Leading the World in Patient-First Genomic Science

It seems that we had a witch in the lab yesterday! She brewed up a lab-safe potion that the whole team enjoyed (from a distance). Happy Halloween! (Thanks Nicole Haas for giving us a little lesson on dry ice!)

We're proud to announce that Eric Kmiec, Ph.D., our chief scientific officer, has been named the Editor in Chief of Gene and Genome Editing, an Elsevier journal! Read more about the developments to come for this prestigious publication below. We're proud to incorporate the expertise of our peers and colleagues in the gene editing community into this journal, and we look forward to shaping its future together.

Yesterday, we met with IP attorneys Basil Krikelis and Jeff Saffron from McCarter & English LLP, who talked to us about the intricacies of patent law and what steps it takes for our research to get to the patent office. This is an important step for any research institute, and we want to stay informed about what it takes to make and protect a patent. Thanks for the presentation!

Scientists have long known that cancer cells typically have extra chromosomes, but what exactly was the extra genetic material doing? According to a team at Yale, these extra chromosomes are used to boost tumor growth and could be targeted as a new pathway for cancer treatment.

The collaboration leverages Scribe’s proprietary CRISPR X-Editing technologies and Sanofi’s targeted non-viral delivery technologies for the treatment and prevention of genetic disorders, starting with the in vivo treatment of SCD, an inherited blood disorder thought to affect between 70,000 and 100,000 U.S. residents. While ex vivo therapies require cells collected from a patient to then be genetically modified in the lab and eventually returned to the patient, in vivo therapies use certain methods that directly modify genes within a patient’s cells.

Microorganisms use the CRISPR-Cas system to fight viral attacks. In genetic engineering, the microbial immune system is used for the targeted modification of the gene. Another function of this specialized genomic sequence has recently been discovered: archaea use them to fight parasites. This new discovery could have drastic ramifications for genetic medicine in the future as scientists study the functions of archaea in more detail.

HbF has been the subject of decades of intense research, and thanks to genome engineering it has become possible to switch HbF expression back on by interfering with its regulators. Reactivation of HbF expression has emerged as an attractive and popular strategy to treat beta thalassemia. We look forward to seeing the future of these potential treatments for genetic diseases!

Since its inception in 2012, World Lung Cancer Day has been observed every year on August 1 in order to raise awareness of lung cancer issues and magnify the need for more lung cancer research funding. A slew of events highlighting the importance of support, care, and research dedicated to those living with lung cancer will be observed today. Join to support here:

The small DNA fragments in CRISPR-Cas systems (called 'spacers') are largely unspecified to what virus they actually recognize. New analysis has uncovered what these systems actually correspond to in a bacterial immunity system, giving us more insight into the purpose and ability of CRISPR, which helps us all in the field of genomic medicine.

Last week, we were thrilled to attend the Drexel Univ. School of Biomedical Engineering, Science and Health Systems BioMed Summer Academy for their presentation day! We saw students present exceptional products and treatments for a variety of healthcare problems, from sports injuries to blood clotting, as well as share their experiences with CRISPR in a Box, our novel gene editing teaching tool. We're honored to work alongside Bristol Myers Squibb and Brandywine Realty Trust to bring educational experiences to innovative young scientists, well on their way to discovering solutions and treatments for age-old medical problems. Thanks for having us!

The Drexel BIOMED Summer Academy is featured on 6ABC! Explore the world of Biomedical Engineering in a one-week STEM program at Drexel BIOMED. Gain hands-on ...

The Leadership Delaware Speaker Series offers unique opportunities for young leaders and innovators to expand their skills and make connections with Delaware business owners, legislators, and other professionals with new experiences and perspectives. Eric Kmiec, Ph.D., our chief scientific officer, presented last Friday for a class of new students in the program. Check out https://leadershipdelaware.org/to learn more and sign up for the next class of Delaware leaders!

A #CRISPR treatment for sickle cell disease is nearing approval in the US, UK and Europe. This is just the start of cures and treatments to come:

Check out our interview with Dr Kelly Banas from ChristianaCare Gene Editing Institute, where she discusses her groundbreaking research on CRISPR-directed gene editing in cancer treatment. 🧬 Want to learn more about the latest developments in cell and gene therapy? Register now to receive our report! 👉🏼 https://bit.ly/3Mst7cn Supported by Advanced Cell Diagnostics, Inc. #celltherapy #genetherapy #CRISPR

A new study shows that gene editing mechanisms like CRISPR, originally found in prokaryotes, is "present across all kingdoms of life." This discovery could lead to more precise gene therapy, because in eukaryotes, the systems are more easily delivered.

Join our CEO, Eric Kmiec, at a conference that explores gene editing and how Philadelphia-area researchers and companies are helping advance the field of medicine through its myriad applications. The event features a power breakfast and a panel discussion of the facts and fictions of gene editing and how companies in the greater Philadelphia area are already applying gene editing to patient treatment. https://www.bizjournals.com/philadelphia/event/169077/2023/gene-editing-where-are-we-where-are-we-going

Amanda Hewes, our education program manager, was recently interviewed for Good Morning Delmarva with 47 ABC - WMDT for her work with our education program! She discussed our next-generation gene editing curriculum and the ways that the Gene Editing Institute provides leadership and guidance for women and girls who are interested in STEM. Check out her interview below!

The "PAGE" system, developed by researchers at the University of Pennsylvania, uses peptides to more easily deliver the #CRISPR for gene editing. This could make gene editing faster and easier for less familiar scientists to adopt, making the process more accessible.

Improving access to STEM education will help close the diversity gap in STEM fields. At the Gene Editing Institute, we believe the same - which is why we're innovating to bring #GeneEditing to classrooms.

Jennifer Doudna, a pioneer of #GeneEditing, said last month; "We don't want to slow down the science, but we certainly want to make sure that it's used in a responsible fashion going forward.” Bioethics are a critical part of the conversation: