The Ability to Deliver siRNAs to the Brain Represents a Major Advance in the Potential Treatment of a Host of CNS Disorders through Modifying Expression of Disease Causing Genes
A pioneering biopharmaceutical company focused on overcoming the limitations of therapeutic drug delivery across the blood-brain barrier, has shown that a newly discovered peptide vector form of Transcend – Transcendpep – effectively delivers siRNA across the blood-brain barrier and into brain cells. This is an exciting development for potential treatment of a variety of brain disorders. RNA interference (RNAi) of gene function can be triggered by small single-stranded RNA molecules (small-interfering RNA; siRNA), which function to silence target genes in a sequence-specific manner. Therapeutics based on siRNA have been heralded to have the potential to reverse and eradicate human disease by targeting specific genes that cause or modify disease outcome. However, since siRNAs on their own do not cross the blood-brain barrier the targeting of genes within the brain was not assumed to be achievable. biOasis has now demonstrated delivery of siRNA across the blood-brain barrier using its new peptide vector, Transcendpep. After systemic injection, siRNA coupled to Transcendpep was shown to shuttle into the brain and became localized within brain cells. Since a variety of different siRNAs (specific for different disease-associated genes) can be delivered, this is a significant achievement for the Company and for the field of molecular therapeutics. Transcendpep-siRNA constructs thus become a potential cornerstone technology for development of new therapeutics for brain disorders, including, cancers of the brain, Alzheimer’s disease, Huntington’s disease, Amyotrophic lateral sclerosis (ALS) and other neuropsychiatric diseases, and for a variety of neuromuscular diseases, pain, and infections.
“RNAi is a promising and novel therapeutic approach for treatment of many CNS diseases. The successful delivery of Transcendpep-siRNA conjugates to the brain is therefore a significant advance and provides the foundation for exploring the potential of siRNA-based therapeutics for a host of different diseases of the Central Nervous System” says biOasis Founding Scientist Professor Wilf Jefferies.