TIER 1: Up to a Total of $50,000

Delesha Carpenter

Division of Pharmaceutical Outcomes and Policy

Non-Invasive Technology to Continuously Monitor and Improve Patient Medication Adherence

Ingestible sensors and smart pills hold the potential to revolutionize how we measure and monitor patient medication adherence; however, the impact of these technologies has been limited by low patient acceptability and high costs. Our goal is to develop a new non-invasive technology that is low cost, acceptable to patients, and provides patients with personalized adherence information.


Alexander Golbraikh

Division of Chemical Biology and Medicinal Chemistry

Large-Scale Polypharmacology Modeling Using Deep Learning

We will explore deep learning methods for polypharmacological modeling to accelerate drug repositioning in different therapeutic areas. Prediction of off-target toxicities with such models can help to reduce the number of drug failures. Analysis of our models will help us to understand the structure-activity profiles as well as relationships between chemical structure and biological function.


Shawn Hingtgen

Division of Molecular Pharmaceutics

Cytotoxic Stem Cell Therapy for Pediatric Brain Cancer

Medulloblastoma (MBM) is the most common brain cancer in children. We will develop a game-changing approach to MBM therapy where tumor-homing neural stem cells seek out invasive MBM cells and deliver anti-cancer gene products to eradicate local and invasive tumor foci. This innovative therapy will address an enormous medical need and move MBM treatment in exciting new directions.


Weigang Huang

Division of Chemical Biology and Medicinal Chemistry

Profiling Cellular Phosphoinositide Metabolism for Disease Diagnosis

Phosphoinositides (PIs) are a family of essential signaling molecules that regulate a wide range of cellular processes. Aberrant regulation of PI metabolism is an established hallmark for many diseases. We propose a novel fluorous approach that will enable the systematic profiling of PI metabolism in live cells, which can serve as a diagnostic tool for diseases.


Robert Hughes

Division of Chemical Biology and Medicinal Chemistry

Optogenetics, Cytoskeletal Dysregulation, and Disease

We are developing a suite of light-activated, protein-based switches that enable the manipulation of cytoskeletal dynamics at will in living cells. These switches may enable us to rescue, rejuvenate, or otherwise prolong the livelihoods of diseased cells and tissues in a wide variety of disease states in a tightly controlled, non-invasive manner.


Alexander Kabanov

Division of Molecular Pharmaceutics

Carolina Nanoformulation Workshop

The Carolina Nanoformulation Workshop (CNW) is an innovative, state-of-the-art didactic-cum-practical training workshop at UNC-Chapel Hill that addresses key drug delivery issues relevant to both industrial and academic scientists. CNW develops a strong comprehensive educational resource, and fosters potential academia-industry partnerships to stimulate entrepreneurial development.


Alexander Kabanov

Division of Molecular Pharmaceutics

RECOPE: Reverse Conceptual Product Engineering

RECOPE is a comprehensive developmental and educational methodology to facilitate pharmaceutical research translation from bench to bedside and increase clinical relevance in academia for faculty and staff. RECOPE will plug into scientific, educational, and commercialization resources and expertise at UNC and RTP to maximize the impact on the entrepreneurial development, research and education.


Dmitri Kireev

Center for Integrative Chemical Biology and Drug Discovery

Molecular Simulations of Ultra-Large Biological Systems

Our ultimate goal is to build a computer system that can reproduce the inner workings of a cell. Such a system will be a major research tool in drug discovery and molecular biology. The objective of this project is to develop a pilot software and perform a proof-of-concept study. The innovation of our approach involves the creation of synergy between the scientific disciplines of molecular dynamics and systems biology.


Samuel Lai

Division of Molecular Pharmaceutics

Shoebox-Sized Plasmapheresis Machine for Cheap & Rapid Generation of Convalescent Serum in Africa

Current healthcare infrastructure in third world, resource-limited settings do not allow for preparation of convalescent serum, an important toolset needed in our fight against emerging infectious diseases. We seek to combine the latest in water sanitation technologies to enable rapid and cheap preparation of convalescent serum.


Rihe Liu

Division of Chemical Biology and Medicinal Chemistry

Decipher a Highly Specific Biomarker for Targeted Treatment of Pancreatic Cancer

Pancreatic ductal adenocarcinoma (PDAC) is among the most aggressive and dismal of all human cancers, often carrying a very poor prognosis and horrendous survival rate. One of the major challenges in developing precision medicine against PDAC is the lack of biomarkers that are highly specific to PDAC. The results from this project will greatly facilitate the early diagnosis and targeted treatment of PDAC.


