siRNA

The biOasis siRNA Gene Therapy Program

Rationale

The mapping of the human genome and advanced research in the role of genetics in many diseases have resulted in the discovery of the genes that either cause diseases or the genes that could be modified to treat certain diseases.

Small-Interfering RNA (“siRNA”) is a technology that can be used to reduce or stop the genetic expression of proteins that cause disease. The “knocking-down” of genes can be a powerful therapeutic tool when used to target disease-causing genes. siRNA binds to messenger RNA, inhibiting the synthesis of the selected protein. However, because siRNA therapeutics do not cross the blood-brain barrier on their own, the targeting of genes within the brain was not assumed to be possible.

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In May, 2014, biOasis announced that its proprietary peptide carrier, MTfp, effectively delivered siRNA across the blood-brain barrier and into brain cells. In July, 2014, the company further announced that it had achieved effective knock-down of the expression of a target gene in the brain in a sequence-specific manner. Independent analysis of brain tissue revealed that it reduced the expression of the target gene by 40 per cent to 50 per cent after a single dose.

Because of the success with MTfp and siRNA in this study, biOasis has established a new in-house siRNA Gene Therapy Program for the advancement of MTfp with siRNA. The in-house siRNA Gene Therapy Program was selected by biOasis using several criteria, including potential commercial value, unmet medical needs and the anticipated speed and cost of development of advancing siRNA therapeutics to clinical trials.

Background Study

Study: siRNA + MTfp

Disease: Undisclosed

Treatment: MTfp+ undisclosed siRNA Therapeutic

Medical Problem: There are many CNS diseases that may be treatable if a means of transporting siRNA across the BBB can be found.

Transcend Therapeutic: MTfp + undisclosed siRNA Therapeutic

Results

  • MTfp delivered the siRNA therapeutic across the BBB to the brain tissue.
  • MTfp + siRNA reduced the expression of the target gene in the brain by 40-50% after a single dose. (Note: Complete gene knock down is not desirable)

Plan of Action

biOasis has selected siRNA target therapies and disorders that will be announced at a later date. Potential candidates include ischemic stroke, amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease), and others.