CRISPR MEDiCiNE 2025
2025-04-07 - 2025-04-11
Meet the global CRISPR Medicine community at the second CRISPR MEDiCiNE conference. The focus will be on tools, delivery, safety/off-target, diseases, pre-clinical/clinical, standards and regulations, functional genomics, target identification, and target validation.
April 7-11th, 2025; Copenhagen, Denmark – Virtual event available April 7th
Learn more at https://crisprmedicinenews.com/crisprmed/crisprmed25/

Genomics of Rare Disease 2025
2025-04-09 - 2025-04-11
The 2025 programme will highlight non-coding variants and the implication of rare disease on common diseases. Advances in therapeutics, next-generation functional testing, and the impact of genomics diagnostics and research on rare disease across the globe will also be showcased.
United Kingdom (Virtual or In-person)
https://coursesandconferences.wellcomeconnectingscience.org/event/genomics-of-rare-disease-20250409/

2nd International Conference on Human Genetics and Genetical Disorders
2025-04-14 - 2025-04-16
Human Genetics and Genetical Disorders-2025 extend our immense pleasure and honor to invite you to attend the 2nd International Conference on Human Genetics and Genetical Disorders which is scheduled on April 14-16, 2024 in Paris, France. One of these directions was the need for genetic studies to identify genes and genetic markers of diagnostic, prognostic and therapeutic value Genetic processes work in combination with an organism’s environment and experiences to influence development and behavior often referred to as nature versus nurture.
Paris, France
Learn more at https://geneticsresearch.net/

ISCT 2025
2025-05-06 - 2025-05-09
International Society of Cell & Gene Therapy 2025 Annual Meeting
New Orleans, LA
https://www.isctglobal.org/annual-meeting/homepage

ASGCT 2025
2025-05-13 - 2025-05-17
Stay tuned for more information on ASGCT’s 2025 annual meeting.
New Orleans, LA

Global Observatory for Genome Editing International Summit
2025-05-21 - 2025-05-23
The Global Observatory’s 2025 Summit foregrounds questions about biotechnologies that may alter the meaning of being human. Genome editing, especially of the human germline, raises fundamental questions of the dignity and integrity of human life. Governance concerns once focused on the escape of potentially risky lab-created organisms. Today the more urgent concerns relate to transgressing moral boundaries and violating human integrity. How should the scientific community respond?

Virtual or In-person in Cambridge, MA

https://www.summit.global-observatory.org/

Gene Therapy Potency Assay Summit
2025-05-28 - 2025-05-29
Engage with analytical leads to discuss shared pain points, new technologies enabling improved predictability, and hear perspectives from those who have been through IND-enabling studies and all the way to commercialisation of their gene therapy products.
Boston, MA
https://genetherapy-potency-assay.com/

NIST Workshops
2025-06-09 - 2025-06-13
At the Workshops on Measurements and Standards for Advanced Therapy, stakeholders from federal agencies, industry, and academia will provide perspectives on identifying measurement challenges and innovative technology solutions, as well as standards to accelerate R&D (including the use of ML/AI for biological data), promote manufacturing innovation, improve supply chain resilience, and support characterization and testing to facilitate regulatory approval.
The workshops will include:
AI and Flow Cytometry Workshop (June 9 to 10, 2025)
Cell Characterization Standardization Workshop (June 11, 2025)
Genome Editing Consortium Workshop (June 12 to 13, 2025)
Rockville, MD
Learn more at: https://www.nist.gov/news-events/events/2025/06/second-series-workshops-measurements-and-standards-advanced-therapy

Cell & Gene Therapy Products 2025
2025-06-10 - 2025-06-12
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. The 2025 Symposium will explore how to continue to adapt concepts applied to other biologics and how to establish novel approaches where they are needed.
Rockville, MD
More info: https://www.casss.org/cell-gene-therapy-products

