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Keystone Symposia

Neurodevelopmental disorders (NDDs), including autism, intellectual disability, and epilepsy, arise from disruptions during brain development and pose major challenges to public health. Although advances in genetics have identified hundreds of causal genetic risk factors, identifying convergent mechanisms underlying their pathobiology remains a challenge, which hinders the development of personalized therapies. This meeting addresses the critical gap between emerging insights into causal factors, neurodevelopment, and therapeutic innovation by uniting experts across basic and translational neuroscience. This conference will foster new collaborations between investigators in genetics, functional genomics, developmental neurobiology, and industry. Specific aims include showcasing breakthroughs in single-cell genomics, genome perturbation technologies, gene-environment interactions, and novel therapeutic strategies such as gene replacement therapies and ASOs. Unlike existing meetings that focus separately on neurodevelopment, stem cell models, or therapeutic pipelines, this conference uniquely fuses mechanistic understanding of genetic and environmental causes with translational application. This meeting will be held jointly with Neural Plasticity and Recovery after Traumatic CNS Injury & Stroke.

Neurobiology, Neuroscience, Neurodevelopmental Disorders, Gene Therapy, Translational Research, Functional Genomics, Genetics, Genome Editing, Neurogenomics

Neurologie,    Molekularbiologie

Keystone Symposia

Genetic medicines are transforming how we treat cardiovascular disease, unlocking opportunities to correct heart and vascular disorders in ways once thought impossible. Basic scientists, clinicians, industry leaders, and innovators will convene at Genetic Medicines to Treat Cardiovascular Disease to explore how gene therapy, RNA-based approaches, gene editing, and advanced disease models are driving these breakthroughs. The program blends cutting-edge science and real-world translation. Sessions will highlight advances in mechanisms underlying cardiovascular genetics, therapeutic target identification, and novel delivery technologies. Plenary speakers and abstract-selected talks will connect foundational research with translational strategies, offering attendees a holistic view of where the field is headed. Additionally, the program will feature panel discussions focused on the steps leading from basic discovery to translation, commercialization, FDA approval, and navigating the venture capital landscape. Key topics include challenges and opportunities in delivery, efficacy, safety, trial design, long-term and off-target effects of therapies, and ethical and regulatory considerations. The program also includes lessons from preclinical and clinical trials, and professional development challenges for early-career researchers. This meeting also emphasizes interdisciplinary collaboration and career development. Career roundtables provide trainees and early-career investigators with direct access to leaders from academia, biotech, and industry for guidance on scientific and professional growth.

Metabolism, Cardiovascular Genetics, RNA Metabolism, Genetics, Geonomics, Stem Cell Biology, Translational Research, Gene Therapy, RNA Therapeutics, Biotech Innovation

Molekularbiologie,    Stammzellforschung und -therapie

Keystone Symposia

The Keystone Symposium on Precision Genome Engineering will highlight transformative advances that are reshaping the future of genetic medicine by pushing the boundaries of what is possible with genome editing technologies. As the field evolves from proof-of-concept to therapeutic application, critical questions around efficiency, specificity, safety, delivery, and scalability remain. This conference will address these gaps by convening world leaders and emerging innovators to explore next-generation tools, delivery strategies, integrative technologies, and clinical findings that are redefining genome engineering. What sets this symposium apart is its deep focus on the precision and evolution of genome editing itself—ranging from mechanistic insights to therapeutic design—paired with timely discussions on how AI and delivery systems are accelerating discovery. Attendees will leave with an overview of where the field is headed and how to contribute to its next breakthroughs. This meeting will be held jointly with Targeted Delivery of Nucleic Acid Medicines.

Genetics, Genomics, RNA, CRISPR, Translational Research, Artificial Intelligence, Gene Delivery, Molecular Biology

Molekularbiologie,    Implementierung und Anwendungen von künstlicher Intelligenz

Keystone Symposia

There have been tremendous advances in developing novel nucleic acid-based strategies for treating human diseases. Recent clinical successes have validated nucleic acid-based platforms, documenting that they can provide life-changing medicines to patients who need them. However, efficient delivery of these nucleic acid payloads to the cell type of interest and the appropriate subcellular compartment still limits their full potential despite significant progress over the past decade. This meeting is designed to bring together scientists from multiple disciplines, all working on delivery of nucleic acid cargos to cells. The meeting will cover the latest designs for viral vectors, lipid nanoparticle systems, virus-like particles, and ligand conjugates to enhance delivery of nucleic acid payloads. This meeting is unique in bringing scientists working on these different strategies for drug delivery into a single venue. Although these strategies have the common goal of enhancing intracellular delivery of nucleic acid cargo they tend to be siloed. A key goal of the meeting is to bring scientists together who have different expertise and perspectives to learn from each other, enabling more rapid and effective solutions for these delivery challenges. This will be a joint conference with the genome engineering meeting, which creates a lot of synergies. The genome engineering will discuss various nucleic acid cargo, while this meeting will focus on delivery technologies for the nucleic acid cargo. This meeting will be held jointly with Precision Genome Engineering: From Laboratory to Clinic.

Genetics, Genomics, RNA, Nucleic Acid Therapeutics, Drug Delivery, Gene Therapy, Viral Vectors, Nanoparticles, Translational Research

Molekularbiologie,    Pharmakologie und Arzneimittelentwicklung