Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and NIH collaborations that address scientific challenges and advance research for new treatments.

TAGC is a unique conference organized by the Genetics Society of America that offers innovative sessions that get to the heart of our shared research interests. The diverse and interdisciplinary scope of TAGC 2024 allows researchers to discover answers and explore new directions in today’s genetics and genomics research.

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.

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.

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. 

The 4th Gene Therapy for Muscular Disorders Summit in April will focus on gene therapies for muscular disorders to tackle the most pressing challenges and together, seek solutions to realize these novel therapeutic potentials for patients in need.

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.

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

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.

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.

This in-person and virtual public workshop will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss considerations for the use of natural history study and registry data in rare disease drug development programs.

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.

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.

Learn directly from the FDA’s regulatory experts in medical product centers: drugs, devices, and biologics. This course is designed to provide participants with a strong, basic foundation in the FDA’s regulatory requirements, and also create awareness of current activities. Attend virtually or in-person. 

In The State of CRISPR and Gene Editing virtual summit, GEN proudly gathers a tantalizing line-up of luminaries from academia and industry to discuss the latest research developments, innovations, and advanced technologies that are expanding the CRISPR toolbox, delivering new therapies to patients and safeguarding our food supply.

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.

This event will focus on finding and optimizing novel vectors to increase specificity, target new cell types, expand packaging capacities, and reduce toxicity risks to create safer, cost-effective and efficacious gene therapies. 

The conference seeks to present the latest research findings in these fields and support early-career investigators, women, and other historically-excluded groups in the field of genome editing.

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 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.

Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic. 

The purpose of the CMC Strategy Forum is to provide a venue for biotechnology/biological product discussion on relevant CMC issues throughout the lifecycle of a product.

The 3rd Next Generation Lipid-Based Nanoparticles Delivery Summit is the only event dedicated entirely to lipid-based nanoparticle delivery, where we bring together 120+ experts in LNP non-clinical, delivery, particle engineering, and formulation from the likes of Teressa, Sanofi, MediciBio, CureVac.

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.

Join 100+ industry leaders at the 5th Gene Therapy for Ophthalmic Disorders Summit to uncover cutting-edge gene therapy breakthroughs. Harness the latest developments in vector engineering to target retinal cells more effectively with higher transduction efficiency.

This webinar will explore the utility of a Universal AAV Standard in assessing the quantity and genomic integrity of AAV vectors.

IEEE-EMBS International Conference on Health Innovations in Point of Care Technology (IEEE HI-POCT) is the premier conference in the areas of Health innovations in point-of-care technologies. In its eighth year, this IEEE EMB Strategic Conference will emphasize funding opportunities and the promise and pitfalls of the journey from idea to patient.

Join the Clinical In Vivo Gene Therapy (CIGT) team at Children’s Hospital of Philadelphia for an exciting one-day educational symposium, as we provide fundamental knowledge on the safe and effective practice of adeno-associated virus (AAV) gene therapy across medical specialties. 

The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases.

The 2024 Policy Summit will include experts on topics such as global regulatory harmonization, clinical development challenges, the payment policy landscape, CGT investment insights, and more.

The 3rd Lipid Nanoparticles Development Summit Europe is your premier [...]

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

The Redefining Early Stage Investments (RESI) conference series connects start-ups and early-stage investors and strategic channel partners. RESI maximizes fundraising companies’ efforts to find partners who are a fit for their technology and stage of development.

CRISPR 2024 is an exclusive conference that unites leaders from industry, government agencies, and academia to discuss the latest advancements in genome editing technologies. The program brings together experts from diverse fields to discuss how to safely and effectively translate these cutting-edge technologies to patients, while remaining mindful of the ethical implications.

During this conference, experts in the field will share their insights on targeting cancer-specific antigens, highlight rational combinations to further enhance the function of CAR engineered cells, describe ground-breaking efforts to engineer CAR engineered cells directly in vivo, and illuminate the biology and risks of viral vector integration.

By gathering real operators from the trenches who have not previously spoken out, not just talking heads, this event covers some of the biggest challenges faced by those who want to move advances out of the lab and into the world. They will discuss everything from drug development and technology invention to dealing with regulators, courting investors, and pricing and selling products.

The Reagan-Udall Foundation for the FDA, in collaboration with FDA’s Rare Disease Innovation Hub, will host a public meeting on October 16, 2024.

The World Orphan Drug Congress is the largest and most established orphan drugs & rare diseases meeting of its kind across the globe.

The 6th Gene Therapy Analytical Development & CMC Summit will unite 250+ analytical development, CMC, QC, and process development experts to showcase innovative analytical and process development methods, assess their readiness, and ultimately guarantee safety, quality, efficacy, and consistency of gene therapies to regulators.

Join ASGCT and the Muscular Dystrophy Association in downtown Chicago for an unparalleled opportunity to delve into the latest advancements in research on gene and cell therapies for muscular dystrophy.

This Scientific Exchange with the FDA seeks to make progress on therapeutic development for rare conditions that are scientifically feasible but commercially non-viable.

The Reagan-Udall Foundation for the FDA, in collaboration with the Food and Drug Administration (FDA), is hosting a public webinar as part of a series of webinars for FDA-issued guidance related to real-world evidence (RWE). This latest session in the webinar series will discuss the draft guidance: Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice.

Stay tuned for more details

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