The collaboration will combine Targeted Genetics’ adeno-associated virus (AAV) delivery platform with Sirna’s Huntington’s disease (HD) program and knowledge of RNA silencing technologies.
The focus of the collaboration will be the development of a therapeutic short interfering RNA (siRNA) targeting the gene that encodes the Huntington’s disease protein.
Sirna has published data demonstrating that the delivery of siRNA, using an AAV vector, efficiently inhibited gene expression in an animal model of spinocerebellar ataxia 1, a member of a class of inherited human neurodegenerative diseases that includes HD.
Under the terms of the agreement, development costs and revenue from partnering and sales of products developed under the collaboration will be shared between Sirna and Targeted Genetics.
“We are excited to continue expanding our AAV-based gene delivery platform into the area of RNA interference by working with Sirna,” said H. Stewart Parker, president and CEO of Targeted Genetics.