UPENN_ITMAT

Identifying ROR2 (receptor tyrosine kinase-like orphan receptor 2) as promising #DrugTarget to treat #RightVentricularFailure doi.org/10.1161/CIRC... #RVfailure #HeartFailure #Proteostasis #TranslationalScience

Armoring #CARTcelltherapy with anti-inflammatory molecules offers new avenues for anti-fibrotic therapies in #ChronicKidneyDisease & other #Fibrosis conditions www.jci.org/articles/vie... @JonEpsteinLab #CellTherapy #CARTCell #TranslationalScience

#Chronotherapy in Translation doi.org/10.1146/annu... @elronan.bsky.social @FitzGerald__Lab #CircadianRhythms #DrugResponse #DrugMetabolism #PrecisionMedicine #Pharmacology #TranslationalScience

#MyeloproliferativeNeoplasms: Unraveling mechanics of contradictory coexistence of #Thrombosis & #Hemorrhage doi.org/10.1126/scit... #JAK2V617F #MPN #TherapeuticOpportunities #TargetedTherapies #TranslationalScience

#Microenvironment #Remodeling in #RightVentricularFailure suggest #antifibrotic & #immunomodulatory approaches & #MitochondrialFunction support as plausible #RightVentricular #TherapeuticOpportunities doi.org/10.1038/s441... @AranyLab

Clock-Dependent Organoid Maturation by Daily Metabolic Cycling by Juan Alvarez, PhD, Assistant Professor of Cell and Developmental Biology @juanalvarezdom.bsky.social @PennMedicine #ITMATSymposium2026 #TranslationalScience

Reception and Dinner: The College of Physicians of Philadelphia with Dinner Discussant: Norman J. Ornstein Retired Scholar, American Enterprise Institute @NormOrnstein @PennMedicine #ITMATSymposium2026 #TranslationalScience

An (un)easy Marriage: Academic-Biotech Partnerships in Translational Research by Saar Gill, MD, PhD, Scientific Co-Director, Cell Therapy and Transplant Program, Professor of Medicine @PennMedicine #ITMATSymposium2026 #TranslationalScience

Outsmarting the Escape Artist: Resistance to CART Immunotherapies for Lymphoid Malignancies by Marco Ruella, MD, Associate Professor of Medicine @MarcoRuella @PennMedicine #ITMATSymposium2026 #TranslationalScience

The Journey of CAR T Cells from HIV to Cancer to Autoimmunity by Carl June, MD, Richard W. Vague Professor in Immunotherapy @carlhjune @PennMedicine #ITMATSymposium2026 #TranslationalScience

From n=1 to n-many: Developing Scalable Genetic Medicines for Rare Disease by James Dowling, MD, PhD, Director, Penn Neurogenetics Therapy Center, Ruth Wagner Van Meter & J. Ray Van Meter Professor of Neurology @PennMedicine #ITMATSymposium2026 #TranslationalScience

Emerging Neurodegenerative Disease Therapies: Targeting Proteinopathies by Edward B. Lee, MD, PhD, John Q. Trojanowski, MD, PhD Professor for Neurodegenerative Disease Research @PennMedicine #ITMATSymposium2026 #TranslationalScience

Islet Cell Sources for Transplantation in Type 1 Diabetes by Michael R. Rickels, MD, MS, Willard and Rhoda Ware Professor in Diabetes & Metabolic Diseases @PennMedicine #ITMATSymposium2026 #TranslationalScience

Alchemy for this Moment: Turning Challenges into Opportunities by Nancy Andrews, MD, PhD, EVP, CSO, Helen M. Ranney, M.D. Chair, Boston Children’s Hospital, Professor in Residence of Pediatrics, Harvard Medical School @PennMedicine #ITMATSymposium2026

Turning Genes into Medicines by Katherine High, MD, CEO, RhyGaze; Visiting Professor, Rockefeller University; Professor Emeritus, Perelman School of Medicine of the University of Pennsylvania; Founding Director, Center for Cellular and Molecular Therapeutics, Children’s Hospital of

