|SEARCH RESULT||Page: 1 Total number of Record(s): 14|
NOVEL NEXGENSEQUENCING TEST DEVELOPED FOR RETINOBLASTOMA
A novel test for mutations in the RB1 tumor suppressor gene based on deep DNA sequencing technology that allows for very high depth of coverage sequencing of the entire RB1 gene identifying inherited mutations that put patients at risk of developing retinoblastoma. The test is rapid taking less than 3 days and provides unprecedented sensitivity. This knowledge can better inform the correct treatment approach for patients when these mutations are identified and alert doctors to discuss with families the potential risks to siblings and offspring who may share or inherit the defective RB1 gene.
NOVEL THERAPEUTIC TREATMENT FOR INFLAMMATORY BOWEL DISEASES
NRG4 administration will provide therapeutic benefit to patients suffering from Crohns disease (CD) and ulcerative colitis (UC), known collectively as inflammatory bowel disease (IBD), as well as pediatric patients with necrotizing enterocolitis (NEC). NRG4 will promote intestinal epithelial cell survival by preventing apoptosis via NRG4 receptor ErbB4 signaling, effectively resulting in mucosal healing and improvement of the disease state. Furthermore, colitis induces recruitment of ErbB4+ pro-inflammatory macrophages that undergo apoptosis upon NRG4 stimulation. Thus ErbB4 signaling is a possible inhibitory feedback mechanism restricting macrophage numbers, and thus represents a potential therapeutic target to regulate inflammation in IBD as well as other inflammatory disorders in which macrophages play a prominent role. Administered NRG4 will supplement endogenous NRG4 protein which is reduced during the disease state, and will activate ErbB4 receptors which are upregulated but inactive due to the absence of endogenous NRG4.
AUTOLOGOUS NATURAL KILLER (NK) CELL-DERIVED EXOSOMES FOR ANTI-CANCER THERAPY
Natural Killer (NK) cells are a group of lymphocytes (a population of cells of the immune system) responsible for killing infected cells and cancer cells. They exert this function by releasing granules (exosomes) that deliver cytotoxic molecules such as perforin and granzyme B to target cells, which cause cell death. Billions of highly cytotoxic NK cells from patients can be grown in vitro and re-infused into the patient to treat cancer or leukemia. It has also been shown that these NK cells release large quantities of exosomes while growing in vitro. It is anticipated that NK-derived exosomes can be produced in therapeutic quantities as a ï¿½by-productï¿½ of growing the cells for treatment. Because the NK cells themselves are such potent killers of cancer cells, it is anticipated that their released exosomes will also be able to kill cancer cells. It is further anticipated that it will be possible to ï¿½engineerï¿½ the NK cells so that their released exosomes will be even more effective killers of cancer cells.
NOVEL COMBINATION THERAPY TO TREAT NEUTROPENIA
The combination of Am80 and G-CSF will provide therapeutic benefit to patients suffering from neutropenia beyond G-CSF alone. Am80, an oral retinoic acid derivative, promotes neutrophil maturation significantly better than G-CSF and effectively differentiates a GCSF-induced immature granulocytes into mature neutrophils. Unlike G-CSF, Am80 promotes the production of differentiated neutrophils that closely mimic fully mature neutrophils. Am80-induced neutrophils exhibit cellular morphology and functional reactive oxygen species (ROS) production characteristic of mature neutrophils. Together, Am80 and G-CSF work synergistically to provide sufficient numbers of mature neutrophils to combat infection and promote survival.
Investors: Yaniv Bar Cohen, MD; Gerald Loeb, MD, PhD
This novel micropacer can be affixed to the epicardial surface in minimally invasive procedures via a catheter or cannula. A single-chamber pacing system that is self-contained and can be percutaneously implanted in a patient without exteriorized leads. Where designed for use in a fetus, the leadless epicardial micropacemaker is deployed through the maternal abdomen under ultrasound guidance and allows for fetal movement without risk of dislodgement of the electrodes. Beyond this immediate application, adaptation of this system could be extended to newborns, infants, and even adults with limited venous access.
This novel micropacemaker design could replace standard single-chamber pacemaker techniques with implantation of the entire pacing system into the patientï¿½s thorax via a minimally invasive technique.
Despite phenomenal advances in MRI analysis methods for neurological research, the current state-of-the-art clinical technique for the detection of brain disorders in children continues to rely on an expert visually inspecting MR images without the benefit of more accurate numerical diagnostic tools. New clinical software that can quantitatively analyze scans of developing brains is urgently needed to address the needs of children with a variety of abnormalities, including hydrocephalus and white matter hypoplasia. Click-Brains, using a set of algorithms for brain structural MRI, detects anomalies in the brain morphology of children in the clinic. Click-Brains will use as input the brain scan of a patient and compare it to a reference image derived from a normal population of the same age/gender in order to pinpoint regions that are not following a standard developmental trajectory. Proof-of-concept is being sought by establishing Click-Brains ability to distinguish between two childhood neurological disorders, hydrocephalus and white matter hypoplasia, conditions that can be difficult to distinguish on MRI images using visual inspection.
RESPIVICE VENTILATION MANAGEMENT SYSTEM
Endotracheal intubation is the most common supportive therapy applied in both adult and pediatric intensive care units. Performing endotracheal intubations particularly in children is complex leading to complications during placement, extubation and weaning such that clinicians could benefit a great deal from additional diagnostic tools to monitor respiratory performance data. The Respivice system will (1) optimize ventilator support during the acute phase of illness (2) improve efficiency of weaning the mechanical ventilator (3) prevent re-intubation related to UAO and (4) optimize the efficiency of non-invasive ventilation management. Hospitals are being paid a flat reimbursement rate based on diagnosis and level of complexity. Hence, longer lengths of time on the ventilator and potentially preventable complications such as upper airway obstruction will lead to longer hospital lengths of stay, and loss of revenue for the hospital. Respivice can improve the quality and efficiency of care thereby substantially reducing hospital length of stay and length of mechanical ventilation.
