Somatic Cell Gene Editing

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

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

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

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

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

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

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/

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/

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

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/

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

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

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

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/

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

Natural History Studies and Registries in the Development of Rare Disease Treatments
2024-05-13 09:00 - 15:00
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

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.

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/

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/

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/

Gene Therapy for Muscular Disorders
2024-04-16 - 2024-04-18
The 4th Gene Therapy for Muscular Disorders Summit in April will focus on gene therapies for muscular disorders to tackle the most pressing challenges and together, seek solutions to realize these novel therapeutic potentials for patients in need.

Learn more at https://genetherapy-muscular.com/

Gene Therapy for Rare Disorders
2024-03-26 - 2024-03-29
Bringing together key figures from industry, regulatory bodies, patient non-profits, payers, and key service providers, this is the field’s foremost meeting for collectively progressing rare gene therapy programs into and through the clinic.
Find more information at: https://genetherapy-conference.com/

Pharma USA 2024
2024-03-26 - 2024-03-27
Join pioneers from Commercial, Marketing, Medical Affairs, RWE, Market Access, Patient Engagement, and key stakeholders, will come together to deliver value to overcome the challenges that threaten the future viability of innovation.
Find more information at: https://events.reutersevents.com/pharma/pharma-usa

Genomics of Rare Disease
2024-03-25 - 2024-03-27
The 18th conference on rare disease combines recent research on genetic mechanisms involved in disease, with advances in approaches for clinical care. This year’s conference will include discussions on the use of a ‘pangenome’ to improve diagnostic yield, the role of common variants in rare disease, and new developments in prenatal and neonatal genomic screening. This highly-interactive meeting fosters knowledge exchange among clinicians, clinical scientists, researchers and bioinformaticians working on rare diseases.
Find more information at: https://coursesandconferences.wellcomeconnectingscience.org/event/genomics-of-rare-disease-20240325/

The Allied Genetics Conference 2024
2024-03-06 - 2024-03-10
The Allied Genetics Conference (TAGC) is a unique conference organized by the Genetics Society of America that offers innovative sessions that get to the heart of our shared research interests. The diverse and interdisciplinary scope of TAGC 2024 allows researchers to discover answers and explore new directions in today’s genetics and genomics research.
Find more information at: https://genetics-gsa.org/tagc-2024/

Rare Disease Day at NIH
2024-02-29
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.
Find more information at https://ncats.nih.gov/news-events/events/rdd

Gene Therapy Comparability Summit
2024-02-13 - 2024-02-15
The 4th Gene Therapy Comparability Summit will share the lessons learnt by biopharma directly dealing with comparability for their clinical-stage assets. Uniting the key stakeholders tackling comparability across regulatory, process development, CMC, and analytics departments, this summit will ensure that you can establish comparability in your clinical phase of development and prove to regulators that your products are comparable when scaling up for commercialization and navigating process changes.
Find more information at: https://genetherapy-comparability.com/

The State of Cell and Gene Therapy 2024
2024-01-24 10:00 - 15:30
In this free virtual event, Genetic Engineering and Biotechnology News is bringing together people from industry and academia to discuss the latest research developments, innovations, and disruptive technologies that are impacting patients’ lives today and will spur cell and gene therapies bigger and better things tomorrow.
SCGE PI Kiran Musunuru will be speaking at the event. Find more details here: Event summary – Event Page | The State of Cell and Gene Therapy 2024 (liebertpub.com)

Precision Medicine World Conference
2024-01-24 - 2024-01-26
PMWC brings together recognized leaders, top global researchers and medical professionals, and innovators across healthcare and biotechnology sectors to showcase practical content that helps close the knowledge gap between different sectors, thereby catalyzing cross-functional fertilization & collaboration in an effort to accelerate the development and spread of precision medicine.
For more information at: https://pmwcintl.com/

WCBP 2024
2024-01-23 - 2024-01-25
The WCBP Symposium is known as the highest-regarded annual conference addressing the role of current and emerging CMC analytical technology among evolving US and international regulatory perspectives. Hear from and engage with leaders on a wide range of topics including CMC development strategies, innovative analytical characterization, global submission strategies, next-generation therapeutics & vaccines, as well as the challenges and promises of CRISPR-based therapeutics. This year, WCBP will be co-sponsored by the US FDA.
Find more information at https://www.casss.org/wcbp-cmc-north-america/wcbp