Craig Lee

Division of Pharmacotherapy and Experimental Therapeutics

Solving the Mystery of Highly Variable Drug Disposition in Pregnant Women: Are Unique Hepatic Drug Metabolizing Enzymes Activated During Pregnancy?

This project will ignite new opportunities for research in the field of clinical pharmacology in special populations. We will accomplish this by determining whether unique drug metabolizing enzymes are (1) activated by the dramatic sex hormone changes that occur throughout pregnancy, and (2) contribute to the unexplained variability in drug clearance and effects that occur in pregnant women.


Xin Ming

Division of Molecular Pharmaceutics

P-Glycoprotein Targeted Antibody Conjugates for Combating Chemoresistant Tumors

P-glycoprotein (Pgp), which contributes to drug resistance in approximately half of known human cancers, remains undruggable after over 30-years of intensive research. We aim to utilize antibody conjugates to detect and treat chemoresistant cancers that overexpress Pgp. By turning Pgp into a “Trojan Horse” for targeted delivery, we may provide the first clinically effective approach to tackle Pgp-mediated drug resistance nearly 40 years after its discovery.


Bill Zamboni

Division of Pharmacotherapy and Experimental Therapeutics

Enhancing Tumor Delivery of Nanoparticle Anticancer Agents Using Microbeam Radiation Therapy

The promise of nanoparticle (NP) anticancer agents remains largely unfulfilled due to low tumor delivery, in which only 10-20% of NPs are distributed to tumors.  Thus, there is a strong need to discover methods that can capitalize on the promise of NPs by significantly and safely enhancing tumor delivery.  We will evaluate if novel microbeam radiation therapy (MRT) significantly enhances NPs delivery to tumors with minimum toxicity to normal tissue.

TIER 2: Up to a Total of $200,000

Emily Hull-Ryde

Center for Integrative Chemical Biology and Drug Discovery

Controlling the Mucus That Kills Pulmonary Patients

50 million Americans suffer from asthma, chronic obstructive pulmonary disease, and cystic fibrosis. A hallmark of these diseases is excessive mucus that can lead to airway plugging. Current therapies are not effective in reducing airway mucus. Our work will lay the foundation for developing novel inhibitors of a protein whose role in airway mucus overproduction was recently discovered at UNC.


Samuel Lai

Division of Molecular Pharmaceutics

Carolina E(I) Lab: A Multidisciplinary, Entrepreneurial Experience in Transforming Bold Ideas Into Successful Ventures

To develop future leaders in pharma/biotech R&D, we must fuse classical graduate student/postdoc (GS+P) education with experience and training in innovation, multidisciplinary teamwork and entrepreneurship. The E(I) Lab is an 8-month program bringing GS+P from diverse disciplines across UNC to conceive and execute innovative solutions to unmet needs in healthcare.


Jacqui McLaughlin

Division of Practice Advancement and Clinical Education

Transforming Data Into Knowledge: Fostering Institutional Effectiveness Through Real-Time Analytics

As higher education becomes increasingly data rich, we must demonstrate achievement of institutional initiatives in new and innovative ways. We propose a state-of-the-art data analytics system that provides decision support across the School and Eshelman Institute for Innovation. This system will advance our efforts to institutionalize assessment and accelerate change in knowledge management.

TIER 3: Up to a Total of $750,000

Shawn Hingtgen

Division of Molecular Pharmaceutics

Transdifferentiation: A Novel Approach to Personalized Cancer Therapy

Our multi-disciplinary team will create a disruptive advancement in the treatment of aggressive brain cancer. We will use next-generation technology to convert the skin of cancer patients into novel personalized tumor-homing neural stem cell therapies (iNSCs). We will then test multiple aspects of iNSC therapy using our unique pre-clinical models that closely mirror clinical cancer treatment.


Leaf Huang

Division of Molecular Pharmaceutics

Priming the Liver to Resist Cancer Metastasis

We will formulate a nanoparticle for the delivery of plasmid DNA to the liver resulting in the local expression of an engineered CXCL12-trap protein. Chemokine, CXCL12, is a key factor in the metastasis of colorectal cancer cells, as well as the recruitment of immunosuppressive cells. This formulation almost completely resolved any occurrence of colorectal liver metastasis. No metastasis in any other organs was found.


