10 May 2018 - - Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) has achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system and is planning to advance a pipeline of investigational RNAi therapeutics into clinical development, the company said.
RNAi therapeutics have the potential to prevent or reverse neurodegenerative diseases caused by dominantly inherited genes, such as in Alzheimer's, Huntington's, Parkinson's, and amyotrophic lateral sclerosis, where there are limited to no treatment options.
The company presented initial results from preclinical studies at the TIDES: Oligonucleotide and Peptide Therapeutics 2018 annual meeting being held May 7-10 in Boston, MA.
In a rat preclinical study, a single intrathecal injection of a novel siRNA conjugate resulted in broad distribution across the brain and spinal cord regions.
Robust and highly durable silencing of a disease target gene transcript was seen in all key anatomical regions of the brain and spinal cord.
Specificity of the silencing effect was confirmed with a second siRNA conjugate targeting an independent gene transcript ubiquitously expressed in the CNS.
The novel siRNA conjugates utilize Alnylam's enhanced stabilization chemistry platform with further modifications to enable broad CNS delivery and efficient uptake in neuronal cells.
Alnylam Pharmaceuticals is a biopharmaceutical company that discovers, develops, and commercializes novel therapeutics based on RNA interference. Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.