R & D
Product Pipeline
Our product pipeline includes drug candidates for the treatment of mitochondrial and other diseases.
R007 Overview: A Novel Approach
Instead of attempting to treat the multiple genetic causes of mitochondrial dysfunction, we aim to treat the consequences of mitochondrial dysfunction, regardless of the cause. We are testing the drug candidate R007.
The preclinical studies indicate that R007 acts in the cytosol outside the mitochondrion, resulting in reversal of the dysregulation of central nodes of the nutrient-sensing signaling network, in particular AMP-activated protein kinase (AMPK), peroxisome proliferator-activated receptor (PPAR), and mammalian target of rapamycin complex 1 (mTORC1).
R007 Mechanism of Action
R007 Expanded Access Policy
RiboNova, Inc. is a clinical stage biotech company committed to the development of safe and effective novel therapies for severe and life-threatening orphan diseases.
Clinical trials that generate safety and efficacy data are the way of ensuring review and decision making by Regulatory Authorities. Approval by a regulatory authority is ultimately the path to enable access to new, safe and effective medicines for patients.
Expanded access (sometimes also referred to as “compassionate use”) refers to the use of an investigational therapy (i.e. not approved and not available outside of clinical trials).
Please refer to the FDA website for more information about the criteria under which a drug can be made available under an Expanded Access Program.
We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy. RiboNova believes that participation in our clinical trials is the most appropriate way to access our investigational drug R007 (probucol). Albeit R007 (probucol) is a drug previously approved for another indication – no data is available for its use in mitochondrial disorders for either adults or children / adolescents. The clinical study of R007 requires numerous safety assessments that are important to monitor the safety and wellbeing of patients. FDA has specifically requested that the initial investigations of R007 be limited to adult patients.
Hence, at this time, RiboNova has decided to make available R007 through a very limited Expanded Access Program, at one clinical site in the USA, for adults patients that are not eligible to enroll in our P2 Clinical Study. Subject to drug availability, and on a patient-by-patient basis – up to 10 patients will be submitted by the site to be evaluated for eligibility.
More information about our Expanded Access Program can be found on clinical trials.gov.
tRNA Drug Discovery Program
In addition to our R007 product candidate, we have developed a proprietary drug discovery platform for new precision medicines that target a novel family of pharmaceutical targets: defective transfer RNAs.
Potential Drug Targets on Transfer RNA
Transfer RNA is the most abundant ribonucleic acid in the human cell, and delivers amino acids to biological factories called ribosomes, to make the proteins that drive all cellular operations. Defects in transfer RNAs are associated with several human diseases.
tRNA Modification Map
RiboNova is applying its technology to map RNA modifications in a wide range of human diseases, to identify clinically useful biomarkers and powerful therapeutic targets for drug development.
Disruptive Technology for Pharmaceutical Innovation
