In The News
American Society of Gene + Cell Therapy 26th Annual Meeting Registration is Now Open
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 Users can explore and build upon both published and unpublished data from SCGE researchers such as validation of novel delivery systems and studies on the biological effects of genome editing.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.
Tune Therapeutics Launches to Deliver Epigenomic Platform
Tune Therapeutics of Durham, NC Launches with Pioneering Epigenomic Control Platform to Master Gene Networks, Treat Broad Range of Diseases. Gene editing experts includes SCGE PI Dr. Charles Gersbach of Duke University.
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.
Important COVID-19 Updates from the NIH
Thank you and stay safe!