Vascarta Reports Positive Phase I Results for Sickle Cell Treatment

The study, led by Dr. Gershwin Blyden, followed five patients over 29 days of sublingual treatment. Researchers observed a stabilization of red blood cell morphology, evidenced by a delayed sickling rate and a decrease in the Area Under the Curve for sickling biomarkers. Beyond physiological improvements, participants reported reduced levels of fatigue and depressive symptoms, with mean pain scores dropping from 7.0 to 6.6.

While the trial demonstrated favorable safety and tolerability, no significant changes were noted in adhesion indices, which measure the risk of vaso-occlusive crises. Vascarta’s leadership views these outcomes as a strong foundation for future dose-optimization studies. The company plans to file an Investigational New Drug application with the FDA later this year. VAS-101 is currently accessible to patients through 503A compounding pharmacies, pending a prescription from a licensed physician.