From Fantasy to Reality: Harnessing Genes to Reverse Blindness by Jean Bennett, MD, PhD, F.M. Kirby Professor Emerita, Ophthalmology @PennMedicine #ITMATSymposium2026 #TranslationalScience

mRNA CAR T Cells to Treat Fibrosis by Jonathan Epstein, MD, Dean, Perelman School of Medicine, Executive Vice President @JonEpsteinLab @PennMedicine #ITMATSymposium2026 #TranslationalScience

Advancing Genome Editing Therapies for Rare Diseases by Rebecca Ahrens-Nicklas, MD, PhD, Associate Professor of Pediatrics@@AhrensNicklas @PennMedicine #ITMATSymposium2026 #TranslationalScience

Nucleoside-Modified mRNA-LNP Therapeutics by Drew Weissman, MD, PhD, Roberts Family Professor in Vaccine Research @WeissmanLab @PennMedicine #ITMATSymposium2026 #TranslationalScience

Welcome address by Jonathan Epstein, MD, Dean, Perelman School of Medicine, Executive Vice President @JonEpsteinLab @PennMedicine #ITMATSymposium2026 #TranslationalScience

Welcome address by Garret A. FitzGerald MD, FRS, Director, ITMAT @FitzGerald__Lab @PennMedicine #ITMATSymposium2026 #TranslationalScience

Welcome to the ITMAT 21st Annual International Symposium "Intermezzo; 20 Years of Translational Therapeutics at PENN and CHOP"

Development of a #Methylation risk score for #AbdominalAorticAneurysm in the #MillionVeteranProgram doi.org/10.1161/CIRC... @DeptVetAffairs @damrauer @bvoight28.bsky.social #Epigenetics #Biomarkers #InflammatoryPathways #TranslationalScience

Establishing #Fruitfly model to discern #MolecularMechanisms of #Sleep & #CircadianClock related #MetabolicDysfunction for developing #TherapeuticOpportunities elifesciences.org/articles/108... @weljielab #Metabolomics #TranslationalScience

#TherapeuticPotential of combining #IRE1α and #KRAS inhibition in #PancreaticDuctalAdenocarcinoma doi.org/10.1073/pnas... #PDAC #Hypoxia #Oncology #LiverCancer #SolidTumor #TranslationalScience

Importance of a life course approach to #Prevention and intervention that addresses #RiskFactors for #FunctionalImpairment in younger individuals and those in early middle age doi.org/10.1371/jour... #HealthyAging #PreventiveHealth #TranslationalScience

Improving the safety and efficiency of adenine base-editing therapies www.nature.com/articles/s41... @kiranmusunuru.bsky.social @ahrensnicklas.bsky.social #CRISPR #Phenylketonuria #PseudoxanthomaElasticum #HereditaryTyrosinemia

Characterizing #Inflammation induced #Metabolomics changes in mouse models of #ImmuneActivation and in tissue culture #Macrophages doi.org/10.1016/j.jb... @Caroline_Bartma @ejohnwherry.bsky.social #ImmuneResponse #TranslationalScience

Building an understanding on the physical & chemical roles of membranes to bind #Coagulation proteins & to affect their function doi.org/10.1073/pnas... #FactorV #FactorVIII #StructuralBiology #MembraneBiology #CryoEM #TranslationalScience

Single #lncRNA integrates #Inflammatory and #Metabolic signals to regulate #CardiovascularHomeostasis doi.org/10.1016/j.ce... @FitzGerald__Lab #PACERR #GeneRegulation #Cyclooxygenase #TranslationalScience

Elucidating pharmacological targets of Sodium-glucose cotransporter 2 inhibitors (SG LT2i) www.science.org/doi/10.1126/... @AranyLab #HeartFailure #SGLT2 #CardiacMetabolism #TranslationalScience

#SpatialNT-Seq to spatially co-map newly synthesized and pre-existing #RNA in intact tissues www.nature.com/articles/s41... @hao_wu_7 #SpatialTranscriptomics #RNAturnover #SpatialBiology #TranslationalScience