Samuel Lai

Division of Molecular Pharmaceutics

Engineered Antibodies With Carefully Tuned Mucin-Affinity for Enhanced Mucosal Protection

All exposed organs not covered by skin are coated with a mucus layer; these mucosal surfaces are prime sites for infectious disease transmission. Building off of our pioneering discovery that antibodies can interact with mucins to trap pathogens, we are focusing on engineering novel antibody constructs that possess even greater ‘muco-trapping’ potency for protection against mucosal infections.


David Lawrence

Division of Chemical Biology and Medicinal Chemistry

Light-Triggered Launching of Anti-Glioblastoma Therapeutics From Cellular Silos

We will explore the preparation and properties of neural stem cell-conveyed phototherapeutics and the wavelength-programmed release of drugs (1) in well-defined 2D co-cultures with target cells, (2) to target cells in 3D culture, and (3) to glioblastoma sites in animal models.


Jian Liu

Division of Chemical Biology and Medicinal Chemistry

Developing Carbohydrate-Based Medicines

Carbohydrates play essential functions in regulating various physiological and pathological processes. Advanced methods in designing and synthesizing carbohydrate molecules will open a new path for drug discovery for cancer, cardiovascular diseases and microbial infections. Three projects are proposed to improve the pharmacology of heparin, a carbohydrate-based drug for anticoagulant therapy.


Mary McClurg

Division of Practice Advancement and Clinical Education

Innovations in Practice Transformation: Advancing Medication Optimization in Primary Care

We are engaged in research to create best practices in optimizing medication use to influence new value-based payment and care delivery models and improve patient care. Through this award, we will address an unmet need by creating innovative technologies to facilitate the assimilation and dissemination of best practices throughout the country. These technologies will be key to accelerating the adoption and scale of new approaches to optimizing medication use.

TIER 4: Up to a Total of $2,000,000

Alexander Tropsha

Division of Chemical Biology and Medicinal Chemistry

Center for Innovation in Pharmacy Simulation (CIPS)

The Center for Innovation in Pharmacy Simulations will develop a state-of-the-art, data-driven technology platform in support of all major missions of the School. We will develop simulation tools based on serious gaming concepts to enhance all phases of education, both professional and graduate, and pharmaceutical research including medication discovery, delivery, optimization, assessment, and practice.


Tim Willson

Structural Genomics Consortium – UNC

The SGC-UNC: A Center for Open and Collaborative Target Discovery

The SGC-UNC promotes innovation through open sharing of research, material, and data. Greater than 80% of the enzymes known as protein kinases are understudied and their role in human biology is poorly understood. We aim to generate a functional map of these kinases to guide discovery of new medicines, through the creation and study of small molecules (called chemical probes) in a wide range of human diseases.

TIER 1: Up to a Total of $50,000

Kristy Ainslie

Division of Molecular Pharmaceutics

Long Acting Formulations for HIV Therapy

This project proposes chemistry for the sustained release of ART therapy for the treatment of HIV/AIDS. Using chemical modifications, we aim to create a pro-drug that can then be formulated for long-acting release of therapeutic drug. Also these proposed methods keep in mind inexpensive scalable production of the formulation.


Elena Batrakova

Center for Nanotechnology in Drug Delivery

Engineering 3D Models of Cancer Metastasis with Pro-Inflammatory Microenvironment for Cancer Immunotherapy

Three-dimensional in vitro models fill the gap between cell cultures and whole-animal systems. We aim to engineer ex vivo cancer metastases with pro-inflammatory microenvironment in these 3D models, and utilize them to develop cancer immunotherapy. We propose a revolutionary approach for cancer treatment by using the patient’s white blood cells, engineered to target and kill cancer metastases.


Albert Bowers

Division of Chemical Biology and Medicinal Chemistry

Evolving ‘Unnatural’ Natural Products to Target Orphan GPCRs

Our ultimate goal is to build synthetic microorganisms that invent new drugs. We will harness natural biosynthetic chemistry for targeted evolution of new therapeutics against pharmacologically important GPCRs. This work will contribute to broadly applicable and transformative technologies for completely cell-based high-throughput screening for drug development.


Masuo Goto

Division of Chemical Biology and Medicinal Chemistry

Novel Strategy for Prevention and Overcoming Multidrug Resistant Cancer by Small Molecule

Multidrug resistance in cancer is often correlated with the overexpression of P-glycoprotein (P-gp), a major ATP-dependent drug efflux pump. We plan to target ectopically expressed intracellular P-gp in MDR cancer by using novel small molecules, which selectively induce apoptosis only in MDR cancer. We will demonstrate a novel therapeutic strategy for overcoming the cancer MDR phenotype.


