Development of sd-rxRNAs as Therapy for Retinoblastoma and Other Malignancies
Company: RXI PHARMACEUTICALS CORPORATION Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: …, and other CNS malignancies.Moreover, transient MYC down-regulation appears to be well tolerated at the organismal level, while dramatically suppressing tumorigenesis. However, MYCN proteins are considered to be 'undruggable' at the present time. siRNA-based drugs… more
New Building Blocks for the Synthesis of Modified RNAs
Company: AM Biotechnologies, LLC Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: … siRNAs targeting 2-secretase. In Phase II, AM will (a) scale reagent production up to commercial quantities and purity; (b) optimize a robust protocol for synthesis of PS2-2'-F-RNA; (c) evaluate the effects of PS2-2'-F modifications on aptamers and siRNA activity in… more
Identifying the drug target in dormant cancer cells
Company: DORMATARG, INC. Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: …f two lead compounds in order to link them to magnetic beads or perform photoaffinity labeling without destroying the activity of the molecule. The second aim is to extract the target protein for identification by mass spectrometry and validate its identity by siRNA knockdown of… more
Combination chemo-siRNA gene therapy of nonmuscle-invading bladder cancer
Company: OPTIMUM THERAPEUTICS, LLC Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: …at a new approach is needed for the remaining patients who are not adequately managed by intravesical MMC therapy. Survivin is a marker/predictor of bladder cancer aggressiveness and recurrence. We have developed a pegylated cationic lipid carrier (PCat) for survivin siRNA that… more
Ligand Directed Transdermal siRNA Delivery to Improve Wound Healing
Company: SIRNAOMICS, INC. Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: … such as transforming growth factor-ss1 (TGF-ss1), and COX-2 are upregulated in adult tissue and show reduced expression in fetal skin. Using RNA interference (RNAi) to down regulate a target gene expression, we have previously demonstrated that a multi-targeted siRNA… more
Engineering of Human Corneal Endothelial Grafts
Company: TISSUETECH, INC. Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: … of the corneoscleral rim normally discarded after conventional corneal transplantation. Thus, in this Phase I application, we would like to prove the concept that the size of HCEC monolayers can further be enlarged by optimizing the regimen of p120- catenin siRNA knockdown… more
High throughput RNAi based functional genomics in primary cells and in vivo
Company: ADVANCED RNA TECHNOLOGIES, LLC Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: … new RNAi platform that enables gene studies in a biologically relevant context with a long term goal of building a functional map of human genome. Introduction of small interfering RNAs (siRNAs) into cells with transfection reagents results in efficient gene silencing. … more
Method Development for a Next Generation Miniaturized Genomic Screening Platform
Company: ENGINEERING ARTS, LLC Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: DESCRIPTION (provided by applicant): Every gene has its own set of small RNA molecules, known as small interfering RNA (siRNA), that inhibit expression of the gene's proteins. The siRNA molecules are part of an ancient natural mechanism of gene… more
A Force-controlled Probe Based Platform for Single Cell Biomolecular Delivery
Company: INFINITESIMAL, LLC Agency/Program/Year/Phase: HHS / STTR / 2012 / 1Abstract: … tip that creates a small rupture in the cell membrane while the outer shell guides biomolecules to the point of injection. Picoliter control in fluid injection Large scale genomic and proteomic studies require delivery of drugs, conjugated nanoparticles, DNA, siRNA, and… more
SBIR Phase I: Biomolecular Cell Injection With Nanofountain Probe Systems
Company: INFINITESIMAL, LLC Agency/Program/Year/Phase: NSF / SBIR / 2012 / 1Abstract: …, and the ability to control injection volumes at the picoliter level. Injection of large number of cells is necessary for drug discovery and associated large scale genomic and proteomic studies. Such studies require delivery of drugs, conjugated nanoparticles, DNA, siRNA,… more
Optimization of SWCNT/siRNA complex formulation for tumor accumulation
Company: ENSYSCE BIOSCIENCES, INC. Agency/Program/Year/Phase: HHS / SBIR / 2012 / 1Abstract: … as other critical carbon nanotube patents, have been in-licensed or applied for by Ensysce and the company has initiated a number of research programs to utilize carbon nanotubesto treat cancer including that described herein to deliver short interfering RNAs (siRNA).… more
Improving self delivering properties of RNAi compounds through medicinal chemistr
Company: RXI PHARMACEUTICALS CORPORATION Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: DESCRIPTION (provided by applicant): Introduction of small interfering RNAs (siRNAs) into cells with transfection reagents results in potent and specific gene silencing by RNA interference (RNAi). Unfortunately, while siRNA-based drugs represent a potentially significant… more
Novel RNAi therapy for ALS and other Neurodegenerative Disorders
