Several years before the global scientific community accepted the reality of RNAi in mammalian cells, Benitec was the first to show the universality of RNAi by demonstrating the down-regulation of target gene expression in human and mammalian cells, inventing a reliable means of triggering the RNAi process using DNA constructs. Benitec's core patents and patent rights are based on this early research and are supported by subsequent filings that extend the scope and jurisdiction of its intellectual property.
Benitec's patent estate represents a dominant position in DNA-directed gene silencing, and potentially the dominant position in RNAi applications for humans and mammals. Benitec has over 60 filed patents and has licensed several additional patents that extend the scope of its patent estate and enhance the utility and value of ddRNAi.
Benitec's main patented technology is DNA-directed RNAi, whereby transiently transfected or stably integrated DNA constructs are transcribed to form double-stranded RNA that induces gene silencing. The issued claims cover the design of such DNA constructs, whether they are inverted repeats coding for hairpin RNA or sense and antisense sequences under the control of separate promoters and regardless of the means of delivery.
On June 4 and 5, 2003, Benitec was awarded the first US and UK patents for the application of RNA interference to mammalian cells. Benitec's ddRNAi patent estate currently comprises 10 issued patents, covering 19 jurisdictions.
In addition to its patents, Benitec has significant proprietary know-how in the design, development and production of constructs, tools and high throughput methodologies for the use and practice of RNA interference.
RNAi patents - Among the companies, Alnylam and Benitec have strong patent positions.
On 5 June 2003, Benitec Ltd was granted its core technology patents in the US and the UK. These patents, with priority dating to 1998, describe a method for silencing any gene in any cell using ddRNAi. US Patent 6,573,099 is entitled "Genetic constructs for delaying or repressing the expression of a target gene" and UK Patent 2353282 is entitled "Control of gene expression". Both patents contain world first claims that describe the effect of RNAi in human cells and the DNA constructs which trigger RNAi.
Benitec Ltd, originally founded in Australia in 1997 (listed on Australian Stock Exchange), pioneered ddRNAi and was the first company to demonstrate RNAi in human cells. It holds a dominant international intellectual property position in RNAi...
Benitec's technology has the potential to treat human disease as a form of gene therapy. The types of diseases amenable to treatment with the company's gene silencing technology include; cancers where the shutdown of oncogenes can lead to disease remission; autoimmune disorders where the knockdown of specific immune system genes can reverse the disease process; and chronic degenerative viral infections where programming cells to silence critical viral genes can prevent infection.
Published with permission from Professor Jain's report entitled "RNAi- Technologies, Companies and Markets, Jain PharmaBiotech, Basel, Switzerland, August 2006.
| Patent/Application Number | Invention Title | Inventor(s) | Patent Summary |
| 6,573,099 (US) | GENETIC CONSTRUCTS FOR DELAYING OR REPRESSING THE EXPRESSION OF A TARGET GENE* | Graham, Michael W Rice, Robert N | The present invention relates generally to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention provides novel synthetic genes and genetic constructs which are capable of repressing delaying or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto. |
| PCT/ AU99/00195(WO99/49029) AU 29163/99 CA 2,323,726 CZ PV2000-3346 (295108) GB GB2353282 HK 01105904.3 NZ 506648 SG 200004917-1 ZA 2000/4507 |
CONTROL OF GENE EXPRESSION * | Graham, Michael W. Rice, Robert N Waterhouse, Peter Wang, MingBo | The present invention relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention utilises recombinant DNA technology post-transcriptionally modify or modulate the expression of a target gene in a cell, tissue, organ or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable or repressing delaying or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto are also provided. |
| US 10/821,726 US 11/180,928 US 11,218,999 | SYNTHETIC GENES AND GENETIC CONSTRUCTS COMPRISING THE SAME* | Waterhouse, Peter Graham, Michael Wang, MingBo Smith, Neil | The present invention relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the present invention utilises recombinant DNA technology to post-transcriptionally modify or modulate the expression of a target gene in a cell, tissue, organ or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable or repressing delaying or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto are also provided. |
| WO99/53050 | METHODS AND MEANS FOR OBTAINING MODIFIED PHENOTYPES * | Waterhouse, Peter; Graham, Michael Wang, MingBo Smith, Neil | Methods and means are provided for reducing the phenotypic expression of a nucleic acid of interest in eucaryotic cells, particularly in plant cells, by introducing chimeric genes encoding sense and antisense RNA molecules directed towards the target nucleic acid, which are capable of forming a double stranded RNA region by base-pairing between the regions with the sense and antisense nucleotide sequence or by introducing the RNA molecules themselves. Preferably, the RNA molecules comprises simultaneously both sense and antisense nucleotide sequence. |