Samuel Lai

Division of Molecular Pharmaceutics

Hijacking Vaccines: Durable Immunity Via Genetic Editing of B Cells by CRISPR/Cas9

Achieving protective levels of antibodies in the peripheral circulation is crucial to effective defense against many infectious diseases; unfortunately, this has proven to be exceedingly challenging for many pathogens. Here, we propose a vaccination strategy that harnesses the latest advances in genomic engineering to bypass current limitations of vaccines and passive immunization strategies.


Melanie Priestman

Division of Chemical Biology and Medicinal Chemistry

Proteasome Sensors as Potential Biomarkers for Neurodegenerative Diseases

Neurodegenerative diseases such as Alzheimer’s and Parkinson’s are estimated to affect six million Americans and cost the U.S. over $800 billion each year. Early diagnosis and treatment are essential if we want to stop the progression of these debilitating diseases. We believe that the decreased activity of the proteasome can be used as a biomarker before clinical symptoms occur.


Tim Wiltshire

Division of Pharmacotherapy and Experimental Therapeutics

Clinical Implementation of Pharmacogenomics: The Actionable Genome

The goal of this project is to provide a direct path to the clinical implementation of pharmacogenomics (PGx). That is, we will develop a novel test that assesses genetic information on 18 genes, which we call the actionable genome. If we know the genetic information from these genes we can more precisely prescribe and dose multiple drugs. This should be the new standard for personalized medicine.


Yu Zhao

Division of Chemical Biology and Medicinal Chemistry

Develop Novel Natural Product Inspired Library of Multi-Target Compounds for Anticancer Drug Discovery

To take full advantage of the unique skeleton of natural product (NP) and the enormous data generated in current drug research, we will integrate polypharmacophores or privileged fragments from drugs or ligands of current top anticancer drug targets into three types of NP. The multi-target leads so discovered should hold great promise for developing new anticancer drugs with excellent efficacy.

TIER 2: Up to a Total of $200,000

Rahima Benhabbour

Division of Molecular Pharmaceutics

Fabrication of Innovative Intravaginal Rings by State-of-the-Art 3D Printing Technology

Women account for more than half of all people living with HIV and over 2,800 women a day are infected with HIV. Our goal is to develop a female-controlled method for prevention using intravaginal rings. Using a state-of-the-art engineering process we will fabricate IVRs in a way that cannot be achieved with conventional engineering, i.e. injection molding and hot-melt extrusion.


Nathaniel Hathaway

Division of Chemical Biology and Medicinal Chemistry

Development of Chemical Heterochromatin Inhibitors as Novel Epigenetic Cancer Therapies

Disruption of core epigenetic pathways underpins a large swath of human cancers. Heterochromatin gene repression has been found at fault in a growing number of human tumors including that of the breast, prostate, pancreas and liver. Here we advance first in class inhibitors of heterochromatin gene repression discovered at UNC and examine their clinical potential using multiple human cancer models.


Samuel Lai

Division of Molecular Pharmaceutics

Engineered Bispecific Fusion Proteins (BFP) for Targeted Delivery of Therapeutic Nanoparticles and Viral Vectors

Specific delivery of a therapeutic payload to target cells remains a major bottleneck in enhancing efficacy and reducing side effects of many particle-based therapies, including both synthetic nanoparticles and viral vectors. Here, we propose engineering bispecific fusion proteins that can broadly facilitate targeting of particles to diverse cells with molecular specificity.


Rihe Liu

Division of Chemical Biology and Medicinal Chemistry

Novel Single Domain Antibody Mimics for Targeted Cancer Therapy

The escape from immunosurveillance is indispensable for the initiation, progression, and metastasis of most cancers. This project focuses on developing novel targeting ligands that can be integrated with chemo/cytokine traps for precise immunotherapies against malignant tumors.


Shawn Hingtgen

Division of Molecular Pharmaceutics

Systemic Stem Cell Therapy for Simultaneous Treatment of Breast Cancer Metastases in Multiple Organs

Thirty percent of women diagnosed with breast cancer develop metastatic disease, yet the disease remains incurable. We propose a new approach to treatment, where tumor-homing engineered stem cells are systemically infused to simultaneously eradicate the primary breast cancer lesion, extracranial metastases, and cancer metastases spread to the brain. This renews hope for a cure.