The State of CRISPR & Genome Editing 2025
2025-06-11 10:00 - 14:30 (America/Chicago)
In The State of CRISPR and Genome Editing virtual summit, GEN brings together an outstanding line-up of pioneers and practitioners in genome editing to provide a compelling and candid overview of the field. Attendees will hear about the latest scientific, technology, commercial and regulatory advances that will enable genome editing to unleash its full potential in the clinic and beyond.
Hear from SCGE researcher Fyodor Urnov PhD on the path to achieving CRISPR cures for hundreds of rare genetic diseases.
Register for this free event at https://webinars.liebertpub.com/e/the-state-of-crispr-and-gene-editing-2025

The Festival of Genomics & Biodata
2025-06-24 - 2025-06-25
The Festival is designed principally for scientists and clinicians who are working in the fields of genomics, other omics, and/or using biodata , to further research, drug discovery, healthcare and – ultimately – deliver better patient outcomes. Topics covered include genomics, single cell/spatial biology, multiomics, biodata, cancer research, drug discovery, AI, microbiome, proteomics, liquid biopsy and so much more. It’s brimming with essential insights from 170+ word class speakers, challenging discussions on essential topics, and powerful learning and networking experiences.

Boston, MA

Learn more at https://festivalofgenomics.com/boston/en/page/boston-home

Patient-Centered Long-Term Follow-up for Gene Therapies
2025-06-26 12:00 - 13:00
This webinar will consider patient-centered approaches in the specific context of Gene Therapy (GT) Long-Term Follow-Up (LTFU) studies. GTs have the potential for long-lasting and transformative health benefits, but there are also possible long-term health risks. Therefore, the FDA recommends long-term safety monitoring of recipients of certain types of GTs to minimize and better understand any health risks. Although important, these LTFU studies can last years and may pose significant burdens on patients, so it is critical to consider patient needs and preferences in the design and conduct of LTFU for GTs. Three panelists will offer different perspectives on how we can put patients at the center of LTFU for GTs.
Register at https://lp.constantcontactpages.com/ev/reg/83r3gyp/lp/13903b2d-e150-44c8-9ae8-8ca48a0ebc62

Cell & Gene Therapy Summit 2025
2025-07-07 - 2025-07-09
We cordially invite you to The Global Cell & Gene Therapy Summit, which will be held in Orlando, FL, from July 07 to 09, 2025. Professionals and researchers are requested to take part as delegate, sponsor, or exhibitor. The three-day conference aims to bring together the global audience to discuss and exchange ideas on the origin, development, clinical applications and translation science of cell & gene therapy.
The inclusion of talks, poster presentation, workshops, discussions and networking events over the three days indicates a comprehensive program design to engage participants of various interactive formats. It allows the attendees to learn from experts, showcase their own research through posters, participate in workshops to gain practical insights, engage in discussions to share perspectives, and connect with professional researchers from diverse backgrounds.
By fostering such an environment, The Global Cell & Gene Therapy Summit, provides an excellent platform for researchers, clinicians, industry professionals, and other stakeholders to stay up to date with the latest advancements in the field of cell & gene therapy. It also facilitates collaboration and the exchange of knowledge, which is crucial for accelerating the translation of scientific discoveries into potential clinical applications.
Orlando, FL (Hybrid)
Learn more at https://cgtsummit.com/

Genome Engineering: CRISPR Frontiers
2025-08-12 - 2025-08-16
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. Hear from numerous SCGE researchers, including David Liu, Rebecca Ahrens-Nicklas, Ben Kleinstiver, Niren Murthy, and Jonathan Weissman.
Cold Spring Harbor Laboratory
https://meetings.cshl.edu/meetings.aspx?meet=CRISPR&year=25

