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Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on geneediting treatments for rare diseases.
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR geneediting inside the body can be safe and effective, a culmination of years of scientific research.
Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo geneediting treatment for sickle cell disease.
Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in geneeditingresearch in recent years.
The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data.
Formed around research from the Broad Institute's David Liu and Andrew Anzalone, Prime has attracted significant funding from a wide range of high-profile biotech backers.
The biotech raised the Series B round during "probably one of the worst biotech markets" emerging drugmakers have faced in some time, a Metagenomi executive told BioPharma Dive.
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
Cutting edge’ is, for once, a truly apt description when it comes to geneediting – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. That would just slow the whole field down.
Adrian Hernandez In this Friday’s PCT Grand Rounds, Adrian Hernandez of Duke University will present “Precision Health to Population Health: Opportunities and Challenges for GeneEditing Therapies.” ” The Grand Rounds session will be held on Friday, August 2, 2024, at 1:00 pm eastern. Join the online meeting.
The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on geneediting medicines.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA geneediting therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.
Moderna is aiming to build a geneediting franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.
Gene-editing therapy aimed at two targets—HIV-1, the virus that causes AIDS, and CCR5, the co-receptor that helps the virus get into cells—can effectively eliminate HIV infection, new research from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) shows.
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo geneediting programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.
The short history of CRISPR geneediting in humans has, with rare exception, been a history of triumphant progress: A patient apparently cured of sickle cell in 2019, six patients with toxic DNA knocked out of their liver last year, another six patients with a different strand of toxic liver DNA knocked out last week.
The research team has designed four bacterial viruses which use CRISPR technology to kill the unwanted bacteria precisely. The team then engineered the phages through geneediting to improve their targeting ability. A mixture of four of these phages, dubbed as SNIPR001, was found to effectively target the bacteria in biofilms.
Expanding upon the CRISPR-Cas9 geneediting system, researchers at MIT have designed a new technique called PASTE geneediting that can cut out defective genes and replace them with new genes in a safer and more efficient way. How does the GeneEditing Tool PASTE Work?
AMES, IA – Through CRISPR and other gene-editing technologies, researchers and developers are poised to bring dozens – if not hundreds – of new products to grocery stores: mushrooms with longer shelf lives, drought-resistant corn and bananas impervious to a fungus threatening the global supply.
UNSW researchers have used CRISPR geneediting – a type of ‘molecular scissors’ – to understand how deletions in one area of the genome can affect the expression of nearby genes.
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.
While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.
Credit: John Innes Centre A gene which has profound effects on the production of seeds has been identified by researchers from the John Innes Centre. Gene-editing techniques helped to identify and explain the key gene, ZIP4, in wheat which is responsible for maintaining 50% of yield in this global crop.
However, for the vast majority of those mutations, researchers are unsure of how they contribute to cancer because there's no easy way to study them in animal models. Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development.
?Gene therapy research delivers breakthroughs this week with a CRISPR-edited droplet that could lead to a cure for heart disease and the possibility of turning glial brain cells into neurons.
Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Editgeneediting platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
5–7, 2022, is a premier global exchange of the latest scientific advancements, research and evidence-based clinical practice updates in cardiovascular science. The meeting, held in person in Chicago and virtually, Nov.
DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 geneediting to treat mice with Duchenne muscular dystrophy. One company was even bought out for over $200 million. Continue to STAT+ to read the full story…
Using the CRISPR-Cas9 geneediting system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.
Credit: Rasgon Lab, Penn State Researchers who developed an improved method of geneediting for the study of arthropods will expand the technology for use in vertebrate species such as mice, fish and birds after receiving new funding from the National Science Foundation.
He Jiankui, the Chinese biophysicist who created the first gene-edited children , had been quiet since completing a three-year prison sentence in April, leaving many to wonder whether he had plans to return to scientific research. Earlier this month, we got his answer.
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