VTx-003 / VectorY - LARVOL DELTA

Therapeutic strategies for Huntington's disease: current approaches and future direction. (PubMed, Neurodegener Dis Manag) - "Additionally, the potential of VTX-003 and ANX005 in mitigating disease progression is explored. Despite these promising therapies, challenges persist, particularly in long-term assessment, delivery strategy, and off-target effects. Considering the future landscape and need, the review has strengthened the need of therapeutic interventions to enhance efficacy and safety, ultimately improving the quality of life of HD patients."

Journal • Review • CNS Disorders • Cognitive Disorders • Gene Therapies • Huntington's Disease • Movement Disorders • Psychiatry • Targeted Protein Degradation

VectorY to Present Poster on Huntington’s Preclinical Data at the Hereditary Disease Foundation, August 24-27 in Cambridge, MA (Businesswire) - "VectorY...announces today that it will present a poster at the Hereditary Disease Foundation 2022 Meeting in Cambridge, Massachusetts, on August 24-27, 2022. The poster shows that VectorY’s AAV vector, dose-dependently transduced AAV iPS derived human neurons and astrocytes ex vivo, and AAV vector-delivered transgenes were expressed long-term in the brains and spinal cords of mice and pigs. Several VecTab candidates were identified that effectively removed huntingtin aggregates from U2OS cells that expressed a CAG-repeat-expanded pathological form of huntingtin....The poster data shows that the platform is also able to target polyglutamine-rich proteins like HTT, demonstrating the clearance of toxic HTT aggregates."

Preclinical • CNS Disorders • Huntington's Disease