Nanoscope Publishes Breakthrough Showing MCO-010 Treatment Arrests Neurodegeneration
Nanoscope Therapeutics Inc., a late-stage clinical biotechnology company developing gene therapies for retinal degenerative diseases today announced a breakthrough discovery in Translational Vision Science & Technology (TVST, an ARVO journal) demonstrating that...
[2024/10/18] 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...
GSK sues Moderna over mRNA vaccine patents, seeks ‘reasonable royalty’
GSK on Tuesday unveiled a lawsuit filed against Moderna in Delaware federal court, alleging that its patented inventions provide the “foundation” for Moderna’s mRNA vaccine portfolio. GSK said it’s looking to recover “a reasonable royalty” for Moderna’s tens of...
Gene Therapy Automatically Converts Omega-6 to Omega-3 Fatty Acids in the Body
Shriners Children's Develops New Technology to Prevent Childhood Obesity ST. LOUIS, Oct. 16, 2024 /PRNewswire/ -- According to the Centers for Disease Control, nearly 20% of children and teens are considered obese. Research shows it can have a dramatic impact on a...
Wave Life Sciences Reports Positive Data for First-Ever RNA Editing in Humans
Wave Life Sciences has announced promising data from the RestorAATion-2 trial, marking the first clinical demonstration of RNA editing in humans. The phase 1b/2a study tests WVE-006, an RNA editing oligonucleotide, for the treatment of alpha-1 antitrypsin deficiency...
Improving Gene Therapy Design and Manufacturability Using Synthetic Biology and AI
Broadcast Date: Tuesday, November 12, 2024Time: 8:00 am PT, 11:00 am ET, 17:00 CET For adeno-associated viral gene therapies to scale, scientists must first address safety and manufacturability challenges. Improving payload design and optimizing production systems can...
Key Trends in the Pharmaceutical Pipeline for 2024 — Part I: Transformative R&D Growth Driven by Breakthroughs in Oncology, Neurological, and Metabolic Therapies
The pharmaceutical industry in 2024 is witnessing a rapid transformation driven by advances in biotechnology, precision medicine, and evolving market needs. The industry is set to build upon the research and development (R&D) momentum from previous years,...
MeiraGTx’s AAV Gene Therapy Shows Promising Results for Parkinson’s
MeiraGTx has reported encouraging results from a midphase trial of its investigational gene therapy, AAV-GAD, for treating Parkinson’s disease. The company is now in discussions with regulators to advance the program to Phase III clinical trials.Significant...
ARTHEx Biotech Announces Publication of Promising Preclinical Research for Myotonic Dystrophy Type 1 (DM1)
-Study highlights dual targeting action, demonstrating significant therapeutic potential for treating myotonic dystrophy type 1 (DM1) across a variety of disease severities – VALENCIA, Spain, Oct. 16, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage...
Gene editing approach helps launch first-in-human clinical trial for rare genetic disease
Credit: Unsplash/CC0 Public DomainA collaborative effort between investigators at the National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH) demonstrates the potential of precise genome...
Study explores how traumatic brain injury may be linked to Alzheimer’s disease
Credit: CC0 Public DomainEach year, about 2.5 million people suffer from traumatic brain injuries (TBI), which often increases their risk of developing Alzheimer's disease later in life. Researchers led by The Ohio State University Wexner Medical Center and College of...
Improving the recipe for gene therapy: New treatment for Gaucher disease shows promise
MRI brain scans of mice with Gaucher disease Type III. The scan of a mouse that received the new treatment (left) shows a significant reduction in tissue inflammation (blue) compared to an untreated mouse (right). Credit: Weizmann Institute of ScienceGregory and...
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