Alexander Kabanov

Division of Molecular Pharmaceutics

Magneto-Mechanical Cancer Nanotherapeutics

We propose a novel cancer therapy approach, in which cancer cells are destroyed without the use of chemotherapeutic drugs by mechanical motion of magnetic nanoparticles actuated remotely by applied alternating current magnetic fields of very low frequency. Such fields and treatments are safe for surrounding tissues but disrupt the cytoskeleton and kill cancer cells while leaving healthy cells intact.


Qisheng Zhang

Division of Chemical Biology and Medicinal Chemistry

“Clickable” Assays of Metabolic Enzymes for Precision Medicine

Endogenous small molecules, which are biosynthesized by metabolic enzymes, present an additional axis to the “central dogma” of life. The proposed work aims to develop an innovative technology platform to assay metabolic enzymes and profile endogenous small molecules at the single cell level. Such an assay and profiling, when used in a patient’s cells, would be novel tools for precision medicine.

TIER 4: Up to a Total of $2,000,000

William Zuercher

Structural Genomics Consortium – UNC

The UNC Catalyst for Rare Disease Drug Discovery

Although mutations have been identified for thousands of monogenic rare diseases, progression of this knowledge to disease understanding and, ultimately, therapy or cure remains challenging. The UNC Catalyst for Rare Disease Drug Discovery engages rare disease foundations and innovatively addresses this gap with an open platform for generating knowledge and providing key research tool materials.

Up to a Total of $25,000

Katelyn Arnold

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

Heparin-Like Carbohydrates for the Treatment of Acute Liver Failure

Heparin products are standard therapeutics for the prevention and treatment of coagulation disorders, yet they have various other unexploited biological roles. While the mechanism of acute liver failure (ALF) is unclear, patients may benefit from treatment with heparin or heparin-like carbohydrates. Using an APAP-overdose mouse model, heparin compounds will be used to probe the mechanism of ALF.


Kimberly Barnash

Ph.D. Candidate
Center for Integrative Chemical Biology and Drug Discovery

Development of MPP8 Inhibitors for HIV Latency Reversal

Current treatments for human immunodeficiency virus permit viral persistence, requiring lifelong medication. HIV latency contributes to maintaining this HIV reservoir that prevents eradication of the disease. We will develop a chemical probe to disrupt essential epigenetic processes of the latent viral state, thereby validating epigenetic intervention as a treatment strategy against HIV.


Laura Bowers

Pharm.D. Student

The Air You Breathe Campaign: A Targeted and Innovative Approach to Promoting Treatment Guidelines and Educating Practitioners

Lymphangioleiomyomatosis (LAM) is a rare disease affecting primarily women of childbearing age. An estimated 250,000 women are currently living with this disease and are undiagnosed, misdiagnosed and untreated. The Air You Breathe Campaign is a practice model to promote the new LAM healthcare guidelines, educate clinicians, raise awareness and positively impact patient outcomes.


Stephen Capuzzi

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

Accelerating the Discovery of Protein-Protein Interaction Stabilizers

Protein-protein interactions (PPI) significantly outnumber single proteins – the traditional targets of drug discovery efforts. Thus, modulation of PPIs would vastly enlarge the druggable genome. Small-molecule stabilization of PPIs is an emerging but under-studied concept in drug discovery. We propose to develop in silico methodologies to accelerate the discovery of PPI stabilizers.


Olexandr Isayev

Postdoctoral Research Associate
Division of Chemical Biology and Medicinal Chemistry

Accurate Property Prediction for Drug Polymorphs

Current cheminformatics QSAR/QSPR models do not take polymorphs into account. In this project, we propose a new method for predicting compound properties that explicitly accounts for drug crystal structure. To achieve this objective, we combine accurate ab initio quantum-chemical calculation of crystals and co-crystals with machine learning to build a predictive model of drug properties.


Felix Lam

Pharm.D. Student

Comparison of Induction of Vaginal Antibody Response Following Intranasal vs. Intramuscular Influenza Vaccination

Despite the success of the HPV vaccine, there is currently no approved vaccine for other common STIs in the female genital tract. We believe our human clinical study that directly compares vaginal antibody response following intranasal vs. intramuscular influenza vaccination will provide valuable insights that will guide design of future vaccines against vaginally transmitted infections.


Andrew Lucas

Postoctoral Research Associate
Division of Pharmacotherapy and Experimental Therapeutics

Phenotypic Probe to Individualize the Treatment of Monoclonal Antibodies and Antibody Drug Conjugates

Compared to small-molecule drugs, monoclonal antibodies and antibody-drug conjugates are cleared via the mononuclear phagocyte system (MPS). Variability in MPS function may be responsible for the clinically relevant variability in the PK and PD of these agents. Thus, there is a need to develop methods, such as phenotypic probes of the MPS, that can be used to individualize the dose of these agents.


