CSO: Dr. Dorit Cohen-Carmon
Ramino Bio is an Israeli private biotech company developing an oral drug to treat metabolic diseases. Per a well-thought-out strategy, its first indication is Maple Syrup Urine Disease (MSUD), a rare genetic disease that leads to severe neurological failure. Furthermore, its unique mechanism of action heralds great promise to other metabolic disorders associated with excess accumulation of branched-chain amino acids (BCAA; valine, leucine, and Isoleucine), mainly insulin resistance and type 2 diabetes (T2D).
Since its inception, Ramino successfully met the yearly milestones and received total funding of ~$3.3M from FutuRx biotech incubator sponsored by the Israeli Innovation Authority, Takeda, J&J, OrbiMed, and RMPG. Our unique technology reduces the excess circulating BCAA by increasing their breakdown.
BCKDK, as an important protein regulator of BCAA metabolism, is a new therapeutic target for treating metabolic disorders caused by BCAA accumulation. BCKDK inhibition alleviates metabolic disease biomarkers and symptoms (East et al., Nature, 2021) since increased BCAA breakdown improves mitochondrial function, glycemia, and adipose tissue redox homeostasis for treating insulin resistance /diabetes (White, Molecular Metabolism, 2021, Review). The mechanism of action of BCKDK inhibition is a disease-modifying treatment, and our synthetic drug is designed to be given orally twice daily.
Ramino-Bio is in preclinical stages with a druggable lead compound, showing efficacy in various MSUD patient-derived cells and PoC in-vivo. An oral drug for lowering BCAA levels can be applied for:
• An unmet need for MSUD: The first Proof-of-concept indication, a genetic metabolic disorder in which mutations lead to elevated BCAA levels. Our technology for MSUD involves a clear and fast path to clinical studies, with a simple clinical non-invasive measurable outcome in blood and urine.
• Metabolic diseases, T2D, and Insulin resistance, in which elevated BCAA levels play a role in disease propagation and comorbidities.
NOVELTY/ COMPETITIVE ADVANTAGE
• Best-in-Class oral BCKDK inhibitors that can address existing needs of various metabolic diseases with the same compound by modifying disease MoA.
• MSUD: a non-invasive patient-derive blood test for clinical study patient selection
• Ramino-Bio’s technology can address the current T2D drugs limitation & provide the unmet need for sustained glycemic control & insulin resistance
Ramino is in the process of exploring strategic partnerships.
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