Gemma Therapeutics · United States

Genetic Medicine Platforms for Rare Disease

University of Southern Denmark · Denmark

Overcoming TIL limitations via epigenetic therapy

Einstein Hospital Israelita · Brazil

Establishing an Advanced Cell Therapy Platform for TIL-Based Immunotherapy

UFMG · Brazil

mRNA vaccines for Chagas disease

Einstein Hospital Israelita · Brazil

Expanding the reach of mRNA-LNP therapeutics: Cancer vaccines and beyond

United States

Artificial Intelligence in the Design of Personalized Therapies and Vaccines

Gemma Therapeutics · United States

Gene Therapy in Developing Countries: Challenges and Prospects

Fiocruz-BA · Brazil

Immunomodulation by genetically engineered mesenchymal stromal cells

UNICAMP · Brazil

The Molecular Architecture of Epilepsy: Integrating Genomics, Multi-omics and Single-Cell Technologies

Instituto Leloir · Argentina

Adenovirus-Based Gene Therapy for Cancer: From Targeted Delivery to Antitumor Immunity

CNPEM · Brazil

Engineering Next-Generation Cancer Immunotherapies: From Gene Delivery to Immunomodulatory Nanoplatforms

Oral presentation · Abstract 80 · Brazil

Neurogenin 2 overexpression in corticospinal neurons modulates early neuronal and microglial responses after spinal cord injury

Oral presentation · Abstract 153 · Brazil

AAV-mediated overexpression of cannabinoid CB1 receptors preserves functional responses in a mouse model of retinitis pigmentosa: Perspectives for a nationally developed gene therapy in a rare disease

INCA-RJ · Brazil

Modeling Leukemic Transformation In Vitro Using iPSCs

USP · Brazil

Decoding Neuroviral Impact on Brain Development Using Human iPSC-Derived Organoids

Oral presentation · Abstract 37 · Brazil

Bioengineering a human lung-on-a-chip platform toward immune-informed preclinical modeling of pulmonary advanced therapies

Oral presentation · Abstract 66 · Brazil

Development and Validation of a CAPN1 Knockout SH-SY5Y Cell Model via CRISPR-Cas9 for SPG76 Studies

FMRP-USP · Brazil

Academic CAR-T in Clinical Practice: Implementation, Challenges, and Real-World Outcomes in Ribeirão Preto

FMRP-USP · Brazil

CAR-T Beyond Cancer: Immune Reset Strategies for Autoimmune Diseases

FMRP-USP · Brazil

CD33-Targeted Immunotherapies in AML: Engineering CAR-T and CAR-NK Strategies

UFRJ · Brazil

Bioengineering of diseased livers: lessons for liver transplantation

FCF-USP · Brazil

Engineering Human Skin Models: From Preclinical Disease Modelling to Personalised Melanoma Immunotherapy

Oral presentation · Abstract 19 · Brazil

Experimental evolution of Zika virus for adaptation in differentiated cells of glioblastoma to enhance oncolytic activity

Oral presentation · Abstract 24 · Brazil

Air-Jet-Generated Alginate Microgels Enable Controlled and Functional Release of AAV Vectors for Gene Therapy Applications

St. Jude Children's Research Hospital · United States

Base editor RNPs for fetal hemoglobin induction: preclinical development of SAGES2 for sickle cell disease

CMRI / Gene2Cure · Australia

AAV-based Gene Therapy Revolution: How did we get here and what is next?

CNPEM · Brazil

From Disease Models to Precision Gene Therapy: CRISPR and Prime Editing in Mucopolysaccharidosis Type I (Hurler Syndrome)

Einstein Hospital Israelita · Brazil

Toward Safer Base Editors: Rewiring the Deaminase-Cas Architecture

Oral presentation · Abstract 107 · Brazil

Sustained reactivation of fetal hemoglobin by adenine base editors in human hematopoietic stem cells for sickle cell disease

Oral presentation · Abstract 28 · Brazil

Gene Therapy with Adeno-Associated Viral Vector Serotype 9 in Cellular Models for Glutaric Acidemia Type I Developed via CRISPR/Cas9 Gene Editing

HCPA-UFRGS · Brazil

NK cell adoptive Immunotherapy for Acute Myeloid Leukemia

Einstein Hospital Israelita · Brazil

From Research to GMP: The role of a Cleanroom Facility in Advanced Therapy Development

Oral presentation · Abstract 88 · Brazil

Lentiviral Vector Platform based on Packaging Cell Line Engineering and GMP-Oriented Process Development

Oral presentation · Abstract 76 · Brazil

Establishing GMP-Compliant CAR-T Cell Manufacturing at INCA: Infrastructure Adaptation and Manufacturing Readiness for an Upcoming Pediatric Clinical Trial in Brazil

University of Antwerp · Belgium

Delivering Regeneration: Non-Viral Gene Transfer Across Immunomodulatory and Regenerative Strategies

Biomanguinhos · Brazil

Nanoparticles for Advanced Therapy Delivery: Towards In Vivo CAR-T Cell Generation

Oral presentation · Abstract 170 · Brazil

Optimization and Preclinical Validation of an mRNA-LNP Vaccine Candidate Against Chikungunya Virus

Oral presentation · Abstract 57 · Brazil

Scaling up the non-viral production of CD19 CAR-T cells using a minicircle-based Sleeping Beauty transposon system.

