Founded in March 2022, DUB Therapeutics has a mission to bring global relief, and increased quality of life, to patients living with fibrosis. Fibrosis, a major underpinning for all cause mortality, is implicated in 1 in every 3 patient fatalities and has caught the recent interest of pharmaceutical development for next generation technologies. Many novel approaches have the advantage of target specificity—opening up a world of new targets previously recognized as “undruggable.”

DUB Therapeutics, DUB Tx, is developing an anti-fibrotic self-delivering siRNA (sdRNA) against a novel cell-specific target. The target was identified by Dr. Audrey Bernstein as part of a post-translational genetic screen in a series of experiments whose conditions did not include the presence of TGF𝛃. These conditions allowed the discovery of a class of proteins, called ‘deubiquitinase,’ whose enzymatic activity diverts proteins from degradation in the cell. Interestingly, these genes were significantly upregulated and became the subject of academic focus and, more recently, commercial drug development. Reducing the overexpression of the deubiquitinase target gene following injury using a class of therapeutics called interfering RNA (RNAi), Dr. Bernstein has demonstrated a phenomenon called regenerative healing. Through the identification of the target and the development of the proprietary technology, over 15 years of academic experience in the Department of Ophthalmology at Mt Sinai and the Center of Visual Research (SUNY Upstate Medical University) has passed leading to the generation of proof-of-concept data supporting the use of sdRNA in vitro, ex vivo (rabbit and human organ tissue), and in two pre-clinical animal models (mouse and rabbit) for corneal traumas where properties of tissue regeneration is observed.

Interestingly, the cornea, a simple highly organized and enervated tissue, has a specialized function to provide a protective barrier against pathogens, yet allow the passage of light through the lens. In this way, the most sensitive (and immune-privileged) tissue in the body may remain pathogen free while the host can still see. Perturbations to the cornea disrupt to the protective barrier and initiate a cascade of events that are characterized as the stages of wound healing. The stages include 1). Hemostasis, 2). Inflammation, 3). Proliferation, and 4). Remodeling, and are key stages of wound healing in all tissues.

While our focus at this time remains on the critical unmet need for appropriate therapeutics for ocular surface diseases, the long-term vision includes relieving the global unmet need of fibrotic disease to patients across multiple therapeutic areas with high-burden.