Promoting Shorter HTT Protein Production Shows Promise in Mice

Promoting Shorter HTT Protein Production Shows Promise in Mice

ProQR Therapeutics’ experimental therapy QRX-704 significantly reduced levels of the toxic huntingtin (HTT) protein clumps that fuel Huntington’s disease in a mouse model of the disease, a study showed. The therapy, an antisense oligonucleotide (ASO) designed to promote the generation of a shorter, but working version of…

Dosing Paused in Phase 2 Branaplam Trial Due to Possible Side Effects

Due to possible side effects, dosing is being paused in the Phase 2 VIBRANT-HD clinical trial of oral branaplam (LMI070) in adults with Huntington’s disease. A scheduled assessment found “early signs” that “branaplam might be causing peripheral neuropathy,” a condition marked by pain or numbness in the extremities caused by damage…