TS-001229 — Mutations in the Myotonic Dystrophy Protein Kinase gene (DMPK) cause an autosomal dominant inherited disease referred to as Myotonic Dystrophy. Myotonic Dystrophy affects more than 1 in 8,000 people worldwide. Myotonic dystrophy results in progressive muscle weakness, stiffness and wasting. Currently, here are no treatments for this disease. A team of researchers at Nationwide Children’s Hospital have designed short hairpin constructs of AAV-shRNA which can be used to silence DMPK mRNA and therefore potentially treat myotonic dystrophy.

TS-001227 — A team of physicians at Nationwide Children's Hospital have developed a process to improve the acceptance of implanted vascular grafts. This Tissue Engineered Vascular Graft (TEVG) is patient-specific. It seeds patient cells onto a biodegradable tubilar scaffold, which is designed to dissolve with hydrolysis so that only the growing vessel remains. This system, the Closed Seeding System, combines patient imaging data, 3D-printing capabilities, and efficient collection and subsequent seeding of patient cells onto the TEVG scaffolding.

TS-001226 — When it comes to medical application, dosage is influenced by physical attributes such as height and weight. In an emergency situation, the acquisition of these measurements can be difficult. A team of researchers at Nationwide Children's Hospital have developed a device that uses motion, optic and sensor technologies in conjunction with advancing software to provide estimated analysis of height and weight. This use of consumer-grade technology allows doctors to improve on existic standard of weight estimation and to make accurate decisions on dosage when in a medical emergency situation.

TS-001225 — Cystic fibrosis is an inherited disorder that affects cells that produce sweat and mucus, causing significant damage to the digestive system, lungs, and other organs. A team at Nationwide Children's Hospital has developed a non-invasive monitoring system to track and test a patient with this disease. This technology is a skin patch that measures the metabolomics of the patients sweat to evaluate the clinical health of patients afflicted with cystic fibrosis.

TS-001224 — Decks of cards have been used to facilitate knowledge for decades. A team of researchers led by Dr. Elise Berlan have developed a series of cards that combine summaries of contraceptive counciling information and patient preferences. This includes key components of contraceptive preferences which can then be used with a care provider to determine the best form of contraceptive for the patient's preference and decreases the stigma associated with discussing topics like contraceptives as adolecents or young adults.

TS-001178 — Currently there are few existing methods on coronary flow pattern automation. The DECFA platform fulfills this unmet need by predicting diseased coronary blood flow by integrating previously unutilized data features from (sonographic) Doppler echocardiography measurements, cardiac functional and other physiological data (e.g. heart rate, body weight, etc) using machine learning. The DECFA program is superior to manual intervention as it provides more efficient analysis, more accurately, and can accept raw video files of PW Doppler and Color Doppler B-mode files, applicable on mouse models, potentially applicable to humans. DECFA can analyze raw PW Doppler AND Color Doppler B-Mode AVI video files to calculate overall coronary blood flow and coronary flow reserve, and the separation of each coronary flow pattern into 4 distinct phases representative of the stages in a cardiac cycle. Benefits: More efficient analysis over manual intervention, less error than manual intervention, capable of accepting raw video files of PW Doppler and Color Doppler B-mode files, applicable on mouse models, potentially applicable to humans (not yet validated). New features include the analysis of raw PW Doppler AND Color Doppler B-Mode AVI video files to calculate overall coronary blood flow and coronary flow reserve, and the separation of each coronary flow pattern into distinct phases representative of the stages in a cardiac cycle. The machine learning aspect brings state-of-the-art technology to determine whether it may be useful in directly predicting/diagnosing coronary microvascular disease. Stage of Development: We are currently in the final stages of completing the data analysis for all of the in vivo coronary and cardiac physiological parameters that will be used to perform the final runs through the machine learning process. We did perform an “interim” analysis using about half of the data, the results of which were promising (inconclusive at this point, but they put the predictive value of coronary flow patterns above 90% for identifying diseased coronary blood flow). This process also uses the whole envelope instead of discrete points of the coronary flow pattern, in addition to the texture-analysis extension. After this process is complete in mice, we plan to obtain human coronary blood flow patterns to determine whether this could be clinically useful beyond research applications. Potential Applications/Markets: Our program could be utilized in a research setting for robust, comprehensive, and more efficient analysis of coronary flow patterns in mice measured through Doppler Echocardiography (It solves the problem of large inter/intra observer error and time required for manual analysis). The program could also be used clinically for use in the medical field for the same analysis if adjusted for human use. It could also be used as an add on feature to the VisualSonics Vevo 2100 software for added capabilities in analyzing PW Doppler coronary flow patterns and Color Doppler B-mode files. The parameters that we identify in our program could be potentially useful in clinical diagnostics/machine learning/prediction modeling for better identifying and predicting disease. Furthermore, we envision that this could be tested and applied to clinical coronary Doppler echocardiograms, with the readout being predictability of coronary microvascular disease based on the machine learning algorithm of coronary flow patterns. Opportunity/Seeking: Development Partner Commercial Partner Licensing IP Status: Know-how based Copyright