Manish Singh

Postdoctoral Research Associate
Division of Chemical Biology and Medicinal Chemistry

A Facile Approach Towards Cell-Permeable and Photoactivatable Phospholipids

Phospholipids are essential signaling molecules that regulate a wide range of cellular processes. Dysregulation of phospholipid metabolism has been implicated in various diseases. Lack of cell-permeability and rapid, dynamic metabolism presents a challenging task for phospholipid studies. We propose a simple and novel methodology to enable phospholipids that are cell-permeable and photoactivatable.


Jimmy Xu

Pharm.D. Student

Improving Patient Retention in Clinical Trials Through the Use of Wearable Devices

Clinical drug trials primarily rely on returned pill counts and e-diaries to assess medication compliance; however, these methods have been shown to be inaccurate and biased. Our revolutionary platform utilizes wearable devices to continuously monitor dosing events in real time. Early detection and intervention empower investigators to employ behavioral modification to improve patient retention.

TIER 1: Up to a Total of $50,000

Cao Carter

Division of Pharmacoengineering and Molecular Pharmaceutics

A Companion Diagnostics for Checkpoint Immunotherapy: Beyond Static Biomarkers

A companion diagnostics we are developing in this project is based on the dynamic properties of cytokine secretion by peripheral T-cells.This diagnostics would fundamentally change current time-frozen static diagnostic approaches to more dynamic characterization of immune functions and its potential responsive status to checkpoint immunotherapy.


Shawn Hingtgen

Division of Pharmacoengineering and Molecular Pharmaceutics

T-STEM: A New Approach to Cancer Therapy Through Cellular Hybrids

Cell therapies are changing the face of cancer treatment. We propose to create a cellular hybrid designed to overcome hurdles that limit effectiveness of current cell therapies. This highly novel approach promises a cell that migrates through solid tissue and co-opts the immune system for unparalleled tumor killing.


Michael Jarstfer

Division of Chemical Biology and Medicinal Chemistry

Rescuing Rare Genetic Disorders by Kinase Inhibition

Many genetic diseases result from mutations called premature termination codons, which result in expression of truncated and dysfunctional protein. In several diseases, such as cystic fibrosis and Duchenne muscular dystrophy, ~10 % of cases are caused by premature termination codons. This project will establish if the function of genes with premature termination codons can be rescued by stabilizing the mutated RNAs. If successful, this would provide a novel approach to treat genetic disorders.


Dmirtri Kireev

Division of Chemical Biology and Medicinal Chemistry

Taming Entropic Pseudo-Forces in Chemical Biology and Drug Discovery

We intend to provide medicinal chemists with new tools for a more effective drug design on challenging protein targets. Our goal is to devise an approach to design more potent and specific leads by harnessing the ligand-protein binding entropy. This goal will be achieved through an innovative blend of high-performance computing (HPC) and machine learning.


Samantha Pattenden

Division of Chemical Biology and Medicinal Chemistry

Developing Small Molecule Probes to Inhibit the Regulatory Capacity of Long Noncoding RNAs

Molecules called long noncoding RNAs (lncRNAs) finely tune the expression of genes essential for normal growth and development. Aberrant lncRNA activity drives many genetic disorders and cancers, but, currently, no approved therapies exist to target lncRNA function. We developed an assay to track lncRNA regulatory activity that we will apply to find inhibitors of two disease-causing lncRNAs.


Chunping Qiao

Division of Pharmacoengineering and Molecular Pharmaceutics

Improving AAV Production Method with CRISPR/Cas9

We will utilize the state of the art CRISPR/Cas9 genome editing technology to create a high titer adeno-associated virus (AAV) producer cell line via targeted delivery. With the successful completion of this project, our producer cell line method will revolutionize the AAV production process, generating an innovative AAV producer cell line to meet the rising demands of AAV preclinical/clinical studies.

TIER 2: Up to a Total of $200,000

Dmitri Kireev

Division of Chemical Biology and Medicinal Chemistry

Cracking the Key to Pluripotency through Molecular Biosystems Computing

The goal of the project is to propose and characterize a dynamic structural model of the OCT4-mediated pluripotency repression. It will be achieved through a multidisciplinary strategy, combining novel Molecular BioSystems (MB) approach with a unique Chromatin in-vivo Assay, to address a challenging question in the field of regenerative biology. Activities will include MB simulations, software development and experimental testing through real-time chromatin imaging.


