Molecular Therapeutics Program (MTP) is a statewide drug discovery consortium supporting early-stage drug discovery research and facilitating collaborative translational research partnerships. The program helps researchers identify complementary, supporting and external partnerships for their work, as well as funding opportunities for early-stage drug discovery projects.
The recipients of 2019 Molecular Therapeutics Program awards are:
- Kevin Vaughan, PhD, University of Notre Dame
- Proposal: In Vivo Screen of Oxysterol Toxicity in a Mouse Model. The Vaughan laboratory has discovered a previously-unknown cellular mechanism of cholesterol transport that is impaired in Niemann Pick Type C (NPC) disease. Developed a cell-based screen and identified individual oxysterols as potent signaling molecules that are deficient in NPC disease.
- Gayle Gordillo, MD, Indiana University
- Proposal: The development of a Nutraceutical Product as a Treatment for Hemangiomas. Hemangiomas are endothelial cell tumors and are the most common soft tissue tumors of infancy, affecting up to 10% of all live births. The product now has FDA IND approval for first in human testing in infants with hemangiomas, supported by the National Institutes of Health.
- Ruchi Pandey, PhD, Indiana University
- Proposal: The pharmacokinetic profiling of novel and potent small molecules for inhibition of a frequently mutated oncogenic receptor tyrosine kinase. Acute myeloid leukemia (AML) is the second most common leukemia with survival rates under 30%. Pandey’s laboratory has optimized and validated two new chemical entities using in vitro assays. Plans to run in vivo efficacy studies using clinically relevant models of AML present in the lab.
- Jaipal Singh, PhD, Indiana University
- Proposal: The formulation and PK study of a novel compound for pulmonary arterial hypertension. Singh’s laboratory has validated a small modulator that metabolizes asymmetric dimethylarginine that contributes to the pathogenesis of pulmonary arterial hypertension. The modulator has shown efficacy in a rat model. Further studies on the development of oral formulation and pharmacokinetic studies are underway.
To find out more about the program, contact Richard Taylor, PhD, Director, or Padma Portonovo, PhD, Program Manager.