The BestDose software uses sophisticated algorithms created by a team of physicians, pharmacologists, mathematicians, statisticians and aerospace engineers to control drug variability, individualize doses, and truly personalize medicine in the 21st century. The software is run by the clinician who is able to set a target for a patient (such as blood concentration of a drug that has been shown to be safe and efficacious). The program then combines the patients personal medical information with a drug model to generate a specific dose recommendation. This method saves lives, improve outcomes, reduce complications, shorten hospital stays, and decrease health-care costs. The software will be integrated with a common hospital electronic medical records system, substantially increasing use of the software.
NEW GUIDED GROWTH PLATE
* New Technology
Children need gentle guidance and correction in many aspects of their life including those with limb growth abnormalities. These children can benefit from a minimally invasive surgical procedure that involves the insertion of a small device (plate). The plate gently guides growth while allowing natural, safe and gradual correction of limb alignment.
In the growing skeleton, shutting down growth of a growth plate is performed for 2 reasons:
Angular deformity - shutting down growth on one side of a growth plate but allowing the other side to grow leads to angular growth, which can correct an angular deformity
Stopping growth - if one leg is too long, shutting down the growth of that leg, with devices on the right and left side of a bone, can allow the shorter leg to catch up
Often times as children get older there is not quite enough growth left to get the amount of desired correction. It usually takes months before the growth plate shuts down after a device is placed across it.
This new Guided Growth plate design reduces the time for the growth plate to shut down and potentially leads to more correction than would otherwise be available with existing devices.
FLUIDIC DEVICE FOR LONG-TERM EXPLANT CULTURE AND IMAGING
* New Technology
The global market for regenerative medicine is set to reach $30 billion by 2021 due to the growing population of the elderly and an increased prevalence of chronic diseases. Two roadblocks in testing potential drugs that enhance organ development and regeneration are that organ explants cannot be grown in culture over the long term, and culture conditions are not conducive to high throughput testing. A technology that circumvents these issues will speed up drug discovery in the regenerative medicine market. People have attempted long-term explant cultures. However, a lack of blood flow into and around the organ will result in shrinkage or non-functioning of the organs.
USC and CHLA researchers have designed and fabricated a fluidic device to maintain flow into the culture, which overcomes the problem of lack of blood flow into and around the organ and ensures the structure of the organs is maintained. Using this fluidic device researchers were able to keep zebrafish hearts and mouse embryos alive for up to 7 to 9 days. They also maintain a stable position for confocal and live imaging.
IMPROVED SEQUENCING GENOMIC VARIANT CALLING TOOL
* New Technology
Investors: Jonathan Buckley, MD, PhD
Detection of genomic variants are the cornerstone of personalized medicine. Variants are detected by sequencing DNA fragments from a patient\'s sample to create discrete runs of DNA sequence and comparing them to the reference human genome. All sequencing technologies are subject to error, in the form of a miscall of a base, skipping over one or more bases (a deletion) or erroneously inserting one or more bases into a read (an insertion). Genomic variant calling can be substantially improved by comparison of the variants observed at each base locus against a reference dataset to determine whether what is seen is consistent with background noise, or represents a true signal. It can be used for any type of variant, but since insertions/deletions (InDels) are the most difficult to call with confidence, it is likely to be particularly valuable for InDel calling.
The method could be implemented as a stand-alone variant caller (software package), or integrated into existing variant calling pipelines.
METHODS FOR KIDNEY GLOMERULAR ANGIOMODULATION AND PROTECTION
Investors: Laura Perin, PhD; Sargis Sedrakyan, PhD; Roger De Felippo, MD
Amniotic fluid stem cells (AFSC) are reported to have renoprotective properties in preclinical models of kidney disease. AFSC secrete extracellular vesicles (EVs) that can modulate glomerular signaling. Specifically EVs were characterized for their content and we identified specific micro-RNA (miRs) that modulate VEGF signaling within the glomerulus of mice affected by glomerular disease (Alport mice) and placenta. Therefore, EVs from AFSCs may have a protective role in diseases characterized by altered VEGF homeostasis in glomeruli, such as Alport syndrome or pre-eclampsia.
GLOMERULUS ON A CHIP
* New Technology
Investors: Laura Perin, PhD; Stefano Da Sacco, PhD; Roger De Felippo, MD
A kidney organ culture comprised of human amniotic fluid-derived podocytes and glomerular endothelial cells to mimic properties of the intact glomerulus both in terms of structure and filtration function. Inventors have worked out details on cell constraints and conditions to make this functional glomerulus-on-a-chip (GOAC).
NK-DERIVED EXOSOMAL MIR-186 INHIBITS CANCER CELL GROWTH AND IMMUNE ESCAPE
* New Technology
Investors: Paolo Neviani, PhD; Robert Seeger, MD; Muller Fabbri, MD, PhD
In neuroblastoma, the interplay between cancer cells and immune cells of the tumor microenvironment contributes to immune escape mechanisms and drug resistance. Targeted delivery of tumor suppressor miR-186 in MYCN-amplified neuroblastoma shows efficacy in preclinical models of disease. MiR-186 is present in NK cell-derived exosomes regardless of NK cell activation status. The investigators found that targeted delivery inhibited tumorigenic potential and overcame TGF-Î²1 dependent inhibition of NK cells. This therapy holds the promise of targeting several factors (MYCN, AURKA, TGF-Î²) that have been shown to drive the evolution of most cancers.