Paul Watkins

Division of Pharmacotherapy and Experimental Therapeutics

An Exosome-Based Assay for Predicting Idiosyncratic Drug-Induced Liver Injury

It is not currently possible to identify drugs that cause very rare but life-threatening liver injury. As a result, the FDA may require large and long clinical trials just to assure liver safety. The objective of this project is to develop a new test of liver safety that capitalizes on our recent discoveries. If successful, this test will make new drugs available faster, more affordable, and safer.


Jeffrey Aubé

Division of Chemical Biology and Medicinal Chemistry

beta-Lactams: New Generations and New Uses

b-Lactams, such as penicillin, are among the most successful drug classes of all time. Although useful in nearly every arena of infectious disease treatment, but with one especially important exception – tuberculosis. This program supports and extends a recent discovery that a modified class of cephalosporins are active in test tube models of TB. The Aubé lab seeks to build upon this by inventing and testing several entirely new classes of b-lactams.


Kevin Frankowski

Division of Chemical Biology and Medicinal Chemistry

Inspired by Nature: Enhancing the UNC Compound Collection

Borrowing inspiration from the privileged structural motifs found in natural products and utilizing nontraditional synthetic methods for library synthesis, we aim to assemble a proprietary screening compound resource for the UNC research community. The compound set will contain natural product-inspired analogues and fragments to mirror the interactions of natural products with biological targets. Screening this diverse collection will facilitate the discovery of small molecule modulators for a broad range of novel therapeutic targets.


Shawn Hingtgen

Division of Pharmacoengineering and Molecular Pharmaceutics

Personalized Therapy for the Incurable: Metastatic Lung Cancer

Metastatic lung cancer remains one of the deadliest types of cancer. We propose a radical new therapy, where tumor-homing cell therapies target and eradicate cancer foci. This novel approach promises a powerful anti-tumor weapon for clinicians and hope for patients.


Rihe Liu

Division of Chemical Biology and Medicinal Chemistry

Biepitopic and Bispecific Chimeric Antigen Receptors for T-Cell Therapy

Chimeric Antigen Receptor (CAR)-T cell therapy is a cell-based treatment that uses engineered T cells to recognize and drastically kill cancer cells. This project is directed to address the urgent unmet need in cancer immunotherapy by developing the next generation CAR-T cells in which the CAR is capable of targeting two epitopes or two antigens with significantly reduced antigen escape.


Jacqueline McLaughlin

Division of Practice Advancement and Clinical Education

Precision Education for Clinical Decision Making

In health professions education, clinical teaching cases are critical in promoting the sophisticated clinical reasoning and decision-making skills required for success in contemporary practice. However, current methods for generating appropriately complex and relevant clinical cases – and the related questions required to assess student learning – are time- and labor-intensive. We aim to develop a dynamic, innovative system that efficiently generates precisely tailored clinical cases on demand. The proposed system will apply cutting-edge technologies to provide health sciences faculty with custom-designed just-in-time instructional and assessment resources.


Stephen Frye and Kenneth Pearce

Division of Chemical Biology and Medicinal Chemistry

Development of DNA-encoded Chemical Library Technology for Enhancement of CICBDD High Throughput Screening Capabilities

Traditional high throughput screening tests small molecules (thousands to millions) in individual wells using a biochemical or cellular assay.  We propose establishing a transformational technology in the CICBDD to utilize DNA-encoded libraries (DELs) where diverse compounds (potentially >100 million) are selected as hits in binding assays versus targets of interest. Initial DELs will be designed using our unique insights into chromatin regulatory domains that modulate gene expression during development and disease.

TIER 3: Up to a Total of $750,000

Nate Hathaway

Division of Chemical Biology and Medicinal Chemistry

Center for Allele Specific Epigenetic Regulation (CASR)

The Center for Allele Specific Regulation (CASR) is a group formed between the divisions of CBMC and DPET. We will create innovative chemically based gene control systems and apply these tools to repress key oncogenic targets in incurable metastatic human prostate cancer. The CASR will create a new hub at the Eshelman School of Pharmacy to bring our promising new platform to labs across UNC.


Alexander Kabanov

Division of Pharmacoengineering and Molecular Pharmaceutics

Systemic Targeting of Mononuclear Phagocytes for Parkinson’s Disease Gene Therapy

We propose a novel gene therapy approach in which a therapeutic gene is first, systemically delivered to blood-borne immune cells and then, carried by these cells to the disease site. This approach allows for non-invasive, safe and efficient access to most secluded sites in the brain, where the therapeutic molecules are produced to treat Parkinson’s disease. Such therapies could improve lives of Parkinson’s and other patients suffering from incurable brain diseases.


