Ramino-Bio
CSO: Dr. Dorit Cohen-Carmon

GENERAL

Ramino Bio is developing an oral drug to treat cancer, metabolic diseases, and rare Maple Syrup Urine Disease (MSUD), characterized by chronic elevated branched-chain amino acids (BCAAs; Valine, leucine, and Isoleucine), which lead to toxic metabolites and alter metabolism. Our unique technology reduces the high circulating BCAAs by increasing their breakdown.

The company was founded in March 2019 at the FutuRx biotech incubator and is financed by the Israel Innovation Authority (IIA), Takeda Ventures Inc., OrbiMed Israel Partners, Johnson & Johnson Innovation (JJDC), and RMGP Bio-Pharma Investment Fund. Ramino successfully met the yearly milestones-based seed funding.

Our novel compounds have good druggability properties, showing efficacy in human MSUD patients’ cells and in-vivo Proof-of-concept in mice.  Currently in preclinical stages towards FIH for rare MSUD and elaborating pipeline for cancer/ metabolic indications.

SCIENTIFIC APPROACH

  • Our technology mechanism-of-action is considered a disease-modifying treatment. BCKDK, our therapeutic target, has become attractive for BCAA-associated diseases and cancers (East et al., Nature, 2021) since it alters metabolism to promote tumorigenesis. In addition, increasing BCAAs breakdown was found to improve mitochondrial function, glycemia, and adipose tissue redox homeostasis for treating insulin resistance /diabetes/Heart failure (White, Molecular Metabolism, 2021, Review).

PIPELINE

  • Our first Proof-of-concept indication is rare Maple Syrup Urine Disease, a genetic metabolic disorder in which mutations in the catabolic BCAAs complex lead to elevated BCAAs levels.
  • A platform for cancer and metabolic diseases, in which elevated BDK and BCAAs levels play a role in disease propagation.

TEAM

Dorit Cohen-Carmon, Ph.D., CSO

Dorit is proficient in leading and managing R&D projects from technology validation to drug development for therapy. Dorit holds a Ph.D. from The Weizmann Institute of Science and was awarded an Eshkol and Teva Post-Doctorate fellowships for applicative research (NNE) at the Hebrew University of Jerusalem. Dorit’s primary motivation is to translate scientific technologies into therapies.

Prof. David Chuang, Principal Investigator, Southwestern University

Prof. David Chuang, Dr. Richard Wynn, and colleagues discovered BT2 as a BDK inhibitor from the structure/function knowledge about BDK (Tso et al., 2014). They paved the way for many BCAAs metabolism discoveries in health and disease, using BT2 as a research tool compound.

Prof. Yibin Wang, Principal Investigator, UCLA

Prof. Wang and Dr. Sun showed an improve cardiac function in the failing mouse heart by enhanced BCAAs breakdown (Sun, Circulation, 2016), and demonstrated the therapeutic efficacy hearts with preexisting dysfunctions (Chen, J Am Heart Assoc, 2019).  Prof. Yibin Wang is Chair, Cardiovascular Theme holds over 200 publications, and an expert in heart failure and metabolic disease models, proof-of-concept validation studies.

 

 

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