enable hospitalized patients to get out of bed and move around sooner, despite sutures, wounds, broken bones, and pain.
Emerging evidence from the labs of our scientific advisors, Lee Rubin, Ph.D. and Amy Wagers, Ph.D., have linked muscle atrophy to the loss of muscle satellite cells and nerve loss to SMA. Drug screens have identified satellite cell activators that can potentially reverse muscle loss in vitro. Experiments are underway to further investigate these small molecules in vivo in standard SMA models.
Spinal Muscle Atrophy (SMA) is one area that presents a unique opportunity for an innovative, regenerative medicine approach through the application of small molecules to repopulate lost cells to repair a biological deficit.
Our founders’ initial discovery of using small molecules to stimulate native stem cells were made while researching site-specific Lgr5 stem cells in the gastrointestinal (GI) tract. GI diseases can cover a wide range of conditions from esophageal disease, gastric indications, and intestinal complications, such as IBD, Crohn’s disease or ulcerative colitis. With the presence of Lgr5 progenitor cells in the GI, our PCA Regeneration approach has the potential to address a multitude of disease indications that affect millions of people in the U.S. and around the world.
Other Potential Indications
Frequency is initially focused on a very large unmet clinical need: reversing hearing loss in humans, a feat that has yet to be medically conquered. But we’re not stopping there. By validating the hearing functional improvements our platform accomplishes, we’re also validating a whole new therapeutic approach: employing small molecules targeted to specific sites in the body to heal numerous cellular maladies and deficiencies. Thus, our Progenitor Cell Activation regenerative medicine platform has the potential to treat a wide range of clinical applications and launch a whole new era of regenerative therapeutic development.