Somatic Cell Gene Editing

CRISPR and Beyond: Perturbations at Scale to Understand Genomes
2024-04-22 - 2024-04-24
The rapid development of CRISPR/Cas-based technologies makes it possible to modulate genomes with relative ease. These tools can help us understand how genetic variation influences phenotype and thereby answer long-standing questions in biology that impact human health, laying the foundations for precision medicine for heritable diseases and cancer treatment.
The 5th CRISPR and Beyond conference provides a forum for biomedical researchers from academia and industry working on high throughput screening, genome engineering, and variant effect interpretation to discuss perturbations at scale to understand genomes.
This year’s meeting will highlight recent advances enabled by emerging technologies and models, including computational approaches and designing DNA for function. Discussions will focus on the recent discoveries in precision genome editing, coding and non-coding variation and genetic interactions. A panel discussion will probe how generative modelling and other applications of machine learning are shaping the present and future of the field, and impacting experimentation.
Find more information at https://coursesandconferences.wellcomeconnectingscience.org/event/crispr-and-beyond-perturbations-at-scale-to-understand-genomes-20240422/

The CRISPR MEDiCiNE Conference
2024-04-23 - 2024-04-25
Meet the European CRISPR Medicine and Genomic Medicine community to discuss tools, delivery, safety, functional genomics, standards and regulations, and more.
Find more information: https://crisprmedicinenews.com/crisprmed24/

Rare Drug Development Symposium
2024-04-29 - 2024-05-01
The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance therapy development for their communities.
Find more information at: https://www.globalgenes.org/event/rare-disease-drug-development/

ASGCT Annual Meeting
2024-05-07 - 2024-05-11
The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting is the premier event for professionals in gene and cell therapy to learn from the latest scientific research, stay up to date on new technologies, and make career-advancing connections with peers.
Learn more at https://annualmeeting.asgct.org/
Want to see the SCGE’s involvement in ASGCT 2024? Click here.

Natural History Studies and Registries in the Development of Rare Disease Treatments
2024-05-13 09:00 - 15:00 (America/Chicago)
This in-person and virtual public workshop will bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss considerations for the use of natural history study and registry data in rare disease drug development programs.
Find more information at https://reaganudall.org/news-and-events/events/natural-history-studies-and-registries-development-rare-disease-treatments

International Society for Cell & Gene Therapy
2024-05-28 - 2024-06-01
Address obstacles at each stage of translation and progress the future of cell and gene therapies to bring scientific innovation to therapeutic reality for patients worldwide.
Vancouver, Canada
Learn more at https://www.isctglobal.org/isct2024/home

Gene Therapy Patient Engagaement
2024-05-29 - 2024-05-31
Join 80+ patient advocacy and engagement professionals at gene therapy companies and patient advocacy groups to gain actionable insights that you can immediately implement into your own gene therapy programs, and come together with the community to develop solutions to the challenges that are unique to the gene therapy space.
Boston, MA
https://genetherapy-patient-engagement.com/

2024 Regulatory Education for Industry
2024-05-29 - 2024-05-30
Learn directly from the FDA’s regulatory experts in medical product centers: drugs, devices, and biologics. This course is designed to provide participants with a strong, basic foundation in the FDA’s regulatory requirements, and also create awareness of current activities. Attend virtually or in-person.

REdI Annual Conference 2024

The State of CRISPR and Gene Editing
2024-06-05 10:00 - 14:30
In The State of CRISPR and Gene Editing virtual summit, GEN proudly gathers a tantalizing line-up of luminaries from academia and industry to discuss the latest research developments, innovations, and advanced technologies that are expanding the CRISPR toolbox, delivering new therapies to patients and safeguarding our food supply. The webinar is virtual and free to attend. SCGE Researcher David Liu will be giving the opening keynote.

Learn more at https://webinars.liebertpub.com/e/the-state-of-crispr-and-gene-editing-2024

Cell and Gene Therapy Products Symposium
2024-06-11 - 2024-06-13
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.
Rockville, MD
https://www.casss.org/cell-gene-therapy-products

Next Generation Gene Therapy Vectors
2024-06-12 - 2024-06-14
This event will focus on finding and optimizing novel vectors to increase specificity, target new cell types, expand packaging capacities, and reduce toxicity risks to create safer, cost-effective and efficacious gene therapies.
Learn more at https://next-gen-genetherapy-vectors.com/

Genome Engineering: Research and Applications
2024-06-16 - 2024-06-20
The conference seeks to present the latest research findings in these fields and support early-career investigators, women, and other historically-excluded groups in the field of genome editing. Through collaboration and networking at this meeting, it can accelerate the translation of fundamental advances into viable genome editing technologies. The conference is being held in Rome, Italy.
SCGE researcher Shengdar Tsai is one of the conference organizers, and SCGE researcher David Liu is the keynote speaker.
Learn more at https://events.faseb.org/event/genome-engineering-research-applications/summary

RNA Therapeutics
2024-06-26 - 2024-06-28
Learn more about RNA Therapeutics from concept to clinic from leaders across the field, including SCGE PI’s Jennifer Doudna, Kiran Musunuru, and Sonia Vallabh.
https://www.umassmed.edu/RNATx/

The Global Cell & Gene Therapy Summit
2024-07-08 - 2024-07-10
The International Summit on Cell and Gene Therapy serves as a platform to explore the fundamental concepts of these transformative therapies. The summit brings together experts from academia and industry to discuss a range of topics related to cell and gene therapy, including ethical considerations, social implications, tools, techniques, marketing strategies, and the commercialization of cell and gene therapy products. It also showcases the latest research progress in CGT and explores opportunities for further enhancements in availability and productivity of these therapies.
Boston, MA (Hybrid)
https://cgtsummit.com/

In Vivo Cell Engineering & Gene Editing Summit
2024-07-09 - 2024-07-11
Achieve Safe & Targeted In Vivo Delivery of Payloads with Clarity in Regulations & Appropriate Models to Advance the Next Frontier of In Vivo Editing from Research to Clinic.
Boston, MA

CMC Strategy Forum North America
2024-07-16 - 2024-07-17
The CMC Strategy Forum has established an excellent reputation for being the premier scientific meeting that brings together regulatory, industry, and academic professionals to discuss key emerging CMC issues that affect biotechnology in an open and transparent workshop format. The purpose of the CMC Strategy Forum is to provide a venue for biotechnology/biological product discussion on relevant CMC issues throughout the lifecycle of a product.

Gaithersburg, MD

Next Generation Lipid-Based Nanoparticles Delivery Summit
2024-08-20 - 2024-08-22
The 3rd Next Generation Lipid-Based Nanoparticles Delivery Summit is the only event dedicated entirely to lipid-based nanoparticle delivery, where we bring together 120+ experts in LNP non-clinical, delivery, particle engineering, and formulation from the likes of Teressa, Sanofi, MediciBio, CureVac. Together, we’re on a mission to revolutionize drug delivery, conquering the intricate challenges of lipid-based nanoparticle delivery beyond RNAs and extending cargos to destinations beyond the liver.
Boston, MA

Genome Engineering: CRISPR Frontiers
2024-08-27 - 2024-08-31
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.
Cold Spring Harbor, NY
https://meetings.cshl.edu/meetings.aspx?meet=crispr&year=24

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
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

Home

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
