YolTech Therapeutics today announced that the first patient has been dosed with YOLT-201, the company’s first in vivo genome editing candidate being developed as a single dose, potentially curative therapy for hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
The study by YolTech is a single-arm, open-label, dose-escalation clinical trial (Clinicaltrials.gov: NCT06082050). This trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of YOLT-201 as well as the identification of an Optimal Biologically Active Dose (OBD).
ATTR-CM, a rare, progressive and debilitating disease. It is caused by the misfolding of unstable Transthyretin Protein (TTR), leading to the deposition of amyloid-like buildup in the myocardial interstitium, causing stiffening of the heart muscle and subsequent heart failure. Studies have reported the median survival time of approximately 3.5 years from the first diagnosis of ATTR-CM if left untreated.
Dr. Yuxuan Wu, Founder and CEO of YolTech, expressed, “The successful FPI in this trial is a significant milestone, representing YolTech’s relentless efforts in the field of in vivo gene editing are gradually becoming a reality. We extend our gratitude to all researchers, partners, and supporters involved in the YOLT-201 project, especially to the dedicated research team at FAHZU for their steadfast support of drug development in vivo gene editing. We believe through this therapeutic intervention, we will provide a novel treatment option for ATTR-CM patients, offering them new hope and an improved quality of life.”
This study is a part of YolTech’s series of clinical trials in vivo gene editing. The company remains committed to advancing research in the treatment of ATTR and other diseases. Through close collaboration with the clinical community, we will continue to pursue excellence, push the boundaries of medical technology, and provide more advanced and innovative medical solutions for patients worldwide.