Translational Candidate

Modified synthetic noncoding RNA molecule

Area of Impact

Systemic Sclerosis

Mechanism of Action

The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue.

Unmet Medical Need

Systemic sclerosis is an incurable disease with no effective therapeutic management strategy. In this proposal we seek to develop an orally-administered engineered RNA therapeutic with remarkable disease-modifying bioactivity in in vitro and in vivo preclinical models.

Project Objective

Obtain data needed to convene a pre-IND meeting.

Major Proposed Activities

• Product characterization
• Preclinical studies assessing dose, toxicity and biomarker development
• Regulatory planning

The target indication is an systemic sclerosis, a crippling, incurable and the most lethal rheumatic disease (30% mortality rate over 10 years) . Systemic sclerosis disproportionately afflicts disadvantaged populations (women, Blacks and Latinos, and Native Americans). Because the therapeutic candidate is universally applicable, the societal benefits of success here are expected to be profound.