‘Epigenome editor’ silences gene that causes deadly brain disorders

SCGE researchers developed CHARM, a molecular-editing tool that’s small enough to be delivered to the brain to shut down the production of proteins that cause prion diseases. 

TARGETED Challenge Phase 1 Winners Announced by NIH

The National Institutes of Health (NIH) recently announced the winners for Phase 1 of the TARGETED (Targeted Genome Editor Delivery) Challenge. In Phase 1, there were 54 proposals that outlined solutions to address one of two target areas: Programmable Delivery System for Gene Editing and Crossing the Blood-Brain Barrier. To see a full list of Phase 1 winners, click the button below.

A Closer Look at the CRISPR/Cas9 Gene Therapy for Sickle Cell Disease

The National Center for Advancing Translational Sciences (NCATS) researchers Dr. P.J. Brooks and Dr. Jeanita Pritchett Clay wrote an article discussing the impact of this discovery on the African American community as well as the potential to apply the approach to other diseases.

New Funding Opportunity to Support Phase 2 of the SCGE Program

The purpose of this new funding opportunity is to support IND-enabling studies for the development of a novel in vivo genome editing therapeutic platform (genome editor plus delivery system) for two or more disease indications, using the same genome editor, route of administration, and delivery system.

NIH Launches Phase 2 of the SCGE Program with a Series of New Awards

Join thousands of gene and cell therapy professionals for the ASGCT 26th Annual Meeting in Los Angeles, California, between May 16-20, 2023. Keynote speakers include Jennifer Doudna, PhD, a 2020 Nobel Prize winner, and David R. Liu, PhD, from the Broad Institute of Harvard and MIT.

NIH Launches the TARGETED Challenge

The National Institutes of Health (NIH) has launched the TARGETED (Targeted Genome Editor Delivery) Challenge to advance genome editing technology by sourcing innovative solutions for delivering genome editors to somatic cells. The Challenge is open to qualified groups or teams from organizations or institutions, particularly those in the genome editing or vehicle delivery fields, and will take place in three phases: Proposal, Preliminary Data, and Final Data, Independent Testing, and Validation.

American Society of Gene + Cell Therapy 26th Annual Meeting

Join thousands of gene and cell therapy professionals for the ASGCT 26th Annual Meeting in Los Angeles, California, between May 16-20, 2023. Keynote speakers include Jennifer Doudna, PhD, a 2020 Nobel Prize winner, and David R. Liu, PhD, from the Broad Institute of Harvard and MIT.

Clinical Update: Graphite Bio Doses First Patient With Potential CRISPR Cure for Sickle Cell Disease

Graphic Bio, a biotechnology startup in San Francisco, dosed their first patient with nulabeglogene autogedtemcel (nula-cel), a CRISPR therapeutic strategy for treating sickle cell disease (SCD). The dose was performed as part of Phase 1/2 of the CEDAR trial, a multi-center, open-label study designed to evaluate the safety, efficacy and pharmacodynamics of the new therapeutic candidate in approximately 15 adult and adolescents with severe SCD.

The SCGE Consortium Launches Their Public-Facing Toolkit

The SCGE Consortium officially launched their public-facing Toolkit, a platform housing data generated by labs across all consortium initiatives. Users can explore and build upon both published and unpublished data from SCGE researchers such as validation of novel delivery systems and studies on the biological effects of genome editing. Check it out!

Verve Therapeutics Doses First Human with an Investigational In Vivo Base Editing Medicine, VERVE-101, as a Potential Treatment for Heterozygous Familial Hypercholesterolemia

Verve Therapeutics, a biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced that the first patient has been dosed with VERVE-101, in its heart-1 clinical trial.

Notice of Change to Application Due Date for RFA-RM-22-016 “Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3, Clinical Trial Required)”

Key dates have changed for due dates for SCGE Phase 2 funding opportunity “Platform Clinical Trials of Genome Editors in Multiple Diseases (UG3/UH3, Clinical Trial Required).” LOIs are now due October 7, 2022 and full applications are due November 20, 2022.

Nobel Prize Co-Winner Dr. Jennifer Doudna Reflects on 10 Years of CRISPR

In 2020, Dr. Jennifer Doudna and Dr. Emmanuelle Charpentier received the Nobel Prize in Chemistry for their transformative work with CRISPR/Cas9 gene editing engineering. 10 years after CRISPR’s debut, Dr. Doudna interviews with STAT News to reflect on the future of CRISPR.

NIH Common Fund Announces Phase II of the SCGE

The NIH Common Fund has announced new funding opportunities to launch Phase II of the Somatic Cell Genome Editing (SCGE) Consortium. Phase II will aim to continue the consortium’s success in gene editing research and ultimately develop gene therapy treatments to treat genetic disorders.

SCGE Consortium Launches Nature Collection

The SCGE Consortium has launched their Nature Collection page highlighting publications on gene editing research and accomplishments made by consortium members.