Cell & Gene Therapy World Conference 2025
2025-09-10 - 2025-09-12
The Cell & Gene Therapy World Conference 2025 (CGTWC 2025), Boston, Massachusetts, USA, focuses on the vital progression of innovations and breakthroughs through cutting-edge research and practice in gene therapy for neurological disorders. Our aim is to explore new pathways in the field and bridge existing gaps in treatment options.
Boston, MA
https://www.cellgenetherapyconference.com/

NORD® Rare Diseases and Orphan Products Breakthrough Summit
2025-10-20 - 2025-10-21
Shape the future of rare disease treatments, research and policy at the NORD® Rare Diseases and Orphan Products Breakthrough Summit.® Together, we can advance equity and innovation for the more than 30 million Americans – and more than 400 million people worldwide – with rare diseases!
Washington DC
nordsummit.org

Molecular Therapy Presents: Advancing AAV Technology
2025-10-22 10:00 - 11:00
“Advancing AAV Technology: From Capsid Design to Scalable Manufacturing” – An ASGCT Webinar
Virtual | Free

Breakthroughs in Targeted In Vivo Gene Editing
2025-11-20 - 2025-11-21
Join ASGCT at the Marriott Marquis San Diego Marina, Nov. 20-21, 2025, for an unparalleled opportunity to immerse yourself in cutting-edge solutions for targeted delivery of gene editing technology in vivo in San Diego. Hear from SCGE researchers Bill Peranteau, Sonia Vallabh, Yong-Hui Jiang, Kiran Musunuru, and Rebecca Ahrens-Nicklas at the event.
Virtual and In Person (San Diego, CA)
https://www.asgct.org/events/breakthroughs

Around the World: Technological Frontiers in South American Gene and Cell Therapy
2025-12-16 10:00 - 11:00
As gene and cell therapies revolutionize modern medicine, South America is emerging as a dynamic player on the global stage—leveraging scientific innovation, regulatory evolution, and regional collaboration. This free virtual program explores the technological frontiers shaping the development and deployment of advanced therapies across the continent.
Webinar | Free

Public Meeting: FDA Rare Disease Day 2026
2026-02-23 08:00 - 15:00
FDA will host Rare Disease Day, a virtual public meeting, on Monday, February 23, 2026 in global observance of Rare Disease Week. The theme is: “Moving Forward. Looking Ahead. An Event for Patients.” The goal of this year’s Rare Disease Day is to explore ways to engage and collaborate with patients and their communities to support and accelerate the development of medical products for rare diseases. We want patients to connect with FDA and stay involved with our outreach and engagement programs to ensure the patient voice informs medical product development and regulation throughout FDA. Panels will discuss:

Patient Focused FDA Initiatives
Patient Engagement Opportunities
Addressing Challenges and Opportunities with AI Technology
Utilizing Real-World Data and Real-World Evidence at FDA

More info at https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/public-meeting-fda-rare-disease-day-2026-02232026

Rare Disease Day at NIH
2026-02-27 08:00 - 16:00
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.
The event will be held at NIH Main Campus (Natcher Conference Center), and there will also be a virtual livestream, with the event archived for replay afterward. The event is free and open to the public.
More info at: https://ncats.nih.gov/news-events/events/rdd

Empowering Patients 2026: A Cell and Gene Therapies Summit
2026-03-11 - 2026-03-12
This free, virtual summit from ASGCT will bring together patients, caregivers, advocates, researchers, and industry leaders for two dynamic half-days of learning the latest insights in cell and gene therapy.
Hear from SCGE researchers Steve Gray and Kiran Musunuru at the event.

