Somatic Cell Gene Editing

Gene Therapy for Ophthalmic Disorders Summit
2024-09-10 - 2024-09-12
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. Understand the crucial role of endpoint selection to clearly demonstrate product efficacy to the FDA & EMA. Understand the risk-benefit profiles of different ocular administration routes including intravitreal, subretinal, and suprachoroidal delivery to choose the best option for your specific indication. Learn from successful clinical trials as well as where trials have fallen short of expectations to take forward crucial lessons learnt on progressing a gene therapy rapidly through the clinic without compromising on safety.
Boston, MA

Webinar: Considerations in leveraging the Universal AAV Standard for dPCR to assess AAV genomes quantity and quality
2024-09-10 10:00 - 10:30 (America/Chicago)
This webinar will explore the utility of a Universal AAV Standard in assessing the quantity and genomic integrity of AAV vectors. The Universal AAV Standard combines multiple target sites for commonly used promoters, poly(A) regions and functional CDS. Stacking of these elements in Universal AAV Standard allows it to serve as a single positive control for evaluating the performance of dPCR assays and assessing the quantity, quality, and genomic integrity of AAV genomes.
Register at https://www.workcast.com/register?cpak=5801990019211892&referrer=tw

HI-POCT 2024
2024-09-19 - 2024-09-20
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. The conference will provide a strategic forum in which clinicians, industry experts, innovation experts, researchers, and students will examine how to define unmet clinical needs and successfully travel along the innovation cycle toward commercialization and patient impact. Clinical, technological, regulatory, and marketing aspects of this learnable process will be stressed. Expert panels will discuss learnings in the development, translation, and commercialization of technologies and challenges in bringing medical care innovation to community health.
Tuscon, AZ

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The Safe and Effective Practice of AAV Gene Therapy
2024-09-20 07:30 - 17:30
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. SCGE researchers Lindsey George, Beverly Davidson, and Rebecca Ahrens-Nicklas are scheduled to be speaking at the event.
Friday September 20, 2024 || Philadelphia, PA
View the registration info here

Meeting 1: Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases
2024-09-20 10:00 - 15:30
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 objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. The meeting will be divided into two sessions.
Register for the Zoom meeting here: https://web.cvent.com/event/0b2da86e-f7d1-4288-9d11-2cb9cabe87c5/regProcessStep
Registration will close on Thursday, September 19, 2024, at 11:59 pm ET. A recording and other relevant meeting materials will be posted online following the event.

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

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/

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/

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/

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

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

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 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

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

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
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/