Chroma Medicine Launches $125M Financing Plan to Deliver Epigenetic Editing Research

Boston-based biotech company Chroma Medicine has launched its $125 million plan to finance epigenetic editing with the support of gene editing experts, including SCGE PI Dr. David Liu of Broad Institute.

Jennifer Doudna’s Biotech Company Mammoth Bio Partners with Vertex Pharmaceuticals to Invest in Cas Enzymes for Gene Therapies

CRISPR pioneer Jennifer Doudna and her biotechnology company Mammoth Bio will receive up to $650 million in future payments to use Mammoth Bio’s ultracompact Cas enzymes for creating gene editing therapies. 

SCGE Consortium Marker Paper Published in Nature

The NIH CommonFund  and the SCGE Consortium is proud to present our published marker paper discussing the central goals and hopes for the consortium in advancing gene editing.  The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach.

Francis Collins & David Liu Paper Accepted in Nature

Base-editing successfully treats Progeria in mice. Correcting the mutation that causes progeria with base editing leads to strong symptom reduction and longer lifespan in an animal model.

Nobel Prize in Chemistry Awarded to Emmanuelle Charpentier and Jennifer A. Doudna

Emmanuelle Charpentier and Jennifer A. Doudna have discovered one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.

Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors

Different approaches are used in the production of recombinant adeno-associated virus (rAAV). The two leading approaches are transiently transfected human HEK293 cells and live baculovirus infection of Spodoptera frugiperda (Sf9) insect cells. Unexplained differences in vector performance have been seen clinically and preclinically.

New Insights into How COVID-19 Causes Heart Damage

COVID-19 was initially identified as a respiratory disease, but scientists now appreciate that it also affects several other organs in the body, including the heart.

mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells

Mesenchymal stem cells (MSCs) are widely used in regenerative medicine and tissue engineering and delivering biological molecules into MSCs has been used to control stem cell behavior.

CRISPR-CasΦ from huge phages is a hypercompact genome editor

Compact defense system in bacteriophages

The CRISPR-Cas system, naturally found in many prokaryotes, is widely used for genome editing. CRISPR arrays in the bacterial genome, derived from the genome of invading viruses, are used to generate a CRISPR RNA that guides the Cas enzyme to destroy repeat viral invaders. Recently, an unexpectedly compact CRISPR-Cas system was identified in huge bacteriophages.

Search-and-replace genome editing without double-strand breaks or donor DNA

10/21/2019 – Congratulations to the Lui Group on the publishing of their exciting new article on prime editing which is a versatile and precise gene editing tool which was published today in Nature.

Please read the commentary by Heidi Ledford titled “Super-precise new CRISPR tool could tackle a plethora of genetic diseases” here.

Mary E. Shimoyama, PhD, associate professor of biomedical engineering, passed away on Feb. 19. She was 67.

Dr. Shimoyama has been involved with MCW’s Rat Genome Database since she first began working here in 2000, helping it evolve into the leading resource for rat genomic, genetic, phenotype and disease data. She earned her PhD in biomedical informatics from the University of Wisconsin-Milwaukee in 2011 and became an assistant professor of surgery at MCW. In 2016, she was promoted to associate professor in the Marquette University and MCW Joint Department of Biomedical Engineering. Dr. Shimoyama was exceptionally, and justifiably, proud of becoming the principal investigator for the Rat Genome Database and leading it through a recent competitive renewal. She was also honored to be the Vice Chair of Research and Clinical Affairs for the department this past year.

NIH Awards $89 Million for Additional Projects to Advance Genome Editing

10/1/2019 – Genome editing is a promising technology that could offer new treatments or cures for diseases, but challenges remain. To help address them, the National Institutes of Health (NIH) has awarded 24 additional grants to researchers across the United States and Canada through the Somatic Cell Genome Editing (SCGE) Program.

Nano-Sized Solution for Efficient and Versatile CRISPR Gene Editing

9/17/2019 – If used to make non-heritable genetic changes, CRISPR gene-editing technology holds tremendous promise for treating or curing a wide range of devastating disorders, including sickle cell disease, vision loss, and muscular dystrophy. Early efforts to deliver CRISPR-based therapies to affected tissues in a patient’s body typically have involved packing the gene-editing tools into viral vectors, which may cause unwanted immune reactions and other adverse effects.

NIH supports international moratorium on clinical application of germline editing

3/13/2019 – Today, leading scientists and ethicists from seven countries have called for an international moratorium on the use of genetic editing to modify the human germline for clinical purposes. The call comes in the wake of irresponsible and unethical research in China, in which twins were born after alterations to their DNA before implantation. This unexpected and unwelcome revelation roiled the scientific community and the general public, and crystalized the need for guiding international principles.  Research on the potential to alter the very biological essence of humanity raises profound safety, ethical, and philosophical issues.