Somatic Cell Gene Editing

6th Annual Gene Therapy Analytical Development & CMC Summit
2024-10-30 - 2024-11-01
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.

Boston, MA
Learn more at https://www.genetherapy-analytical-cmc.com/

Breakthroughs in Muscular Dystrophy
2024-11-19 - 2024-11-20
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 1.5-day event will bring together leading researchers and clinicians from the neuromuscular disease community to explore cutting-edge gene and cell therapies and technologies for muscular dystrophy.
Chicago, IL
https://www.asgct.org/events/breakthroughs-in-muscular-dystrophy/registration

FDA Scientific Exchange 2024: Advancing Gene Editing Platforms for Rare Diseases
2024-11-20 08:00 - 15:00 (America/Chicago)
On November 20th, ISCT, ARM, and Danaher will convene key stakeholders to explore how to advance gene editing platforms. 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 Exchange will review case studies from academics and industry developers focused on gene editing, with the objective of defining repeatable platforms that streamline development and regulatory requirements across multiple therapies for rare populations. Successful platforms hold promise to deliver faster and broader patient access to novel gene therapies while maintaining favorable risk-benefit balance and appropriate regulatory rigor. The attached background materials provide additional context.
The event will be live-streamed.
Register at https://www.isctglobal.org/event/fda-scientific-exchange-2024

Real-World Evidence Webinar Series: Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice
2024-11-22 12:00 - 12:45
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.
The guidance document addresses randomized controlled trials (RCTs) that have been integrated within routine clinical practice. Such RCTs have streamlined protocols that focus on collection of essential data and are often referred to as point-of-care or large, simple trials. The guidance document discusses how leveraging established health care institutions can help reduce startup time and support trial enrollment. Involving local health care providers also offers convenience and accessibility to diverse patient populations who may not otherwise have the opportunity to participate.
Register at https://reaganudall.org/news-and-events/events/real-world-evidence-webinar-series-integrating-randomized-controlled-trials

Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data
2025-01-22 08:00 - 2025-01-23 12:00
ASGCT and FDA will host a virtual scientific workshop over two half days. 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. The workshop will provide valuable insights for professionals in both academia and industry as it addresses pressing challenges in AAV gene therapies and new approaches.
Register at: https://www.asgct.org/asgct-events/january-2025/class-considerations-on-immunogenicity-for-aav-gen

WCBP Symposium
2025-01-28 - 2025-01-30
Stay tuned for more details.

The Mayflower Hotel in Washington D.C., United States.

https://www.casss.org/meetings-and-events/event/2025/01/28/default-calendar/wcbp-2025

The Festival of Genomics & Biodata London
2025-01-29 - 2025-01-30
With well over 5,000 attendees the Festival is now the UK’s largest annual life sciences event. Thanks to the support of many people and organisations, the Festival has become the annual get-together for the UK genomics community. It’s also expanded to cover so much more than genomics, including biodata, diagnostics, drug discovery, AI, proteomics, multi-omics and single-cell & spatial analysis.
London, UK
Learn more at https://festivalofgenomics.com/london/en/page/home

FDA-NIH Rare Disease Day 2025
2025-02-27 - 2025-02-28
This year, NCATS and the U.S. Food and Drug Administration (FDA) will sponsor a shared Rare Disease Day event as part of this global observance. FDA-NIH Rare Disease Day aims to provide the rare disease community — patients, caregivers, families, patient advocate groups, researchers, clinicians, health care providers, and trainees/students —with information relevant to their lived experiences, including activities by FDA and NIH that promote research and product development for this underserved population.
Virtual and In-Person
More info: https://ncats.nih.gov/news-events/events/rdd

Empowering Patients 2025
2025-03-12 - 2025-03-13
This inaugural virtual event March 12 and 13, 2025 (9 a.m. – 12:30 p.m. ET on both days), will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates, patients, and caregivers who are vital in developing treatments. Attendees will hear from experts in the CGT field, as well as patient advocacy group leaders. There will be opportunities to ask questions and deepen your knowledge of the community’s CGT journey.
Virtual
https://www.asgct.org/asgct-events/march-2025/empowering-patients-2025-a-cell-and-gene-therapie

Advanced Therapies 2025
2025-03-18 - 2025-03-19
The Advanced Therapies Congress is Europe’s largest commerical cell and gene therapy conference and exhibition. The event is for the leaders of pioneering ATMP companies and their most senior executives in charge of the latest tech and strategies that are driving the industry forward. In 2025, we’ll be bringing our community of 2,500 global executives back together once again at London’s ExCel, to be inspired by 300 speakers and 100 start-ups over 2 incredible days. The Advanced Therapies Congress features speakers from across the entire value chain of cell and gene therapy development; forward-thinking pharma, biotech and start-up companies, researchers, clinicians, academics, HTAs, payers and regulators.
ExCel, London, UK
Learn more at https://www.terrapinn.com/congress/advanced-therapies/index.stm

Rare Disease Summit 2025
2025-03-18 - 2025-03-20
The Rare Disease Summit connects key stakeholders to drive therapeutic progress, propel commercial strategies and inspire impactful advocacy. You’ll generate solution-focused approaches surrounding patient access, reimbursement, commercialization, launch, partnering and more, as you unify in areas of unmet medical need.
Philadelphia, PA
https://informaconnect.com/rare-disease-summit/

8th Gene Therapy Development Summit
2025-03-25 - 2025-03-27
Join peers to drive gene therapy innovation across translational, clinical, regulatory, and manufacturing tracks. This event is designed to help attendees navigate the entire gene therapy pipeline. By refining gene delivery platforms, optimizing manufacturing processes, and navigating regulatory pathways, we can revolutionize the future of gene therapies—bringing them to broader patient populations and significantly enhancing clinical outcomes.
https://genetherapy-conference.com/
Boston, MA

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/

Around the World: Frontline of AAV Vectors for Gene Therapy Research in Japan
2025-04-08 19:00
Explore cutting-edge advancements in the use of AAV vectors for gene therapy research in Japan. This event brings together leading scientists, researchers, and professionals in the field to discuss innovative approaches, challenges, and breakthroughs in gene delivery systems, specifically focusing on AAV technology.
Virtual
Register: https://www.asgct.org/asgct-events/april-2025/aav-vectors-gene-therapy-research-japan

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
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
