Genomics of Rare Disease

The 18th conference on rare disease combines recent research on genetic mechanisms involved in disease, with advances in approaches for clinical care. This year’s conference will include discussions on the use of a ‘pangenome’ to improve diagnostic yield, the role of common variants in rare disease, and new developments in prenatal and neonatal genomic screening.

Pharma USA 2024

Join pioneers from Commercial, Marketing, Medical Affairs, RWE, Market Access, Patient Engagement, and key stakeholders, will come together to deliver value to overcome the challenges that threaten the future viability of innovation.

Gene Therapy for Rare Disorders

Bringing together key figures from industry, regulatory bodies, patient non-profits, payers, and key service providers, this is the field’s foremost meeting for collectively progressing rare gene therapy programs into and through the clinic. 

CRISPR and Beyond: Perturbations at Scale to Understand Genomes

The 5th CRISPR and Beyond conference provides a forum for biomedical researchers from academia and industry working on high throughput screening, genome engineering, and variant effect interpretation to discuss perturbations at scale to understand genomes.

The CRISPR MEDiCiNE Conference

Meet the European CRISPR Medicine and Genomic Medicine community to discuss tools, delivery, safety, functional genomics, standards and regulations, and more. 

Rare Drug Development Symposium

The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance therapy development for their communities.

ASGCT Annual Meeting

The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting is the premier event for professionals in gene and cell therapy to learn from the latest scientific research, stay up to date on new technologies, and make career-advancing connections with peers.

International Society for Cell & Gene Therapy

Address obstacles at each stage of translation and progress the future of cell and gene therapies to bring scientific innovation to therapeutic reality for patients worldwide.

Gene Therapy Patient Engagaement

Join 80+ patient advocacy and engagement professionals at gene therapy companies and patient advocacy groups to gain actionable insights that you can immediately implement into your own gene therapy programs, and come together with the community to develop solutions to the challenges that are unique to the gene therapy space.

Cell and Gene Therapy Products Symposium

The Cell and Gene Therapy Products (CGTP) Symposium: Manufacturing, Quality and Regulatory Considerations enables the exchange of scientific ideas and dialogue with regulators that form the basis of evolving regulatory practices in the development of these diverse and innovative products.

RNA Therapeutics

Learn more about RNA Therapeutics from concept to clinic from leaders across the field, including SCGE PI's Jennifer Doudna, Kiran Musunuru, and Sonia Vallabh. 

The Global Cell & Gene Therapy Summit

The International Summit on Cell and Gene Therapy serves as a platform to explore the fundamental concepts of these transformative therapies. The summit brings together experts from academia and industry to discuss a range of topics related to cell and gene therapy, including ethical considerations, social implications, tools, techniques, marketing strategies, and the commercialization of cell and gene therapy products. It also showcases the latest research progress in CGT and explores opportunities for further enhancements in availability and productivity of these therapies.

Genome Engineering: CRISPR Frontiers

The specific goal for this meeting is to foster fruitful and creative interactions between researchers interested in applying these systems to genome engineering and related advances in a wide variety of organisms, together with scientists studying the basic biology of CRISPR-Cas and related bacterial defense systems.

Week in RARE

Week in RARE, presented by Global Genes, features the RARE Health Equity Forum and the RARE Advocacy Summit.