Tau-gene-specific non-coding RNA genes for the therapeutic reduction of tau levels

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During genome transcription, both coding and non-coding transcripts are generated with a wide range of both size and coding potential. Among these are long non-coding RNA (lncRNA) genome transcripts, some of which are antisense to protein-coding genes (AS-lncRNAs). These AS-lncRNAs have been shown to regulate chromatin state, transcription, RNA stability, and translation of the gene.

Researchers at UCL have developed a therapeutic RNA molecule that corresponds with an AS-lncRNA, which can modulate the expression of a target gene. This has potential as a novel therapy for diseases where therapeutic gene suppression is desirable, for example therapeutic reduction of the tau protein in Alzheimer’s Disease and other tauopathies.

The Technology and its Advantages

The MAPT gene expresses the microtubule-associated tau protein, which is associated with several neurodegenerative disorders collectively known as tauopothies, the most prominent being Alzheimer’s disease (AD). Researchers at UCL have characterised a lncRNA gene, MAPT-AS1, which is antisense to the human MAPT gene. They have identified RNA transcripts of MAPT-AS1 that inhibit MAPT expression, as well as transcripts that can lead to enhancement of MAPT expression.

It has also been shown that other RNA molecules with sequences corresponding to an AS-lncRNA can modulate the expression of a target gene that is associated with an AS-lncRNA. Hence, this technology has been extended from MAPT to include other genes that also have an associated AS-lncRNA.

The researchers demonstrated that AS-lncRNA modulation occurs at the translational level, as opposed to the transcriptional level. This presents an advantage over conventional RNAi therapeutics, such as siRNA, in the form of potential higher potency and fewer off-target effects.

Market Opportunity

In 2014, the global antisense and RNAi therapeutics market was valued at USD 878.7 million and is expected to grow to USD 4.58 billion by 2022. The deals carried out in 2014 were valued at nearly USD 5.6 billion, the largest annual total ever in this space. There has been a resurgence in clinical investment, with several antisense and RNAi drugs in pivotal clinical trials and an increasing number of early clinical successes. This extensive product portfolio in the pipeline and expected commercialisations are poised to drive growth in demand.

Although RNAi technologies dominate the market currently, there is room for newer antisense technologies that have advantages over RNAi to provide a competitive edge for companies in this space.

Intellectual Property Status

Patent filed.

Further Information

Please contact Derek Reay, UCL Business PLC |+44 (0) 20 3456 2374 | d.reay@uclb.com