| GB2377221 (GB), P-91678 (SG) 2002/7428 (ZA) AU01/000297 0170904 (WO) |
GENETIC SILENCING | Graham, Michael W. Rice, Robert N Reed, Kenneth C Murphy, Kathleen | The present invention relates generally to a method of inducing, promoting or otherwise facilitating a change in the phenotype of an animal cell or group of animal cells including a animal comprising said cells. The modulation of phenotypic expression is conveniently accomplished via genotypic manipulation through such means as reducing translation of transcript to proteinaceous product. The ability to induce, promote or otherwise facilitate the silencing of expressible genetic sequences provides a means for modulating the phenotype in, for example, the medical, veterinary and the animal husbandry industries. Expressible genetic sequences contemplated by the present invention including not only genes normally resident in a particular animal cell (i.e. indigenous genes) but also genes introduced through recombinant means or through infection by pathogenic agents such as viruses. |
| 10/861191 (US) 2527907 (CA) 04735856.9 (EP) 172191 (IL) 2006/508084(JP) 200507474-5 (SG) 2005/09813 (ZA) AU04/00075 (WO) |
DOUBLE-STRANDED NUCLEIC ACID | Graham, Michael W. Rice, Robert N Roelvink, Petrus W Suhy, David A Kolkykhalov, Alexander A Harrison, Bruce T Reed, Kenneth C. | The invention is directed towards constructs for RNAi techniques. The invention provides a ribonucleic acid (RNA) for use as interfering RNA in gene silencing techniques to silence a target gene comprising in a 5’ to 3’ direction at least a first effector sequence, a second effector sequence, a sequence substantially complementary to the second effector sequence and a sequence substantially complementary to the first effector sequence, wherein the complementary sequences are4 capable of forming double stranded regions with their respective effector sequences and wherein at least one of these sequences is substantially identical to the predicted transcript of a region of the target gene, and a nucleic acid construct encoding such an RNA. |
| 11/072592 (US) PCT/US2005/0017447 2005/087926 (WO) 2005222084 (AU) 2558771 (CA) 0580013979.5 (CN) 05727680.0 (EP) 177862 (IL) 2007-502094 (JP) 20067020986 (KR) 550284 (NZ) |
MULTIPLE PROMOTER EXPRESSION CASSETTES FOR SIMULTANEOUS DELIVERY OF RNAI AGENTS | Roelvink, Petrus W Suhy, David A Kolkykhalov, Alexander A | The present invention provides multiple-promoter expression cassettes for simultaneous delivery of RNAi, preferably to mammalian cells in vivo. |
| 11/251,076 (US) US05/037210 (WO) |
THERAPEUTIC RNAI AGENTS FOR TREATING RESTENOSIS | Couto, Linda Brashears, Sarah; Cunningham, Sara Mary | The present invention provides compositions and methods suitable for delivering RNAi agents against genetic targets in vascular and adjacent tissue in vivo so as to treat restenosis. |
| 11/256666 (US) US05/038139 (WO) |
THERAPEUTIC RNAi AGENTS FOR TREATING PSORIASIS | Reed, Kenneth C.; Brashears, Sarah J. | The present invention provides compositions and methods suitable for delivering RNAi agents against genetic targets in skin tissues so as to treat psoriasis. |
| 11/325244 (US) US06/000091 (WO) |
RNAi AGENTS FOR MAINTENANCE OF STEM CELLS | Evertsz, Elisabeth; Brashears, Sarah J. | The present invention provides compositions and methods suitable for delivering RNAi agents against genetic targets in stem cells so as to direct cell growth and differentiation |
| 11/340830 (US) | METHOD FOR DETECTION AND CHARACTERIZATION OF SHORT NUCLEIC ACIDS | Kolykhalov, Alexander; Schroeder, A. R. | A method of detecting and characterizing a target short RNA is provided. |
| 11/347028 (US) US06/004003 (WO) |
RNAi EXPRESSION CONSTRUCTS | Roelvink, Petrus W Suhy David A Kolkykhalov, Alexander A Couto, Linda | The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases or disorders. |
| 11/355516 (US) | RNAi EXPRESSION CONSTRUCTS WITH LIVER-SPECIFIC ENHANCER/PROMOTER | Roelvink, Petrus W Suhy, David A Kolkykhalov, Alexander A 'Kay, Mark A Giering, Jeffery C | The present invention provides compositions and methods suitable for RNAi specifically in the liver so as to treat diseases or disorders. |
| 11/413628 (US) US06/016507 (WO) |
MULTIPLE RNAi EXPRESSION CASSETTES FOR SIMULTANEOUS DELIVERY OF RNAi AGENTS RELATED TO HETEROZYGOTIC EXPRESSION PATTERNS | Evertsz, Elisabeth; Brashears, Sarah J. | The present invention provides compositions and methods suitable for expressing y-x multiple-RNAi agents against an allele or alleles of interest in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases. |
| 60/554861 (US) | EXPRESSION MODULATING AGENTS-II | Reed, Kenneth C. | US Provisional Filed 19 March 2004. |
| 60/792008 (US) | DIFFERENTIAL EXPRESSION OF SHORT HAIRPIN RNA BY MUTAGENIZED OR HYBRID RNA POL III PROMOTERS | Roelvink, Petrus W Suhy, David A | The present invention provides compositions and methods suitable for expressing y-x multiple-RNAi agents against an allele or alleles of interest in cells, tissues or organs of interest in vitro and in vivo so as to treat diseases. |
| 11/244314 (US) | MODULATION OF HAIR GROWTH | Reed, Kenneth C. | The present invention provides methods and compositions including RNAi agents for modulating the growth, development, or maintenance of hair or hair follicles in vertebrate animals. |
