Activation of BMP3 to Treat Pulmonary Hypertension

THE CHALLENGE

The major business challenge in addressing pulmonary arterial hypertension (PAH) lies in the urgent need for breakthrough therapies that go beyond symptom management to truly modify disease progression. Current drugs offer limited clinical benefit, are burdened by significant side effects, and fail to reverse the underlying biological mechanisms such as pulmonary vascular remodeling in PAH. This creates a substantial market gap for innovative treatments that can effectively target root causes like endothelial dysfunction and smooth muscle cell proliferation. For biotech and pharmaceutical companies, this represents both a high-risk and high-reward opportunity—developing next-generation therapeutics that meet stringent regulatory demands while demonstrating clear advantages over existing therapies in efficacy, safety, and long-term patient outcomes. Bridging this gap requires strategic investment in translational science and precision medicine platforms to deliver solutions that are both clinically impactful and commercially viable.

OUR SOLUTION

We present a groundbreaking therapeutic approach for a severe lung disease, pulmonary hypertension, by targeting a key molecular driver of disease progression. This invention is based on the therapeutic effect of Bone Morphogenetic Protein 3 overexpression in reversing pulmonary vascular remodeling in pulmonary hypertension via the restoration of the balance between the TGF-b and BMP pathways through a cell-cell communication mechanism. This strategy has been validated through robust in vitro and in vivo studies, including the use of gene therapy and recombinant peptide platforms, demonstrating reversal of disease pathology and restoration of heart and lung function. By focusing on disease modification rather than symptom control, and leveraging underexplored biological pathways with strong translational potential, this technology offers a compelling commercial opportunity to deliver first-in-class therapy to large, underserved patient populations worldwide.

Figure: BMP3 mechanism in action in PAH

Advantages:

• Inhibits endothelial dysfunction and reverses pulmonary vascular remodeling
• Improves cardiac function and cardiac hemodynamics
• Demonstrates strong efficacy in preclinical models with high translational potential
• Advantage vs Sotatercept: reduced side effects via a lung-targeted overexpression of BMP3, does not require training for self-injection, regular clinic visits, or frequent monitoring

Potential Application:

• BMP3 represents a promising disease-modifying agent with potential for clinical translation in the treatment of PAH
• BMP3-based gene therapy platform (Overexpressing BMP3 with Adeno-Associated Viruses has therapeutic potential to treat PAH with minimal side effects).