Company: RXI PHARMACEUTICALS CORPORATION Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: … is caused by dominantly transmitted mutations in the gene encoding cytosolic superoxide dismutase (SOD1). Transgenic expression of mutant SOD1 causes ALS in mice. In thesemice, suppression of expression of the transgene using either antisense oligonucleotides or siRNA slows… more
Cell Regulation with Polyvalent Nanoparticles
Company: AuraSense, LLC Agency/Program/Year/Phase: DOD / SBIR / 2011 / 1Abstract: … in the control of gene and protein expression. Objective 1 experiments will be designed to demonstrate and optimize: 1) Ability to deliver conjugate nanostructures into both prokaryotic and eukaryotic cells, 2) Ability to deliver oligonucleotides including siRNA, DNA… more
New Reagents for the Synthesis of Phosphorodithioate-Modified 2'-O-Methyl RNAs
Company: AM Biotechnologies, LLC Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: … of siRNAs targeting 2-secretase. In Phase II, AM will (a) scale reagent production up to commercial quantities and purity; (b) optimize a robust protocol for synthesis of PS2-2'OMe-RNA; (c) evaluate the effects ofPS2-2'-OMe modifications on aptamers and siRNA… more
Development of Specific Gene Silencing Methods and Reagents
Company: BOSTON BIOMEDICAL, INC. Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: … RNAi to specifically down-regulate the expression of any gene has had a profound impact on the study of gene function, and holds great promise for in vivo functional genomics. The current paradigm for inducing RNAi in mammalian cells relies on the use of a 21-bp siRNA… more
Neural Tract-tracing Nucleic Acid Carriers
Company: Innovative Surface Technologies, Inc. Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: … acid carrier under development at Innovative Surface Technologies, Inc. (ISurTec) for universal transfection of neurons in vitro and in vivo. This technology is platform-based and consists of a polymeric nanoparticle for high-efficiency non-viralgene and siRNA… more
Nanoparticles and Nanocapsules for Glioma Targeting
Company: LNKCHEMSOLUTIONS Agency/Program/Year/Phase: HHS / SBIR / 2011 / 2Abstract: … imaged-guided treatment of brain tumors. Throughout the study period, NPs will be manufactured at LNK and subsequently tested at UCH. In Aim 1, we will use both in vitro and animal models to critically examine NPs for targeting and delivery of chemotherapy and siRNA. After… more
Pachyonychia congenita clinical trial using therapeutic self-delivery siRNAs
Company: TRANSDERM, INC. Agency/Program/Year/Phase: HHS / SBIR / 2011 / 2Abstract: …, in the genes encoding keratins 6, 16, and 17. The major complaint of PC patients is the debilitating painful callusing and blistering which occurs on or near the pressure points of the feet. These defined regions on the soles of the feet were targeted for local siRNA… more
SBIR Phase I: Targeted Delivery of siRNA using Engineered Toxin Bodies
Company: Molecular Templates Agency/Program/Year/Phase: NSF / SBIR / 2011 / 1Abstract: This Small Business Innovation Research (SBIR) Phase I project will demonstrate targeted delivery of small interfering RNA (siRNA) to HER2 overexpressing tumor cells using a unique Shiga-like toxin 1A (SLT1A) scaffold. Molecular Templates has developed a SLT1A library of… more
Development of algorithm for identification of functional self-delivering RNAi co
Company: ADVANCED RNA TECHNOLOGIES, LLC Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: DESCRIPTION (provided by applicant): Introduction of small interfering RNAs (siRNAs) into cells with transfection reagents results in efficient gene silencing. Unfortunately, while siRNA-based functional genomics is widely used in vitro, the ability to apply this technology to… more
Ultrasound-triggered delivery of siRNA as treatment for diabetic kidney disease
Company: TARGESON, INC. Agency/Program/Year/Phase: HHS / SBIR / 2011 / 2Abstract: … in making gene therapy for kidney disease a reality. In the current proposal, we aim to develop a novel microbubble-based delivery vehicle for targeted TGF-2 gene therapy. We have developed a microbubble that can be conjugated with a payload of plasmid or siRNA, and… more
Characterizing an AMPA Receptor Splice Modulator in Preventing Epileptogenesis
Company: LIFESPLICE PHARMA, LLC Agency/Program/Year/Phase: HHS / SBIR / 2011 / 1Abstract: … and seizure susceptibility. Thus normalizing aberrant splicing represents a novel therapeutic strategy for preventing epileptogenesis. Splice modulating oligonucleotides (SMOs) have unique chemistries and distinct advantages over classic antisense oligonucleotides and siRNA,… more
ANTI-STATHMIN BIFUNCTIONAL SMALL HAIRPIN RNA: PRECLINICAL DEVE
Company: MUREX PHARMA Agency/Program/Year/Phase: HHS / SBIR / 2010 / 1Abstract: … a tumor selective, non-immunogenic, RES-stealthed delivery vehicle. Stathmin 1 (STMN1), a critical regulator of tubulin polymerization/depolymerization, is overexpressed in 86% of our patients. We have constructed a unique bi-functional shRNA (bi-sh) with enhanced siRNA and… more
Modular internalizing molecules for treatment of more than one stage of malignanc
Company: B3 BIO, INC. Agency/Program/Year/Phase: HHS / SBIR / 2010 / 1Abstract: … a drug to specific cells or tissues which could benefit from the drug activity
, and avoid delivery of the drug to other areas where exposure to the drug could result in overt toxicity or other undesirable side effects. Functional RNAi molecules (e.g., shRNA, siRNA, RNAi,… more