Liver disease is a significant unmet need
Conventional approaches to liver disease have been unsuccessful. Many large pharmaceutical companies have invested in developing novel liver therapeutics, but they have faced two major challenges:
1. A lack of understanding of the complexities of chronic liver disease. This has led companies to focus on a small number of known targets, many of which have already failed in clinical trials (and were often repurposed anti-fibrotic or diabetes drugs).
2. A reliance on poorly predictive preclinical models (in vitro / animal). These models often do not accurately reflect the human disease state, making it difficult to identify effective treatments.
Novartis, Pfizer, Gilead and Novo Nordisk have all invested in liver programs, however, there is a high degree of overlap across pipelines, with few novel targets in development.
Unique platform + RNA therapies
Given those challenges, Ochre has taken a very different approach to all aspects of the therapeutic path from discovery to clinic. The first part of this section focuses on their unique platform capabilities (discovery and validation), the second part focuses on their approach to turning this process into an actionable pipeline of RNA therapies.
Ochre is focusing on RNA therapies that they can take to trials with readouts in weeks (vs. months/years for their competitors) and can accelerate through a hugely valuable, broadly derisking first indication, improving liver transplant outcomes (an orphan indication where the therapies may also have utility in patients living with chronic liver disease).
Ochre is emerging as a leader in the liver disease field
Ochre has built the world’s largest liver-specific knowledge database. By combining this with closed loop learning, we are able to hone in on the most relevant targets for the disease - faster, cheaper, and more accurately.
Ochre’s two most advanced programs are their metabolism (generally alcohol mediated) and resilience (transplant and NASH) programs. In both cases, the company has progressed from target discovery through to perfusing leads in human livers in New York City. Beyond these lead candidates, the company is looking to progress other metabolic targets and others in cancer and regeneration.
The scale of Ochre's work is uniquely powered to address the complexities of liver disease.
Scott Friedman, MD; Chief, Division of Liver Diseases; Icahn School of Medicine at Mount Sinai
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