Shots:

• Voyager’s program for Huntington’s Disease and Amyotrophic Lateral Sclerosis assessing VY-HTT01 at five wks. post-dosing in adult non-human primates & VY-SOD102 one-time intraparenchymal infusion after laminectomy
• The program demonstrated reduction in HTT mRNA by 68% in the caudate- 67% in the putamen- 73% in the thalamus- and 32% in cortical neurons & reduction in SOD1 mRNA in the spinal cord by 70% & 50% in the cervical and thoracic regions- 82% near the site of cervical injection & 22% in the lumbar region
• VY-SOD102 & VY-HTT01 is composed of an adeno-associated virus capsid (AAVrh10) & adeno-associated virus capsid (AAV1) respectively. The preclinical data will support its IND application in 2019

Ref: Voyager Therapeutics | Image:  Market Exclusive