The Need

THERAPEUTICS AREAS

Chronic progressive diseases such as congestive heart failure (CHF) and type 2 diabetes afflict hundreds of millions of people worldwide, and the incidence is growing rapidly. At present there are no definitive treatments for either disease that can fully restore a person’s health. Obesity and non-alcoholic fatty liver disease (NAFLD) are also two growing threats to global health, and pulmonary hypertension remains a rare yet burdensome and fatal disease. All of these debilitating chronic diseases pose an unsustainable burden on the lives of patients and their families, on medical practitioners, and on economies crippled by health care costs.

RT-100 FOR CONGESTIVE HEART FAILURE (CHF)

The Company’s most advanced investigational therapy, RT-100 (Ad5.hAC6), is a single dose gene therapy for CHF, one of the most common and devastating chronic diseases in the world.  According to the NIH, HF affects more than 64 million people worldwide and is the leading cause of death in the United States.  All pre-clinical work has been completed, and the phase 2 human trial for the AC6 gene therapy product candidate for congestive heart failure has been successfully completed with promising results.  RT-100 has been cleared for phase 2b/3 trials by the FDA.   

In addition to the IND, the Company has filed an Innovative Licensing and Access Pathway (ILAP) application in the UK through the Medicines and Healthcare Products Regulatory Agency (MHRA) in June 2022, and the Company is evaluating other pathways that will expand market opportunities to other regions with a potential to accelerate commercialization.

RT-200 FOR TYPE 2 DIABETES MELLITUS

RT-200 (AAV8.UCn2) is a first-of-its-kind intravenous single-dose gene therapy product candidate that has been shown to be a promising treatment for T 2DM. Diabetes is highly prevalent affecting 537 million people worldwide or about 10% of the world’s population. This number is predicted to rise to 643 million by 2030 and 783 million by 2045. RT-200 is designed to provide improved outcomes in type 2 diabetes patients by normalizing blood glucose and A1c levels.

Savarese et al., “Global burden of heart failure: A comprehensive and updated review of epidemiology,” Feb. 12, 2022.

 IDF Diabetes Atlas website as of June 5, 2022.  https://diabetesatlas.org/

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