In a recent interview, Thomas Langenickel, MD, Chief Medical Officer at Ethris, shed light on the hurdles faced in the development of mRNA therapeutics for pulmonary diseases. Langenickel’s expertise in clinical development and translational medicine in cardiovascular and respiratory areas brings valuable insights to the field.
One of the major challenges lies in maintaining stability throughout the supply chain, ensuring end-use stability, and optimizing equal access to mRNA therapeutics. Additionally, the biological deployment conditions greatly influence the biodistribution of these therapeutics. Ethris has made significant progress by successfully storing lyophilized lipid nanoparticles (LNPs) at ambient temperatures for up to six months. This breakthrough can potentially address stability concerns.
Mechanical stress during manufacturing and handling can lead to aggregation of LNPs or leakage of mRNA from the LNP. These issues can result in increased immunogenicity, difficulties in nebulizing, and even manufacturing failures. Ethris aims to overcome these challenges by utilizing a manufacturing platform that eliminates the need for high-pressure liquid chromatography, reducing costs and minimizing the impact of sheer forces.
One critical drawback observed with current mRNA-based vaccines is their broad biodistribution. When injected into the musculature, these vaccines reach various organs, including the brain, liver, spleen, and other major perfused organs. The ideal scenario would be to specifically target the affected lung tissue. This precise targeting can potentially enhance the therapeutic efficacy and minimize off-target effects.
Langenickel’s experience and expertise in the industry, including his previous positions at Novartis and Bristol-Myers Squibb, positions him as a key figure in advancing the development of mRNA therapeutics for pulmonary diseases. His insights provide valuable guidance and highlight the ongoing challenges that need to be addressed.
Q: What is the main challenge in developing mRNA therapeutics for pulmonary diseases?
A: The main challenge lies in managing stability during the supply chain, ensuring end-use stability, and optimizing equal access throughout the process.
Q: How has Ethris addressed stability concerns?
A: Ethris has achieved success in storing lyophilized lipid nanoparticles (LNPs) at ambient temperatures for up to six months.
Q: What issues can arise from mechanical stress during manufacturing and handling?
A: Mechanical stress can cause aggregation of LNPs or leakage of mRNA, leading to increased immunogenicity, nebulization difficulties, and manufacturing failures.
Q: What is the goal of targeting lung tissue specifically?
A: Targeting lung tissue specifically can enhance therapeutic efficacy and minimize off-target effects.