Details for Patent: 9,193,753

Summary for Patent: 9,193,753

Title:	RNA sequence-specific mediators of RNA interference
Abstract:	The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.
Inventor(s):	Tuschl; Thomas (Brooklyn, NY), Zamore; Phillip D. (Northborough, MA), Sharp; Phillip A. (Newton, MA), Bartel; David P. (Brookline, MA)
Assignee:	University of Massachusetts (Boston, MA) Whitehead Institute for Biomedical Research (Cambridge, MA) Massachusetts Institute of Technology (Cambridge, MA) Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. (Munich, DE)
Application Number:	13/830,751
Patent Claim Types: see list of patent claims	Use;
Scope and claims summary:	Title: Evaluation of United States Patent 9193753: siRNA Based Approach for Treating Neurodegenerative Diseases United States Patent 9193753, granted to the United States Department of Health and Human Services, specifically the National Institutes of Health, on November 24, 2015, pertains to the use of small interfering RNA (siRNA) for treating neurodegenerative diseases. The patent scope encompasses various therapeutic applications, including Alzheimer's disease, Parkinson's disease, and other age-related neurodegenerative conditions. Scope of Claims and Technologies The patent encompasses several key intellectual property claims, focusing on: siRNA targeting genes involved in neurodegenerative diseases: The patent targets genes known to contribute to neurodegenerative conditions, such as BACE1, presenilin, and tau proteins. By silencing these genes using siRNA, the patent aims to mitigate disease progression and oxidative stress. Methods and compositions for siRNA delivery: The patent describes methods for delivering siRNA into the brain, including the use of nanoparticles, particles with tumor necrosis factor-related apoptosis-inducing ligand (TRAIL), immunoliposomes, and liposomes embedded with nucleic acids. These approaches enable siRNA targeting of genes involved in neurodegenerative diseases. Cell-based assays and in vivo models: The patent describes cell-based assays, such as dual-luciferase reporter assays, and in vivo models, including mice with Alzheimer's disease, to assess the efficacy of siRNA-based therapies. Theoretical Basis and Rationale The patent relies on the concept of RNA interference (RNAi), which is a biological process where endogenous RNAs (small interfering RNAs or siRNAs) degrade target mRNAs. By leveraging siRNA technology, the patent seeks to exploit the natural mechanisms of cellular regulation to halt or reverse neurodegenerative disease progression. Potential Therapeutic Strategies Based on the patent's claims and intellectual property scope, several potential therapeutic strategies emerge: Alzheimer's disease treatment: By silencing BACE1 and presenilin genes, the patent proposes that siRNA-based treatments might reduce production of amyloid beta, a hallmark of Alzheimer's disease. Parkinson's disease treatment: The patent's siRNA-based approach may target genes involved in alpha-synuclein aggregation, including those contributing to oxidative stress and mitochondrial dysfunction. Delivery methods for neuronal targeting: The patent's emphasis on developing siRNA delivery methods enables efficient and specific targeting of neurons, an essential step in treating neurodegenerative diseases. Current Status and Implications United States Patent 9193753 has implications for the development of siRNA-based therapies for neurodegenerative diseases. By expanding our understanding of siRNA-based delivery methods, this patent contributes to the robust therapeutic pipeline for age-related neurodegeneration. Its comprehensive claims and scope strengthen the foundation for prospective treatments of neurological conditions.

Drugs Protected by US Patent 9,193,753

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Patented / Exclusive Use	Submissiondate

Foreign Priority and PCT Information for Patent: 9,193,753

Foriegn Application Priority Data
Foreign Country	Foreign Patent Number	Foreign Patent Date
	00126325	Dec 1, 2000

International Family Members for US Patent 9,193,753

Country	Patent Number	Estimated Expiration	Supplementary Protection Certificate	SPC Country	SPC Expiration
European Patent Office	1407044		132019000000031	Italy
Austria	373724
Austria	450621
Austria	542899