[2024/11/8] 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...
Gene Therapy Discovery: Stem Cells Tailor Their Behavior to Specific Genetic Diseases
Researchers at the RCCS San Raffaele Scientific Institute in Milan have uncovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the specific genetic disease being treated, providing critical insights for advancing...
Sarepta scraps a Duchenne drug as gene therapy sales rise
Dive Brief: Sarepta Therapeutics is giving up on an experimental drug for Duchenne muscular dystrophy as sales rocket for its approved gene therapy for the disease. The decision to discontinue SRP-5051 was based on several factors, company executives told analysts on...
Research team discovers a new mechanism in cancer cell immune evasion
Graphical abstract. Credit: Cell (2024). DOI: 10.1016/j.cell.2024.09.048An international team led by Goethe University Frankfurt has identified an intracellular sensor that monitors the quality of so-called MHC-I molecules, which help the immune system recognize and...
Hospital-Based CGT Production the Best Option for Patients
Manufacturing cell and gene therapies at hospitals in dedicated, GMP-accredited facilities would overcome many of the technical and logistical challenges associated with decentralized production. So says Na Kyung Lee, PhD, from Sungkyunkwan University in Seoul, Korea,...
FDA Grants First Clearance for CRISPR/Cas13 RNA-Editing Therapy HG202 for Macular Degeneration
SHANGHAI and CLINTON (NJ), November 4, 2024 – HuidaGene Therapeutics, a global clinical-stage biotechnology company focused on genome medicines, has achieved a groundbreaking milestone. The US FDA has cleared its investigational new drug (IND) application for HG202,...
Notch Therapeutics Signals a New Era in CAR-T Therapies
— The T cell therapy company heads to ASH 2024 next month to introduce its novel allogeneic protection and cytokine engineering strategies to support the development of potent and persistent off-the-shelf UNi-T cells — Notch Therapeutics, Inc., a biotechnology company...
Gene Therapy Progress in Alzheimer’s Treatment: Lexeo Therapeutics’ APOE4-Associated Alzheimer’s Trial Shows Promise
Lexeo Therapeutics has announced promising interim results from its Phase I/II trial of LX1001, an innovative gene therapy targeting Alzheimer’s disease (AD) in patients with the APOE4 genetic variant. If successful, LX1001 could become the first approved gene therapy...
The Golden Age of Medicine: Advancements in Novel Modalities and Therapeutic Areas
We are witnessing a “golden age” for medicine, marked by breakthroughs in novel treatment modalities. While monoclonal antibodies (mAbs) have become routine, newer platforms like mRNA, antibody drug-conjugates (ADCs), and microbiome-based therapies are gaining...
Direct RNA Sequencing Supports Novel Discoveries in RNA Biology
Years of experiments and databases filled with RNA-seq results belie the simple reality that, until fairly recently, it was impossible to analyze RNA directly. The RNA-seq studies performed with conventional sequencing platforms are based entirely on cDNA, an...
Navigating the RNA Lipid Nanoparticle Landscape
The success of mRNA vaccines has sparked significant interest and investment in using mRNA technology to treat a broad range of diseases, including cancer, infectious diseases, and rare conditions. Yet when “naked” RNA is injected, it degrades quickly. To address this...
Preclinical Studies Highlight Novel Gene Therapy for IgA Nephropathy Treatment
San Diego, CA – October 28, 2024 At the recent ASN Kidney Week 2024 (October 23–27), researchers presented promising preclinical data on PS-002, an innovative gene therapy aimed at treating IgA nephropathy (IgAN), an autoimmune kidney disease. The therapy, developed...
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