Bioasis's Transcendpep brings siRNA into brain cells
2014-05-06 09:08 ET - News Release
Mr. Rob Hutchison reports
BIOASIS DELIVERS SIRNA TO THE BRAIN USING ITS TRANSCEND PEPTIDE VECTOR; 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
Bioasis Technologies Inc. has shown that a newly discovered peptide vector form of Transcend -- Transcendpep -- effectively delivers siRNA (small interfering ribonucleic acid) 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.
About Transcend & Transcendpep
Transcend and Transcendpep (a family of peptides derived from Transcend) offer the creation of a new class of drugs that can cross the blood-brain barrier to address unmet medical needs in the treatment of brain metastatic cancer and other brain disorders, such as neurodegeneration and metabolic disease. The blood-brain barrier represents the single greatest challenge in treating diseases and other disorders within the brain. Diseases such as cancer are readily treated in many areas of the body, but drugs designed to treat cancer and other brain disorders simply cannot penetrate the blood- brain barrier. The ability to more effectively permeate the blood-brain barrier opens the door for the creation of new drugs designed to treat a wide variety of neurological diseases and disorders. Additionally, Transcend offers the potential to take existing clinically approved drugs that are near their end of patent life and extend them.
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