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

With well over 5,000 attendees the Festival is now the [...]

The Advanced Therapies Congress is Europe’s largest commerical cell and [...]

Meet the global CRISPR Medicine community at the second CRISPR MEDiCiNE conference.

Human Genetics and Genetical Disorders-2025 extend our immense pleasure and honor [...]

Stay tuned for more information on ASGCT's 2025 annual meeting. [...]

The Festival is designed principally for scientists and clinicians who are [...]

We cordially invite you to The Global Cell & Gene [...]