RNA Silencing FANA Technology

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The sequencing of the first human genome took about 13 years and US $3 billion. Today a similar task can be done in less than 24 hours for US $1000. The scientific community generates a plethora of genetic information and always seeks to understand the function of both coding and non-coding RNA. With advances in genetic regulatory networks and sequencing technologies, researchers are now able to efficiently link diseases/disorders with certain genes. This new understanding of many diseases’ genetic etiology creates a huge demand for technologies that can effectively address and treat the genetic component. To this end, AUM LifeTech aims to provide innovative, potent, and cost effective genomics solutions in the area of basic biomedical research, clinical diagnostics, and therapeutics.

AUM LifeTech is a Philadelphia based biotechnology company with a revolutionary nucleic acids platform in the gene silencing and regulation space. Our mission is to leverage the power and adaptability of our unique FANA RNA silencing technology to treat and cure some of the world’s most difficult diseases. In order to do so, our technology relies on one of the most important molecules in the body: RNA.

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RNA is the link between DNA and proteins.
DNA is the blueprint that contains the instructions for all proteins in the human body. However just as in the construction of a building, there are many intermediate steps between the plans and the finished product. DNA must first be transcribed into a molecule known as ribonucleic acid, or RNA, before the body can make the proteins it codes for. Certain types of RNA govern what DNA is transcribed and at what time. Overall, RNA serves many functions, and is vital to gene expression and regulation.

RNA is composed of four different building blocks, called nucleotides, which are arranged in a certain sequence. Depending on the order of these nucleotides, different amino acids will be strung together to form proteins. The RNA that copies genetic information from your genome and is used as a template to make proteins is called messenger RNA, or mRNA. Any disruption in the synthesis or function of mRNA would stop the production of the particular protein it codes for. When this disruption is done on purpose, it is called RNA silencing.

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Not all RNA that is transcribed codes for a protein, however, and instead functions as an important part of gene regulation pathways. In particular, a type of short non-coding RNA called microRNA (miRNA) regulates gene expression through RNA silencing. It cleaves mRNA that is complementary to its nucleotide sequence, which prevents the protein the mRNA coded for from being produced. Non-coding RNA includes both short and long non-coding RNA (lncRNA), both of which influence transcription factors and so modulate the expression of different genes. Using technology to block or destroy certain miRNA or lncRNA molecules can fundamentally change what proteins are made by the cell, and thus can potentially be turned to therapeutic use.

Conventional RNA silencing technologies face many challenges with their use.
There are many conventional technologies available to dissect and explore the wealth of genetic information now known, but many of them suffer from significant challenges. The biggest challenge is delivery, as numerous technologies are too large, negatively charged, or easily degraded to successfully enter cells and effect gene silencing on their own. They require a delivery vehicle, which can add substantial cost, toxicity, and off-target effects to the therapeutic. Off-target effects occur when the therapeutic blocks or destroys RNA that is not precisely complementary to its sequence, thus changing gene expression and protein production in an unintended, undesirable way.

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Further, researchers invest a lot of time in identifying the perfect RNA target for a disease or condition in the discovery phase, but then must often resort to small molecules and biologics when they translate to pre-clinical or clinical development. Many times this changes the therapeutic dynamics and ends in failure.

AUM LifeTech’s FANA RNA silencing technology holds the key to successful Nucleic Acid Therapeutics.
Our goal is to successfully address these challenges with our next-generation FANA RNA silencing and regulation technology. In doing so, we aim to bring discoveries to the clinic as soon as possible. AUM’s FANA RNA silencing and regulation technology can be used to very efficiently target and silence a variety of RNA modalities, including mRNA, microRNA, and long non-coding RNA. FANA technology can be used for discovery, translation, and potentially for therapeutic development. AUM has established several key collaborations and is currently in discovery and pre-clinical stages of therapeutic development for a wide spectrum of genetic diseases. Additionally, AUM is now making this versatile next-generation RNA silencing and regulation platform available to biomedical researchers across the globe.