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Gene silencing and gene editing: Page 2
Genevant Sciences forges $114M deal with Tome Biosciences on liver disease gene therapy
By
LabPulse.com staff writers
Biotech firm Genevant Sciences will collaborate with biotech startup Tome Biosciences on an in vivo gene editing treatment for a rare liver disorder.
January 16, 2024
IGI, Danaher launch CRISPR gene therapy research center
By
LabPulse.com staff writers
Danaher and the Innovative Genomics Institute (IGI) have launched a collaborative research center aimed at developing CRISPR-based gene therapies for a wide array of diseases.
January 12, 2024
Gene therapy, companion diagnostic hit milestones for hemophilia A
By
Liz Carey
Six months after the FDA approved a one-time gene therapy for severe hemophilia A reportedly priced around $2.5 million per patient, the Center for Inherited Blood Disorders, a 340B treatment center in California, has announced the drug's first infusion.
January 11, 2024
Tome Biosciences acquires Replace Therapeutics in $65M deal
By
LabPulse.com staff writers
Less than a month after Tome Biosciences emerged with $213 million in funding, the gene editing company will acquire Replace Therapeutics for $65 million in upfront and near-term milestones, with a total deal value of up to $185 million.
January 5, 2024
KSQ Therapeutics receives FDA clearance for trial of CRISPR-Cas9 engineered tumor therapy
By
LabPulse.com staff writers
Biotech firm KSQ Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with a phase I and II trial of KSQ-001EX, KSQ’s CRISPR-Cas9 engineered tumor-infiltrating lymphocyte program.
November 29, 2023
U.K. regulators approve 'world-first' sickle-cell gene therapy
By
Matt Limb
U.K. medical regulators have approved a “world-first” gene therapy that aims to cure two blood disorders: sickle cell disease and transfusion-dependent β-thalassemia. They are the first regulatory body to approve the treatment.
November 20, 2023
U.K. trial approved for Hunter syndrome gene therapy
By
Matt Limb
U.K. regulators have approved the “first ever” trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a rare lysosomal storage disorder.
November 9, 2023
CRISPR Therapeutics to present data from gene therapy programs at AHA Scientific Sessions
By
LabPulse.com staff writers
Biopharmaceutical company CRISPR Therapeutics will present preclinical data from its investigational gene therapy treatment programs for cardiovascular disease at the AHA Scientific Sessions 2023 in Philadelphia on November 11.
November 6, 2023
Vertex Pharmaceuticals, CRISPR Therapeutics gene-editing therapy to undergo FDA advisory committee review
By
LabPulse.com staff writers
An FDA advisory committee is scheduled to meet on October 31 to discuss and make recommendations on Vertex Pharmaceuticals' and CRISPR Therapeutics’ CRISPR-Cas9 gene-editing-based therapy, exagamglogene autotemcel.
October 30, 2023
Software reduces DANGER of CRISPR-Cas9 by shedding light on off-target edits
By
Nick Paul Taylor
A new software can improve the safety and utility of CRISPR-Cas9 gene editing by detecting off-target changes and eliminating the need for reference genomes, according to a paper published in
Bioinformatics Advances
.
October 27, 2023
CRISPR-Cas3 technique restores dystrophin function in iPSCs from DMD patients
By
Maryam Payne
Researchers have developed a dual CRISPR-Cas3 gene editing technique to restore dystrophin function in induced pluripotent stem cells (iPSCs) from patients with Duchenne muscular dystrophy (DMD).
August 24, 2023
Messenger RNA shows preclinical promise as in vivo gene editing treatment for sickle cell
By
Nick Paul Taylor
The delivery of the editing system resulted in the near-complete correction of hematopoietic sickle cells. A preclinical test linked the approach to a 91.7% increase in functional hemoglobin and a significant fall in the number of sickled cells.
August 14, 2023
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