Pioneering mRNA Therapies to Combat Hypercholesterolemia and Improve Long-Term Health
Repair Biotechnologies, a preclinical biotech, is developing innovative therapies for age-related diseases such as hypercholesterolemia. Their lead asset aims to address the underlying cause of atherosclerosis, representing their ambition to solve some of the most challenging issues associated with aging. The proprietary technology behind Repair’s approach is a novel Cholesterol Degrading Protein (CDP) that targets serum cholesterol and has shown great promise in preclinical models of atherosclerosis.
Repair determined their CDP would be most effective through a gene transfer approach, using LNP-mRNA to encode the CDP. This method causes the liver to transiently secrete the CDP into the serum, where it degrades cholesterol. Preclinically, this technology has demonstrated a profound reversal of atherosclerotic plaque formation.
Collaborative mRNA Manufacturing Accelerated Repair’s Preclinical Success and Cut Costs
Vernal Biosciences stepped in as a true partner, approaching the mRNA manufacturing relationship with Repair with a focus on collaboration and shared success. Vernal’s responsive, flexible approach, combined with deep mRNA expertise, was a game-changer. By partnering with Vernal, Repair reduced the friction around on-time supply of mRNA for critical preclinical studies. The consistent supply of high-quality mRNA allowed Repair to rapidly engineer and improve their drug substance, resulting in a 2-fold improvement in preclinical efficacy.
These improvements in potency have reduced clinical dosing requirements significantly, which will benefit both patients and the cost of drug product manufacturing. Vernal’s commitment to quality and fast turnaround times empowered Repair to implement designed improvements with clear, actionable results.
Enhance Efficacy, Cut Costs & Streamline Clinical Trial Feasibility
Download the full case study to discover how Repair Biotechnologies boosted efficacy, slashed costs, and streamlined clinical trials with Vernal Biosciences—paving the way for the next wave of breakthrough treatments.
The Challenge: Maximizing mRNA Delivery Without Sacrificing Safety
- Technical Complexities
Overcoming challenges in mRNA delivery systems requires precise control over how the therapeutic molecules are packaged and delivered to the right cells, without causing harmful side effects. Striking the right balance between potency and safety is crucial to ensuring the long-term success of gene therapies, particularly for complex conditions like cholesterol management and aging-related diseases. - Costs & Timelines
Previous partnerships with larger, less responsive mRNA manufacturers resulted in missed deadlines, budget overruns, and a slower pace of progress. Repair Biotechnologies sought a more efficient, agile approach, cutting down on both the financial burden and the lengthy timelines that typically slow down innovation in gene therapy. - Regulatory
Regulatory approval is one of the biggest hurdles in bringing gene therapies to market. From safety to efficacy, every element of the mRNA delivery system must adhere to strict guidelines set by health authorities.
It’s been incredibly valuable to have a team that’s responsive and actively interested in helping us test new ideas and refine our mRNA. Vernal’s dedication to continuous improvement has made all the difference.
Reason
Co-Founder & CEO, Repair Biotechnologies
