Liss' Lowdown #27

Preclinical Breakthrough of the month:

Gene-Editing Therapy Targets Lung Cells in Cystic Fibrosis Study

A recent preclinical study demonstrated promising results for a lung-targeted gene-editing therapy developed by ReCode Therapeutics . Using their Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), the therapy successfully corrected a disease-causing CFTR gene mutation in lung stem cells of mice. This approach, delivered via intravenous infusion, achieved sustained gene editing in over 70% of lung stem cells and more than 80% of mature lung cells for up to 660 days.

The study, published in Science, marks a significant advancement towards developing durable treatments for cystic fibrosis (CF) and other genetic diseases. ReCode is pursuing further research to validate these findings and plans to conduct safety studies in larger animals before advancing to clinical trials. Their lead candidate, RCT2100, is also progressing through clinical testing, targeting CF patients with Class 1 CFTR mutations who currently lack effective treatment options like Trikafta.

Clinical Breakthrough of the month:

Rosacea Linked to Increased Health Risks Including Malignant Melanoma: Study Highlights Urgent Need for Further Research

Recent research has uncovered that individuals with rosacea face heightened risks of heart disease, joint problems, metabolic disorders, visual impairments, and type 2 diabetes. Notably, Caucasians with rosacea also exhibit a significantly increased risk of malignant melanoma, a severe type of skin cancer.

Rosacea, characterized by facial redness and rash development on the cheeks, chin, nose, and forehead, predominantly affects women aged 30 to 50, particularly those of Celtic and northern European descent. Despite various theories—including UV exposure, smoking, alcohol, stress, and genetics—the exact causes remain unproven, and there is no permanent cure.

A German study involving 122,444 rosacea patients compared with a control group highlighted these associations. The researchers stressed the need for further investigation into this complex condition, especially concerning its link to serious health outcomes like malignant melanoma. They noted that while rosacea's association with malignant melanoma was significant in Caucasians, no such association was observed in an Asian subgroup.

These findings underscore the urgent need for deeper understanding and targeted research efforts into rosacea's underlying mechanisms and health implications.

Partnership of the month:

Bridging Biopharmaceutical Opportunities in the Middle East/GCC: ILIKOS Consulting Group and Phoenix Clinical Research

ILIKOS Consulting Group and Phoenix Clinical Research collaborate to facilitate access to the Middle East/GCC markets for biopharmaceutical and medical technology sponsors. ILIKOS, based in Paphos, Cyprus, advocates for exploring new regions despite industry complexities and risks, leveraging their location and network.

Phoenix Clinical Research, headquartered in Lebanon, specializes in patient access across MENA/GCC for chronic, acute, and rare conditions. They mitigate risks, manage regulations, and ensure operational excellence throughout clinical studies.

Together, ILIKOS and Phoenix bridge geographical, linguistic, and cultural gaps in clinical research. Their partnership focuses on streamlined communication, effective patient recruitment, and advancing therapeutic development to achieve significant milestones.