Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Pharmaceutical Technology

JANUARY 10, 2023

“Through this collaboration, we have an opportunity to advance AskBio’s gene editing and nonviral delivery efforts, which support and align with our commitment to serving as an industry-leading engine of gene therapy innovation.”. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

XTalks

FEBRUARY 25, 2025

Meanwhile, Vivet Therapeutics is advancing an investigational gene therapy candidate, VTX806, which employs an adenoassociated virus (AAV) vector engineered to reinstate CYP27A1 enzymatic activity.

FDA Approval 59

FDA Approval Gene Therapy Gene Genetics 59

pharmaphorum

OCTOBER 4, 2022

US-based LogicBio is a clinical-stage genetic medicine company focused on genome editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood. As per the agreement, Alexion will initiate a cash tender offer through a subsidiary to acquire all outstanding shares of LogicBio for $2.07

Gene Editing 64

Gene Editing Gene Genome Genomics 64

Scienmag

JANUARY 13, 2021

Study demonstrates the efficacy and therapeutic potential of select ionizable lipid nanoparticles for delivering mRNA to treat genetic diseases before birth Credit: Children’s Hospital of Philadelphia Philadelphia, January 13, 2021–Researchers at Children’s Hospital of Philadelphia and the School of Engineering and Applied Science (..)

Genetic Disease 40

Genetic Disease Research Engineer Genetics 40

The Pharma Data

JULY 6, 2021

Data will include the final analysis from the phase IIIb STASEY study of Hemlibra® (emicizumab) and updated data from the phase I/II study of SPK-8011, an AAV-based gene therapy in development by Spark Therapeutics (a member of the Roche Group). Roche’s Chief Medical Officer and Head of Global Product Development.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Pharmaceutical Technology

APRIL 24, 2023

Langer’s engineering lab was instrumental in the development of these lipid nanoparticles, playing into his role in co-founding Moderna. Conde says the use of nanoparticle delivery for gene therapies has become more common over recent years. Lipid nanoparticle drug delivery was in the spotlight during the Covid-19 pandemic.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147