Dissecting mechanistic leads to map #Craniofacial & #Cardiovascular defects in #CongenitalDisorders including #Kleefstra syndrome doi.org/10.1172/JCI2... @JonEpsteinLab #RareDisease #TranslationalScience

Novel #MouseModel allows study of #Senolytic mechanisms linked to specific cell types doi.org/10.1016/j.jc... @kaestner_klaus #LiverDisease #AgingResearch #CellularSenescence #TranslationalScience

Defining therapeutic options to target advanced disease refractory to conventional #CARTCell therapy through nonactivated #CARTCell as distinct cell biological state doi.org/10.1182/bloo... @carlhjune #CARTcelltherapy #CellTherapy #TranslationalScience

Characterizing #Immunoregulatory roles of #Itch inducing neurons in #Psoriasis doi.org/10.1016/j.ji... #Pruritus #GammaDeltaTCells #Neuroimmunology #Immunology #SensoryNeurons #TranslationalScience

Towards safer #Therapeutics: Structured semantic data enhances #ToxicityPrediction of molecule substructures doi.org/10.1016/j.is... @JDRomano2 #MachineLearning #SemanticModel #TranslationalScience

Magnitude of time-of-day differences in #GeneExpression vary considerably between individuals journals.sagepub.com/doi/10.1177/... #Circadian #BiologicalRhythm #TranslationalScience

#RetinalAge emerges as #Biomarker for #EarlyDetection of #CognitiveDecline doi.org/10.1016/j.xo... #BiologicalAge #DeepLearning #TranslationalScience

Foundation to understand #BiologicalMechanism of #CentromereEvolution and its implications for #Chromosome segregation fidelity and #HumanDisease doi.org/10.1038/s415... @glennislogsdon.bsky.social #Centromere #TranslationalScience

Plasticity in #CellCellCommunication may be central for how cell fate is robustly established in complex patterned tissues doi.org/10.1038/s415... @ksusztak.bsky.social #SingleCellRNASequencing #SpatialTranscriptomics #KidneyDevelopment

Telomere length within normal human range represents critical determinant of hematopoietic reserve capacity under proliferative stress doi.org/10.1016/j.jb... #TelomereBiologyDisorders #DyskeratosisCongenita #HoyeraalHreidarssonSyndrome

Uncovering new #TherapeuticOpportunities to reduce #Dyslipidemia and susceptibility to #LiverDisease by studying variants of glucokinase regulatory protein doi.org/10.1016/j.mo... @PaulTitchenell #GKRP #CholesterolMetabolism #TranslationalScience

WHERE ART AND SCIENCE MEET: community still life + student showcase. ITMAT Education invites you to draw, observe and reflect this Thursday from 5-7 PM at the Institute of Contemporary Art, RSVP required, virtual option available, details on flyer

Neither #Microbiome nor LPS-TLR4 #Signaling is required for #CerebralCavernousMalformations formation in the adult #MouseModel insight.jci.org/articles/vie... #Brain #VascularMalformation #NullResult #TranslationalScience

Disentangling the role of #CircadianClocks in #Tanycytes with implications for sex-specific modulation of #BodyComposition and #Neurogenesis doi.org/10.1016/j.ce... @amitaseh @chronosleepinst #SexualDimorphism #Neuroscience #TranslationalScience

Structured #Workforce feedback through embedded well-being consultants reduces lag between problem identification & solution implementation in #ClinicalCare catalyst.nejm.org/doi/10.1056/... #HealthSystemInnovation #BurnoutPrevention #TranslationalScience

Finetuning #Pharmacotherapy options for managing #Hypertension in #HeartFailure With #PreservedEjectionFraction

Happy 4th of July and 250th Anniversary!

Prospective longitudinal cohort study of #BreastCancer patients determines potential #MolecularBiomarkers for #Cancer therapy-related #CardioToxicity doi.org/10.1093/eurh... #CardioOncology #CardioOnc #TranslationalS