The findings were the result of research partnership between Alnylam and Medtronic and supported by CHDI.
The results demonstrated that a small interfering RNA (siRNA) targeting huntingtin gene, when administered by intrastriatal infusion with convection-enhanced delivery (CED), results in widespread distribution of the siRNA.
The results also demonstrated significant silencing of the huntingtin mRNA throughout the striatum and administration of the RNAi therapeutic was well tolerated in these studies.
Alnylam research vice president Dinah Sah said the pre-clinical results extend earlier data on siRNA biodistribution in the central nervous system and degree and scope of therapeutic huntingtin gene silencing.