TS-001037 — When it comes to the safety of our children, innovation never stops. There have been many improvements to the safety of devices and receptacles that can be toxic or life threatening if consumed or exposed to skin. A team of researchers at Nationwide Children’s Hospital have incorporated this desire for security into a common household product: the spray bottle. Often filled with harmful chemicals, spray bottles remain one of the leading causes of chemical exposure injuries in children. The team at NCH has developed a “two-step authentication” spray nozzle that requires the dexterity beyond that of a small child. This dual trigger approach requires a full grip that prevents kids from accessing the contents of the spray bottle, while remaining easily usable by adults and seniors.

TS-001036 — School can be a daunting experience. Constant motion, public speaking and a new environment can increase anxiety in children, sometimes leading to school avoidance. A team at Nationwide Children’s Hospital has developed a solution, where exposure therapy procedures are combined with modern technology can improve the school experience for people of all ages. Using a Virtual Reality (VR) Simulation, clinicians are able to use the multi-user capability to interact with and guide their patient through new environments such as classrooms, hallways and lunchrooms, as well as scenarios known to trigger increased anxiety such as public speaking or asking for help. Biofeedback components help collect data so that the clinician can adapt the experience to the user. Although targeted for school-aged children, this technology can be modified to treat any person with school or public phobia.

TS-001035 — Shiny R is an open source platform that allows a framework to develop online applications. With minimal required background in coding principles, a team of researchers at Nationwide Children’s Hospital were able to display and interact with the analysis made of single-cell transcriptome. Shiny R generates visualizations that include UMAP plots and presents features of single-cell RNA and transcriptomic data without extensive training in R programming. Improvements made on this existing technology includes importing data, cell filtration, principle component analysis, clustering, dimensional reduction, merging datasets, and Graphical user interface (GUI)-based generations of gene expression plots. This significantly improves the visualization and analysis of single-cell transcriptome analysis.

TS-001034 — Salmonella is often caused by contact with animals that carry bacteria, contaminated food, or water. It has been observed that children are commonly afflicted by salmonella, and typically treatment includes fluids, medical care, and sometimes pharmaceuticals. A team of researchers at Nationwide Children’s hospital have identified a lead compound that includes a biofilm with anti-salmonella characteristics and acts as an inhibitor. Use of this compound with the antibiotic ciprofloxacin improves the elimination of bacterial infection in at-risk organs such as the liver and spleen.

TS-001033 — Intravascular Ultrasound Images (IVUS) is a process that uses micro technology to provide images of blood vessels, their inner walls (endothelium) and the inside of veins. The analysis of these images allows clinicians to analyze luminal and scaffold boundaries, identify the presence of stenosis, and perform computations of various geometric quantities. This process is fully automated and therefore eliminates inconsistencies and inefficiencies that are a direct result of current semi-automated or complex fully automated systems already in place.

TS-001029 — A major complication associated with breast implant prostheses is the occurrence of capsular contracture, occurring in 20%-25% of patients. Severe forms of capsular contracture constitute failure of the reconstruction with significant implications for increased cost owing to an increased need for recurrent medical interventions, as well diminished quality of life for patients. Capsular contracture occurs as the result of the patient’s immunologic ‘foreign body’ response to the implant material. The inventors’ vision is to develop a technology whereby the active metabolites of Tamoxifen (endoxifen) are conjugated to implant biomaterial in a manner allowing for localized delivery of endoxifen. They anticipate that local delivery of endoxifen will successfully reduce capsule formation around implant material by reducing the immunologic foreign body response. This is a technology that could be licensed to implant manufacturers (breast, implantable cardiac devices, etc.) Technology Overview: Breast augmentation and reconstruction is a common practice, especially in those afflicted by breast cancer. One of the most common issues that comes with this process is the formation of capsular contracture. This is a direct result of the patient’s immunologic ‘foreign body’ response to the implant material, which can impact the need for significant medical interventions and diminished quality of life. The current pharmacologic treatment for breast cancer is the chemical compound known as Tamoxifen, which acts as a chemo-preventative medication for hormone sensitive breast cancers. A team of researchers at Nationwide Children’s Hospital and Ohio State University aims to localize the delivery of tamoxifen to significantly reduce the immunologic foreign body response around implant material for use in both cancer-based breast reconstruction and cosmetic procedures Benefits: No pharmacotherapeutics currently exist to address capsular contracture and no biomaterial advances have been made to specifically reduce the foreign body response to breast implants Stage of Development: Mouse studies are currently underway using systemic delivery of Tamoxifen for treatment of capsular contracture in breast implants. A manuscript is in preparation. Future mouse studies will focus on local delivery of endoxifen for treatment of capsular contracture in breast implants; then look at other implant types and different coating types. Potential Applications / Markets: According to the report published by Allied Market Research, the medical implant industry estimated $85.38 billion in 2019, and is estimated to generate $147.46 billion by 2027, manifesting a CAGR of 7.2% from 2020 to 2027. According to a report published by Fortune Business Insights, the breast implant market was worth $2.76 billion in 2019 and is projected to reach $3.05 billion by the end of 2027, exhibiting a CAGR of 7.2% during the forecast period, 2020-2027. Opportunity / Seeking: -Licensing IP Status: Patent application submitted

TS-000973 — A recombinant oncolytic virus encoding either an adenosine deaminase or heterologous proteins can be used in treatment of a variety of diseases, as the primary purpose of these are to maintain and develop the immune system. A team of researchers at Nationwide Children’s Hospital have found a method that can address the delivery of adenosine deaminase into cancer cells, immune cells and the tumor microenvironment to aid in treatment for any disease or condition associated with adenosine or other associated markers.

TS-000972 — Natural Killer (NK) cells express a range of receptors to activate or inhibit certain cellular behavior to kill cancer cells. When an NK cell is deficient or dysfunctional, the efficiency of the NK cells is severely limited. VISTA is a protein sequence that activates T cells and acts as a moderator for the immune system. It has low-to-moderate expression but has been the target of study by a team of researchers at Nationwide Children’s Hospital led by Dr. Dean Lee. By removing VISTA in expanded NK cells, the inhibitory signal will be eliminated and thus resulting in an enhanced ability of NK cells to target cancers and overcome the immune-suppressive signals for improved cancer immunotherapy.

TS-000971 — The novel disease Coronavirus, also denoted as COVID-19, was recognized by the World Health Organization as an unknown etiology in December of 2019. Severe Acute Respiratory Syndrome (SARS) is a disease that presents flu-like symptoms that is caused by coronavirus (SARS-CoV). The current pandemic of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is causing tremendous economical, emotional, and public health burdens. A team of researchers at Nationwide Children’s Hospital have reengineered a live attenuated recombinant mumps virus to create a novel SARS-CoV-2 vaccine for infants and children under the age of twelve. As vaccination is the most effective strategy to prevent infectious diseases, this development is instrumental to the outcome of the COVID-19 pandemic.

TS-000970 — As technology has adapted to the modern expectations of instant communication, Artificial Intelligence (AI)- Based Embodied Conversational Agents, also called “chatbots,” have become more relevant. They are considered a convenient and natural conversation method, which can be adapted to healthcare so that clinicians and researchers will be able to identify and address the root causes of persistent health disparities. A team at Nationwide Children’s Hospital intends to use this application to focus on the social and mental health aspects in targeted urban neighborhoods. This considers rate of poverty, opportunity, and concentrations of racial/ethnic minority populations. The chatbot app would include a customizable avatar and will be able to improve traditional screening approaches as it enables data-driven and AI supported screening for at-risk populations.

TS-000969 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Adeno-associated virus (AAV) serotypes such as AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74 have properties optimized by these researchers. Chimeric Antigen Receptor T cells (CAR T) are comprised of an extracellular antigen recognition domain, intracellular T cell activation and co-stimulatory domains. These cells allow for potent and specific targeting of cancer cells, bypassing the need for antigen presentation and T cell receptor recognition. Generating CAR T cells using the process of lentiviral transduction has limitations stemming from the random integration of transgenesis, where oncogene activation, gene silencing, or negative effects on the CAR T antitumor efficacy are possible.

TS-000913 — Peptides can be used to stimulate antigen-specific T cells, allowing activated T cells to be isolated from immune individuals to be used in vaccination or treatment in others. The novel disease Coronavirus, also denoted as 2019-nCoV, was recognized by the World Health Organization as an unknown etiology in December of 2019. Severe Acute Respiratory Syndrome (SARS) is a disease that presents flu-like symptoms that is caused by coronavirus (SARS-CoV). A current process widely applicable to many pathogens uses the Miltenyi Prodigy device. In a study led by Dr. Dean Lee, his team found that this process can be adapted to SARS-CoV-2 using a specialty mix of peptides to isolate T cell immunity.

TS-000912 — There are a few prominent diseases that affect the kidney, such as nephrotic syndrome and diabetic nephropathy. To treat Type II diabetes, there is a readily available pharmaceutical known as pioglitazone that is often used in conjuncture with other compounds to reduce proteinuria in patients with kidney diseases. A team of researchers at Nationwide Children’s Hospital have developed a novel compound to act as a treatment agent in cases of kidney disease. The new design has similar insulin sensitizing effects as pioglitazone as well as its ability to reduce proteinuria. This compound, titled GQ-16, has similar efficacy as traditional Type II diabetes drugs and acts as a new indication for nephrotic syndrome or kidney diseases, with a significant reduction in side effects such as weight gain or adipogenesis.

TS-000911 — Gene therapy experts at Nationwide Children’s Hospital have made significant advancements in designing optimal viral vectors for producing Good Manufacturing Practice (GMP)-grade viral vector products. Adeno-associated virus (AAV) serotypes such as AAV1, 2, 2.5, 3, 5, 6, 8, 9 and rh74 have properties optimized by these researchers. A research team led by Dr. Kristin Heller has developed two AAV cloning backbones that use a stuffer sequence so that the plasmid backbone is oversized, thus reducing the frequency of packaging during AAV manufacturing. Using multiple cloning sites and optimized Inverted Terminal Repeat (ITR) regions, this process improves the AAV processes in the public domain. These cloning backbones include Kanamycin and Ampicillin resistance genes.

TS-000907 — Being able to detect the early onset of diseases and identify risk factors make early intervention and risk management possible. With the increasing volume of Electronic Health Records (EHR), many deep learning models have been developed and applied to various healthcare tasks, such as disease predictions, phenotyping, embedded learning and future cost prediction. A team of researchers at Nationwide Children’s Hospital has created a deep learning-based model for future medical risk prediction, named the Multilevel Self-Attention Model (MSAM) that offers a series of improvements to existing processes. The capabilities of MSAM include capturing the temporal information within medical claims, advancing deep learning approaches for the underlying relationships between medical codes, and providing clinically meaningful interpretations.

TS-000906 — Natural Killer (NK) cells express a range of receptors to activate or inhibit certain cellular behavior to kill cancer cells. Additionally, CD38 presenting cells have been used as a marker for cancer stem cells, specifically those that often avoid recognition when common surface antigen processes are used. CARK1 is a phosphorylate that impacts cell growth and development. A team led by Dr. Dean Lee has developed CD38k0 NK cells that they combine with CARK1 to create a series of monoclonal antibodies that targets cancer cells. Along with better cell targeting, this combination improves the efficacy of treatment in comparison to the CAR, NK, or CD38 antibodies as independent components.

TS-000878 — Chimeric Antigen Receptor T cells (CAR T) are comprised of an extracellular antigen recognition domain, intracellular T cell activation and co-stimulatory domains. These cells allow for potent and specific targeting of cancer cells, bypassing the need for antigen presentation and T cell receptor recognition. CAR T is mostly generated using lentiviral transduction of autologous T cells in conjunction with non-specific expansion in cell culture. CAR T-specific stimulation has been described using genetically engineered artificial antigen presenting cells (aAPCs). A strategy designed by Dr. Dean Lee and his team can select and expand any CAR T cell product in culture. This allows for safely infused exosomes and other membrane particles derived from aAPCs to also be envisioned as a pharmacologic, given to patients receiving CAR T to promote in vivo expansion and persistence of said cells. This approach could also be applied to antigen-specific TCR stimulation by modifying the aAPC to express the appropriate MCH-peptide as the antigen.

TS-000877 — Tracking mood regularly after a diagnosis provides data essential to the evaluation of treatment effectiveness. This includes medications, dosages, and alternative methods such as non-pharmalogical management. At this point, these assessments are only made in clinical visits that can have large spans of time between appointments and in a setting that can skew data, as it is primarily self-reported or clinically observed. A team at Nationwide Children’s Hospital have developed an app in phases that can generate objective mood assessment using vocal recordings. This app is remote, making it accessible in both a home and clinical setting. It would be useful as a screening tool and as an assessment tool, allowing it to track the progress made through therapeutic application. It connects patients with diagnosed mood disorders.

TS-000874 — Currently, the process to perform analysis of proliferation and migration rates of cells in wound healing arrays is very hands-on and time consuming. The Wound Healing Automated Analysis Pipeline (WHAAP) is a fully automated pipeline that can track cell migration, proliferation, and density or confluency of a live culture. After setting the threshold for cell recognition, it enables the user to effortlessly analyze proliferation and migration rates of cells in wound healing assays. Users can specify both test and control groups, as well as replicate as many experimental trials as desired, all stemming from an input of microscopic images taken at regular intervals. Each set is evaluated with unbiased image recognition held at a constant state. The data collected is used for a variety of calculations and estimations. WHAAP efficiently conducts all stages of analysis without requiring the user to possess any advanced knowledge of computer programming or biology. The output of this program is publication-ready, which only requires the user to interpret the data provided quickly and efficiently. Benefits: WHAAP adopts previous ideas for the analysis of wound healing assays and constructs them in a novel pipeline that enables automated analysis. WHAAP produces more in-depth data analysis and the enhanced data visualizations, all in a shorter runtime. Our software has the potential for an array of applications that require tracking cell movement and proliferation in culture. Stage of Development Preliminary testing was successful. After positive demos to faculty, we decided to take the software to production environment. We are currently finalizing the backend code ,after which, the UI will be created that would allow use via interaction with a web app hosted in an AWS serverless environment. At a later date, use of Self-learning deep neural network might be implemented to further improve the cell recognition of the software and enable full automation (user need only submit images to application) Potential Applications/Markets: WHAAP reduces the time and effort spent analysing the results of wound healing assays, a very popular laboratory technique used for functional analysis. Due to the sheer number of images now able to be produced during live cell experiments, other groups have developed tools to try eliminating the need to manually count cells and measure the change in leading edge position in wound healing assays. These other methods are all exclusively based on a simplistic measurement of % confluency. The issue that our lab encountered (and I'm sure other labs have experienced as well) is that the use of% confluency as a measurement more often than not fails to provide meaningful results. Furthermore, combining the data of multiple replicates and then multiple experiments remains time consuming and difficult to perform. WHAAP allows users to quickly and easily adjust cell recognition. In a future version of WHAAP, we plan to implement a machine learning based approach, specifically a self-learning deep neural network, to solve the problem of cellular detection to even more accurately track the rate of cell growth and movement in response to a wound. WHAAP introduces several new metrics that are captured during the assay, allowing generation of a wholistic data set that accurately captures the complete picture of the wound healing response. Our own preliminary data shows that without WHAAP's increased sensitivity and separation of multiple contributing features to wound healing, statistically significant changes in protein function (caused by genetic variation) would have gone undetected. WHAAP has a number of additional features that we believe further add to the value of the product: •eliminates the need for manual processing between steps of analysis (minimizing errors, bias, and need for background knowledge or expertise) •analyzes images more rapidly than other comparable software •performs statistical analysis on any combination of experimental conditions provided by the user •creates figures, tables, and charts that are formatted such that they can be immediately interpreted and used for presentations, manuscripts, grant applications, etc. Not only have the demonstrated improvements to the process of analyzing wound healing assays, but we predict that WHAAP will become invaluable to researchers performing these assays and has the potential to become the leading analysis tool in this market. Because wound healing assays are commonly used across a wide breadth of research concentrations, WHAAP has the potential for a high level of impact and a high number of customers. As previously mentioned, future planned improvements include performing cell recognition using machine learning methods. We would also like to enable WHAAP to analyze different wound shapes (specifically, circular wounds) which would further improve WHAAP's utility and reach. Seeking: Development Partner Commercial Partner Licensing

TS-000873 — The current timeframe required for current Good Manufacturing Product (cGMP) validation and approval is significant. The delay between development and approval is time intensive and the advancements that can improve treatment can be outdated by the time they reach the market. Genetic modifications would lead to restarting the production and approval process and delaying the introduction of the entry into human trials. Dr. Kevin Cassady and his team found that combinations of oncolytic viruses (OV) can be combined using current, approved cGMP stocks can be employed effectively and subsequently saving time that would have been spent on re-engineering, production, and validation, as well as the expense associated. The clinical outcome would be improved as this new process allows for a more rapid and cost-effective approach to clinical translation using existing stock of virus.

TS-000859 — Nephrotic syndrome (NS) is a common kidney disease found in children that creates an overabundance of protein in the urine, comparable to proteinuria in adults. As of now, there are no approved safe and effective treatment for NS, especially for those whose NS is steroid or multi-drug resistant. A team of researchers have identified a series of new molecular targets for future drug development. Using glomerular transcriptomes and informatic analysis, clinicians will be able to identify immunosuppressive approaches that are distinct from the current procedures.

TS-000858 — A laryngoscope is used to analyze and diagnose head and neck disorders, and aids in the treatment of what is found. This tool is often used for airway management and designed in a material that is not traditionally disposable. Due to the sensitive nature of the ailments that require a laryngoscope, efficiency and safety is key in an emergency. Using a modified 3D printed composition, the new design simplifies the burden of transportation and assembly, has a level of sterilization due to its disposable nature, and decreases the risk of contamination in urgent and emergent settings.

TS-000857 — CD33 is a receptor that spans multiple membranes and is expressed on myeloid lineage cells and sometimes lymphoid cells. A team of researchers led by Dr. Dean Lee have found a new way to modify existing CD33 processes to provide Natural Killer (NK) cells the additional ability to recognize cell targets expressing CD33. Acute Myeloid Leukemia (AML) expresses multiple antigens, where CD33 is the most common. This new process, labeled as CD33-CAR, shows a significant advantage in targeting AML that presents with CD33, especially in cases that resist unmodified NK cells.

TS-000856 — Alveolar Rhabdomyosarcoma is a malignancy that forms in a muscle, stemming from a soft tissue and residing in the lungs. A team of researchers led by Dr. Benjamin Stanton have developed an initial characterization of PAX3-FOXO1 that is localized across the genome associated with this disease. PAX3-FOXO1 localizes to both repressed and active regions of the tumor genome and is initially targeted as a driver oncogene. Additionally, the immediate-early targets of localization encode metabolic genes and cell cycle genes. By understanding this rare tumor oncogene, caregivers will be able to identify target genes and implement FDA-approved therapies.