David Lawrence

Division of Chemical Biology and Medicinal Chemistry

Optogenetic Manipulation of Striatal Neurons

Light-activatable ion channels or pumps have been encoded into neurons in order to map neural pathways. However, this technology is unable to probe the relationship between intracellular biochemistry and neuronal/animal behavior. We’ve developed a strategy to photo-activate specific proteins within specific compartments of individual neurons. The ability to control compartmentalized signaling in the brain will be used to assess the basis of addictive behavior and dysfunctional motor control.


Tim Wiltshire

Division of Pharmacotherapy and Experimental Therapeutics

Pharmacogenetic Implementation: The Actionable Genome

The goal of this proposal is to demonstrate that a preemptive pharmacogenetic testing program is cost-effective, and improve potential for prescribing of the right drug or right dose of drug. This will ultimately lead to direct improved health outcomes for patients. The comprehensive pharmacogenetic test used, DNA2Rx TM, has been developed at UNC, and covers all of the actionable gene/drug combinations. It will be implemented in cardiology, oncology, pain, transplant and psychiatry clinics.


Xiao Xiao

Division of Pharmacoengineering and Molecular Pharmaceutics

Targetable and Regulatable Gene Delivery Platform Technology for Gene Therapy

Our proposal aims to make key contributions to the next generation gene therapy technology, a platform that enables the creation of novel targetable, regulatable and invisible gene vectors. We will use forced molecular evolution and structural rational design to engineer novel adeno-associated virus (AAV) vectors for the treatment of human diseases.

Up to a Total of $25,000

Kayley Lyons

Ph.D. Candidate
Division of Practice Advancement and Clinical Education
Collabucate: Your Team’s Personal Collaboration Coach

In classrooms, professors assign group work, but simply placing students in groups does not guarantee students will learn to work as a team and it may even produce opposite effects. Collabucate is a mobile application which supports optimal student collaboration by fostering social awareness and providing teams with personalized group learning techniques.


Courtney Schaal

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

Optimization of a Novel, Two-Step High Throughput Method to Assess Chromatin for Screening of Compounds that Alter Chromatin Accessibility in Cancer

Chromatin modulation has gained increasing attention for therapeutic efforts as its importance in cancer and other disease continues to be unveiled; however, no high-throughput methods to interrogate chromatin exist. This project will deliver a first-in-class, high-throughput assay for chromatin-focused drug discovery. Its application will be used to screen large compound libraries to identify “hit” compounds with therapeutic potential, it will be readily transferable to other labs, and can be applied to study basic chromatin biology.


Sherif Farag

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

Re-Engineering Bacteria with NRPLinker: A Computational Approach to Designing Novel Non-Ribosomal Peptide Antibiotics

As the threat of antibiotic resistance continues to rise, the need of novel antibiotics is greater than ever. We propose the development of “NRPS-Linker” the first computational tool to guide rational design of novel NRP antibiotics. The tool explores the importance of Inter-Modular Linkers (IMLs) and their key role in coordination between modules within a non-ribosomal peptide synthetase (NRPS). NRPS-Linker will be a valuable asset for researchers interested in combinatorial biosynthesis of novel NRPs.


Jonathan Bogart

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

A Chemoezymatic Platform for the Discovery of Bicycle Thiopeptide Therapeutics

Antibiotic resistance/infectious diseases are constant threats to global public health. Thiopeptides are an emerging family of macrocyclic natural products that represent a huge, untapped potential for new antibiotic scaffolds. These scaffolds remain elusive due to their structural complexity and the few, limited strategies used to access them. I propose to use a chemoenzymatic approach to access new bicyclic thiopeptide analogs and develop more clinically relevant bicyclic thiopeptide therapeutics.


jingjing_li

Jingjing Li

Ph.D. Candidate
Division of Chemical Biology and Medicinal Chemistry

Screening of PD-1-Binding Epitopes from a PD-L1 Library for the Development of Novel PD-1 Inhibitors and Vaccines

PD-1 and PD-L1 targeting antibody-based immunotherapy that is extensively used in clinical anti-cancer therapy is costly and tends to cause toxicities and resistance. In this proposal, protein engineering combined with high throughput protein display technology will be adopted to develop peptide based vaccines to stimulate active immune response and inhibitors to block PD-1/PD-L1 signaling, which hopefully address aforementioned problems.