About Inflammasome Therapeutics

Inflammasome Therapeutics is a science-based company founded to develop therapies for prevalent, degenerative diseases such as Alzheimer’s disease, multiple sclerosis, macular degeneration and type 2 diabetes.

The precise causes of these diseases are unknown; however they share many of the same risk factors – smoking, obesity, hypertension, and age. They all are multifactorial diseases with multiple biochemical factors, many shared, contributing to their progression.

The Role of Inflammasomes

It is now recognized that these diseases are all characterized by upregulation of the inflammatory process, driven by prolonged activation of inflammasomes. Inflammasomes are intracellular multiprotein complexes that help trigger and maintain the inflammatory response normally as part of the innate immune system. However, in Alzheimer’s disease, multiple sclerosis, macular degeneration, and type 2 diabetes, inflammasomes become and remain activated causing the chronic release of inflammatory molecules such as caspase -1, IL-18 and IL-1β, which in turn cause progressive tissue damage and disease progression.

Inflammasome Therapeutics’ focus is on reducing the dysregulation of the inflammatory process by preventing aberrant inflammasome activation.

This represents a new approach to these diseases. To date, the main strategy in developing new therapies for Alzheimer’s and geographic atrophy has been to focus on attacking a single, causal element. Typically this has been amyloid β for Alzheimer’s disease and various forms of complement for geographic atrophy. These attempts have all failed in large scale clinical trials. We believe that targeting a single factor, when multiple factors are present, is not optimal.

The multiple elements elevated in tissues of patients with geographic atrophy (Amyloid β, Complement, Alu RNA, Iron) have a common feature: they all cause, or are products of, inflammasome activation. In pre-clinical models of this disease, these factors initiate inflammasome activation and produce phenotypes resembling human disease. Most importantly, inhibition of inflammasome activation effectively treats these diseases in multiple animal models.


We have identified and licensed a series of molecules – Kamuvudines – that successfully inhibit inflammasome activation in cell culture and animal models. Inflammasome Therapeutics is poised to initiate clinical trials of our lead compounds in the near future.

In 2014, our co-Founder’s lab first reported in the journal Science that some of the existing FDA-approved drugs used in the treatment of HIV and hepatitis B are powerful inhibitors of inflammasome activation.

We have found NRTIs to be highly effective in multiple, disease-relevant pre-clinical models of inflammasome-mediated diseases such as macular degeneration and type 2 diabetes.

Unfortunately the side effects of NRTIs (primarily mitochondrial toxicity) precludes their use in any but the most serious, life-threatening conditions such as HIV infection or hepatitis. This toxicity is due to the intracellular metabolism of the drugs and is unrelated to their activity against inflammasome activation (but is key to their anti-viral properties).

Kamuvudines are chemical derivatives of NRTIs that have more potent anti-inflammasome activity than the parent molecules yet cannot undergo the same metabolism and have no detectable mitochondrial toxicity. In multiple pre-clinical tests they have been found to be over 1,000-times less toxic than their parent molecules and to be highly effective in multiple models of macular degeneration and type 2 diabetes.


We strive to maintain and expand a robust intellectual property estate for our drug candidate and drug delivery assets. The early work in Kamuvudines was conducted in Dr. Ambati’s laboratory at the University of Kentucky, and we have an exclusive worldwide license to the patents arising from that work from the University of Kentucky Research Foundation. These patents cover use of NRTIs and Kamuvudines for Dry AMD, GA, Rheumatoid Arthritis, and Diabetes. Seven of these patents have issued in the US and three have been issued in other important countries. Additional patents are in prosecution and protection is pending in multiple countries.

We have also developed new IP covering the administration of these compounds to the eye as sustained release depot formulations. Patent applications on this technology are being filed.

Our Co-Founders

After graduating from Johns Hopkins University at 17 years of age, Jayakrishna Ambati has gone on to become one of the world’s leading authorities on degenerative diseases. He teamed up with Paul Ashton, who has developed four new drugs through to FDA approval, to found Inflammasome Therapeutics. Their desire to build a company combining scientific excellence with solid developmental expertise is reflected in the makeup of the Board of Directors:

  • Mary Weber, Esq. is a former senior partner at Ropes and Gray.
  • Balamurali Ambati, graduated Mount Sinai Medical School at 17 years of age and is now a respected ocular surgeon and entrepreneur.
  • Joon-youb Lee is CEO of a pharmaceutical company.
  • Napoleone Ferrara is the recipient of both the Lasker-DeBakey Award and the Breakthrough Prize in Life Sciences for his work in developing two multi-billion dollar therapies: Avastin (cancer) and Lucentis (wet macular degeneration).