BD112

BD112 is an innovative in vivo gene editing therapy developed for the treatment of Huntington's disease. It utilizes a virus-like particle (VLP) delivery system to enable efficient and transient delivery of CRISPR-Cas9 gene editing machinery directly into target cells. The therapy specifically targets the human HTT gene, aiming to modulate its expression as a potential approach for treating Huntington's disease. The VLP-based platform offers advantages in technical performance and clinical development progress. As of August 2023, BD112 has received Orphan Drug Designation from the European Commission but remains at the preclinical stage with supporting data primarily from animal models[1][2].

Other names

Virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene