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

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.

This free, public event will explore key immunology-related topics that have implications across the class of AAV gene therapies, including immune responses, antibody testing, and redosing strategies.

Stay tuned for more details

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

This year, NCATS and the U.S. Food and Drug Administration [...]

This inaugural virtual event March 12 and 13, 2025 (9 [...]

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

The Rare Disease Summit connects key stakeholders to drive therapeutic [...]

Join peers to drive gene therapy innovation across translational, clinical, regulatory, and manufacturing tracks.

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

Explore cutting-edge advancements in the use of AAV vectors for [...]

The 2025 programme will highlight non-coding variants and the implication [...]

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

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

Engage with analytical leads to discuss shared pain points, new [...]

At the Workshops on Measurements and Standards for Advanced Therapy, [...]

The Cell and Gene Therapy Products (CGTP) Symposium: Manufacturing, Quality [...]

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

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

The specific goal for this meeting is to foster fruitful [...]

The Cell & Gene Therapy World Conference 2025 (CGTWC 2025), [...]

Shape the future of rare disease treatments, research and policy [...]