Research and Development

Gene Silencing

Genesis was one of the early developers of RNAi technology, initially in plant science and then in human therapeutics. Due to the difficulty in achieving effective delivery of dsRNA in vivo, Genesis explored other options for achieving gene silencing as a therapeutic tool.

Genesis established a subsidiary company named Solirna Biosciences Ltd (soli: plural of solo) to develop the delivery of dual single strands of RNA to achieve gene inhibition through the RNAi mechanism. This gene silencing technology was invented by Genesis scientists. A Japanese investment group provided funding to Solirna, with Genesis retaining a substantial ownership position.

The technology is designed to deliver separate complementary strands of RNA and let them anneal inside the cell where they can induce gene specific RNAi, thus combining the best aspects of antisense and siRNA technologies.

Technology
Advantages
Disadvantages
Antisense

Enters cells easily in vivo

Difficult to design efficient antisense

RNAi
Easy to design
Potent activity

Difficult to deliver into cells in vivo

Genesis ssRNAi Gene Silencing Technology

Potent activity, easy to design and enters cells readily in vivo

 

The novel gene silencing technology being developed by Solirna has the potential to solve the siRNA delivery problem by combining the delivery advantages of antisense oligonucleotides with the potent mRNA knockdown of siRNA, to yield a novel and vehicle-free gene silencing technology.

The oligonucleotide will be delivered without a vehicle as chemically-modified, nucleobase-masked, single-stranded RNAs, which will form an active double-stranded molecule only when the masking groups are removed inside cells.

Intellectual Property

Multiple US provisional patent applications have been filed covering the design, modification and delivery of single-stranded oligonucleotides which anneal intra-cellularly and then initiate RNAi.