RNA-targeting molecule from Arrakis allows muscles to relax in mice with myotonic dystrophy
"Dune"-inspired Arrakis Therapeutics has tested its spice—an RNA-targeting small molecule—in a mouse model of myotonic dystrophy type 1 (DM1), with the drug reversing the prolonged muscle contraction that is characteristic of the disease. Arrakis, based not on a...
Researchers discover cell-type-specific link between alternative splicing and autoimmune disease inheritance
Population-scale 5′ scRNA-seq identified 21 cell types and thousands of alternatively spliced genes per cell type. Credit: Nature Genetics (2024). DOI: 10.1038/s41588-024-02019-8Computational biologists from the National University of Singapore (NUS) have uncovered...
Bluebird and Vertex’s sickle cell gene therapies to kick off new Medicaid access model
The Biden Administration said Wednesday that it has secured agreements with Vertex Pharmaceuticals and bluebird bio for their sickle cell disease gene therapies to be the first in a new access model from the Centers for Medicare and Medicaid Services. The voluntary...
Private Medicare insurers are likely to benefit in Trump’s new administration
Medicare Advantage insurers thrived under the first Trump administration, and it’s expected to happen again now that Trump is returning to the White House and Republicans are taking control of Congress. The Medicare Advantage program is expected to cost taxpayers and...
Cartesian tees up Phase 3 mRNA CAR-T study in myasthenia gravis
Cartesian Therapeutics reported longer-term results from a mid-stage study of its mRNA CAR-T therapy for myasthenia gravis, suggesting that the therapy is durable beyond the initial three-month study period. In 12 patients, the mean decrease at six months in a...
Antibiotic Resistance Can Emerge If Bacteria Lose rRNA Modifications
Antibiotic-driven modifications to ribosomal RNA (rRNA) have been suspected of giving rise to antibiotic resistance. For example, antibiotics can alter methylation patterns in rRNA, changing rRNA structures such that they bind antibiotics less effectively. To study...
[2024/11/22] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
Novartis seeks more bolt-on deals as it purchases neuro startup for up to $1.1B
Novartis is buying gene therapy and neuroscience biotech Kate Therapeutics in a deal worth $1.1 billion in upfront and milestone payments, the Swiss pharma confirmed to Endpoints News on Thursday. And Novartis is not done yet. It is still on the lookout for bolt-on...
Genprex Signs Exclusive License to Additional Gene Therapy Technologies with the University of Michigan for the Treatment of Lung Cancer
License includes Genprex's Reqorsa® Gene Therapy in Combination with ALK-Inhibitors for the Potential Treatment of ALK-Positive Lung Cancer AUSTIN, Texas, Nov. 20, 2024 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage...
Vyriad announces strategic collaboration with Novartis to develop in vivo CAR-T cell therapies
Collaboration will combine Vyriad's lentiviral vector platform and Novartis expertise and leadership in cell therapy innovation ROCHESTER, Minn., Nov. 20, 2024 /PRNewswire/ -- Vyriad, Inc., a clinical-stage biotechnology company developing the next generation of...
Epigenic Therapeutics to Enter Clinical Development of EPI-003: A First-in-Class Epigenetic Inactivator for the Treatment of Chronic Hepatitis B
SHANGHAI, Nov. 19, 2024 /PRNewswire/ -- Epigenic Therapeutics, a leading biotechnology company dedicated to developing next-generation gene modulation therapy, today announced that it has received approval of clinical trial application (CTA) from the New Zealand...
Rocket Pharmaceuticals Announces New England Journal of Medicine Publication of Phase 1 RP-A501 Long-Term Data and Presents at Late-Breaking Scientific Sessions at 2024 American Heart Association Conference
All six evaluable Phase 1 patients with Danon disease are alive and transplant-free up to age 25 years RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and...
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