9th Annual Gene Therapy Development Summit
2026-03-24 - 2026-03-26
The 9th Gene Therapy Development Summit returns to Boston as the meeting point for this community. Join 100+ leading voices from the likes of Bayer, Astellas Pharmaceuticals, REGENXBIO, Ocugen, AskBio, CureDuchenne Ventures, and more to come together to share ideas, restore confidence, and chart a sustainable path forward for gene therapy. Connect with your peers in pre-clinical development, manufacturing, analytical development, clinical operations, and regulatory affairs for an unrivalled learning and networking opportunity across the end-to-end of gene therapy development.
Boston, MA

Molecular Therapy Presents: Lipid Nanoparticles for Gene and Cell Therapy
2026-03-25 10:00 - 11:00
Lipid nanoparticles (LNPs) have emerged as promising vehicles for delivering therapeutic payloads for cell and gene therapy. They offer several advantages, such as biocompatibility, low immunogenicity, and the ability to encapsulate a wide range of cargoes. From the development of mRNA vaccines to the targeted delivery of gene editing tools and RNA interference therapeutics, LNPs are driving transformative breakthroughs in treating a myriad of diseases, including cancers, genetic disorders, and infectious diseases.
This free webinar will explore the complex interplay between advanced nanotechnology and biomedical innovation, focusing on the transformative potential of LNPs in developing precise and effective therapeutic interventions.
Learn more at https://www.asgct.org/events-meetings/molecular-therapy-presents-lipid-nanoparticles-for-gene-and-cell-therapy

PPQ Readiness: What to Know Before Starting a PPQ Campaign
2026-04-08 11:00 - 12:00
Process Performance Qualification (PPQ) verifies that a therapy’s manufacturing process consistently produces high-quality doses; regulators rely on the results before allowing wider use. This free webinar will define what must be in place before a campaign launch, outline the activities that precede PPQ, and highlight common gaps and pitfalls, along with practical mitigation steps.
Learn more at https://www.asgct.org/events-meetings/ppq-readiness-what-to-know-before-starting-a-ppq-campaign

NORD® Rare Disease Scientific Symposium 2026
2026-04-14 - 2026-04-15
The NORD® Rare Disease Scientific Symposium is bringing together a dynamic community of researchers, clinicians, industry leaders, and advocates to spark the conversations and connections that drive science forward.
This immersive, 1.5 day event is designed to push boundaries and bridge gaps across the rare disease landscape. Through engaging case studies, interactive panel discussions, and extended audience Q&A, participants will explore critical topics such as:

Innovative Study Designs and Methods for Rare Disease Research
Approaches to Address the Challenges of Small Trial Enrollment and Disease Heterogeneity
Successes and Challenges of Global Clinical Trials
Using Alternative and Real-World Data for Rare Disease Research
Multi-Stakeholder Collaboration in Research
Strategies for Earlier Pediatric Inclusion in Clinical Trials

Learn more at: https://nordscience.org/

2nd Annual Gene Therapy Symposium
2026-04-24 - 2026-04-25
The 2nd Annual Gene Therapy Symposium brings together leading scientists, clinicians, and industry innovators shaping the future of genetic medicine. Our speakers represent a diverse cross-section of expertise across research, clinical translation, regulatory science, and biotechnology.

SCGE researcher Becca Ahrens-Nicklas will be speaking at the event.

Columbus, OH

Learn more at https://web.cvent.com/event/2c97ab51-3da3-46df-abb4-544a428dcfa6/summary

ISCT 2026
2026-05-06 - 2026-05-09
International Society for Cell & Gene Therapy 2026 Annual Meeting
Dublin, Ireland
https://www.isctglobal.org/meetings/annual-meeting/isct-2026-dublin

ASGCT 2026 Annual Meeting
2026-05-12 - 2026-05-16
Boston, MA or Virtual
https://annualmeeting.asgct.org/

Breakthroughs in Cell & Gene Therapy 2026
2026-05-15 08:00 - 17:00
For the third year, ASGCT brings Breakthroughs in Cell & Gene Therapy to a new region, and this time, we’re heading to Music City! Join us November 12-13 at the Gaylord Opryland Resort in Nashville for our most expansive program yet: three concurrent tracks designed for the scientists, clinicians, and industry leaders shaping what’s next in the field.
Learn more at: https://www.asgct.org/events-meetings/breakthroughs-in-cell-gene-therapy