ANVISA · Brazil

Advancing ATMP Regulation in Brazil: Innovation, Challenges and Opportunities

ARO-EINSTEIN · Brazil

Clinical Research Regulatory Landscape: Recent Updates

MD Anderson Cancer Center · United States

The Emerging Field of Viroimmunotherapy for Pediatric Brain Tumors

MD Anderson Cancer Center · United States

From Glioma Cartography to Clinic: Mapping the Tumor Ecosystem to Predict Survival after Oncolytic Virus Therapy

UNICAMP · Brazil

Nanotechnological Platforms in Immuno-Oncology: Innovation and Therapeutic Impact

Einstein Hospital Israelita · Brazil

The Brazilian Experience in Academic CAR-T Cell Manufacturing: Operational, Regulatory, and Clinical Challenges

Einstein Hospital Israelita · Brazil

Innovation Ecosystems in Advanced Therapies: Partnerships, Local Manufacturing, and Competitiveness

Einstein Hospital Israelita · Brazil

The New Innovation Triad: Industry, Academia, and Patients

UNESP · Araraquara · Brazil

CAR application for the treatment of invasive fungal infections

Instituto D'Or · Brazil

Generation of human CD34+ derived-macrophages expressing CARs for solid tumors control.

Oral presentation · Abstract 14 · Brazil

A Lentiviral EDV Platform for simultaneous CAR Engineering and CRISPR Gene Editing in Primary T and NK Cells

Oral presentation · Abstract 79 · Brazil

Cell therapy targeting fungal pathogens: optimization of Mannan-Specific Chimeric Antigen Receptor (M-CAR)

UnB · Brazil

RNA-Based Therapeutics for Genetic Neurological Diseases: Promises, Challenges, and Future Directions

Fiocruz · Brazil

Network-Guided RNA Therapeutics: Using siRNA to Validate Novel Targets for Precision Oncology

Oral presentation · Abstract 42 · Brazil

Isoform-resolved lncRNA remodeling as a secondary readout of SCN2A dosage and CRISPRa-mediated activation in human iPSC-derived cortical excitatory neurons

Oral presentation · Abstract 34 · Brazil

From cells to patients: gene therapy with p14ARF and interferon-β reveals antitumor and immunomodulatory mechanisms in canine neoplasms

University of Vermont · United States

MSCs: Why Aren't they Working in Clinical Trials

UFRJ · Brazil

Beyond Stem Cells: Extracellular Vesicles as a Novel Therapeutic Strategy for ARDS

Innovative Genomics Institute · United States

Peptide-enabled ribonucleoprotein delivery for CRISPR engineering (PERC) in primary human immune cells and hematopoietic stem cells

Instituto D'Or · Brazil

Human Brain Organoids at the Crossroads of Viruses, Psychedelics, and Artificial Intelligence

UFRJ · Brazil

Extracellular vesicles derived from MSCs for Lung Diseases - Preclinical Evidences

Instituto D'Or · Brazil

When the Secretome Becomes the Product: Translating MSC-Derived Extracellular Vesicles into an ARDS Clinical Trial

Oral presentation · Abstract 59 · Brazil

Investigation of the neuroprotective role of extracellular vesicles from physical exercise and associated with FNDC5/irisin in preclinical models of Alzheimer's disease

Oral presentation · Abstract 104 · Brazil

3D Bioprinted Wound Dressings Containing 5-Azacytidine-Preconditioned Human Umbilical Cord Mesenchymal Stromal Cells for the Treatment of Severe Cutaneous Wounds in a Murine Model of Sickle Cell Disease

UFRJ · Brazil

Agnostic Gene Therapy

UFRGS · Brazil

Gene therapy for inborn errors of metabolism

Oral presentation · Abstract 17 · Brazil

Construction and evaluation of an HSV-1-based oncolytic virus expressing the pro-apoptotic protein sfTRAIL, for the treatment of head and neck tumors.

Oral presentation · Abstract 146 · Brazil

Therapeutic DNA Vaccination Targeting HPV16 E5 and E7 Oncoproteins: Evaluation of Different Immunization Regimens in a Preclinical Tumor Model

University of Florida · United States

Need for the development of capsid- and genome-modified next generation of AAV vectors

France

From Capsid Development to the Clinic

CHOP · United States

Transferring Point-of-Care CAR-T Manufacturing Between Institutions: What Travels and What Has to Be Rebuilt

University College London · United Kingdom

From Concept to Clinic: Challenges in the Translation of Next-Generation CAR T Cell Therapies

Oral presentation · Abstract 142 · Brazil

Phage display selection of scFvs specific for the HER2 S310F mutation from rationally designed libraries based on pertuzumab

Oral presentation · Abstract 6 · Brazil

Engineering CAR-T Cells to Enhance Tumor Infiltration in Solid Tumors

Escola Paulista de Medicina · Brazil

Advanced Therapies in Ophthalmology

UFRGS · Brazil

Gene Therapies in Practice: From Clinical Care to Early-Phase Clinical Trials

Oral presentation · Abstract 187 · Brazil

Will It Reach Patients? An AI Model Predicts Clinical Translation of Gene Therapies from Modifiable Preclinical Design Choices

Oral presentation · Abstract 198 · Brazil

Safety and Preliminary Efficacy of a Brazil-Developed CMV pp65-Specific T-Cell Therapy for Clinically Significant CMV Infection After Allogeneic Hematopoietic Stem Cell Transplantation: Interim Results of a Phase I Study

PUC-RJ · Brazil

Rational Design of Inhibitors and Silencers of Cancer-Associated Genes Using Artificial Intelligence

UFSC · Brazil

What are the primary obstacles to achieving tissue- or tumor-selective gene therapy?

UFMG · Brazil

Nanoparticle-based cancer immunotherapy for nucleic acid delivery

USP · Brazil

Flow-Driven Regulation of Endothelial Morphology and Tumor Extracellular Vesicle Dynamics

Brazil

Organ-on-a-Chip Platforms: New Frontiers in CNS Disease Modeling

UNICAMP · Brazil

Use of 3D cellular models to study the dysregulation of chemotactic signaling and neuronal migration in autism