Somatic Cell Gene Editing

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

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/

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/

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

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

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/

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/

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

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/

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

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/

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

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

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

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

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

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

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

FDA Scientific Exchange 2024: Advancing Gene Editing Platforms for Rare Diseases
2024-11-20 08:00 - 15:00
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

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

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/

World Orphan Drug Congress Europe
2024-10-22 - 2024-10-25
The World Orphan Drug Congress is the largest and most established orphan drugs & rare diseases meeting of its kind across the globe.
Barcelona, Spain

Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub
2024-10-16 09:00 - 13:30
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 meeting will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the recently announced Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work.
Hybrid Public Meeting: In-Person and Virtual
October 16, 2024 | 10am-2:30pm ET
Register at: https://reaganudall.org/news-and-events/events/advancing-rare-disease-therapies-through-an-fda-rare-disease-innovation-hub

2024 STAT Summit
2024-10-16 - 2024-10-17
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.
Hear from SCGE Phase 2 researchers Sonia Vallabh and Allyson Berent at this event! Sonia presents at 12:05pm ET on 10/16, and Allyson presents at 3:45pm ET on 10/17.
Find more info at: https://www.statnews.com/2024/summit/2024-stat-summit-2/
Boston, MA or Virtual

Advancing Gene and Cell Therapies for Cancer
2024-10-16 - 2024-10-17
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.
Philadelphia, PA and Virtual

7th International Conference on CRISPR Technologies
2024-10-14 - 2024-10-16
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.
San Diego, CA

RESI Boston 2024
2024-09-25 - 2024-09-27
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. RESI is uniquely cross-border and cross-domain, connecting start-ups with 10 categories of global investors across the silos of drugs, devices, diagnostics and digital health (4 Ds). RESI caters to both the earliest stage start-ups, those seeking grants, seed and angel capital, and the early-stage firms who seek series A and B funding. RESI is a unique and powerful tool for sourcing assets and advancing innovation across early-stage life science and healthcare.
9/25 – Boston
9/26 and 9/27 – Virtual
https://resiconference.com/

Week in RARE
2024-09-25 - 2024-09-28
Week in RARE, presented by Global Genes, features the RARE Health Equity Forum and the RARE Advocacy Summit.
The RARE Health Equity Forum convenes stakeholders in the rare disease space to identify ways in which we can better serve marginalized populations within the rare disease community. In its third year, the event will be focused on the theme of “Equity In Action”.
The RARE Advocacy Summit provides the opportunity to forge meaningful connections with others in the rare disease community for future collaboration. Sessions provide attendees with insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies they can take home and implement immediately to accelerate change.
Find more information at: https://www.globalgenes.org/week-in-rare/

3rd Annual Lipid Nanoparticles Development Summit Europe
2024-09-24 - 2024-09-26
The 3rd Lipid Nanoparticles Development Summit Europe is your premier forum to collaborate with a carefully curated community of experts across Discovery, Characterisation, Translatability, Formulation, Biodistribution, Manufacturing and much more. These industry leaders are gathering in Berlin this September to bring the greatest challenges in LNP development to the fore, tackling them head on to ensure 2024 is the year of the LNP.
Berlin, Germany
Learn more at: https://lipid-nanoparticle-development-europe.com/

2024 Policy Summit
2024-09-23 - 2024-09-24
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. Join other cell and gene therapy professionals at this 1.5-day event to learn about public policies and regulatory trends impacting the field.
Washington DC/Hybrid
Register at https://www.asgct.org/advocacy/events-and-opportunities/policy-summit/registration
