News

BioSpace5d
Cell and Gene Therapy Sector Enters ‘Critical’ Period After Marks’ FDA Exit
As tariffs, HHS workforce cuts and the ouster of CBER Director Peter Marks threaten the “lifeblood” of the cell and gene ...
US FDA approves first cell-based gene therapy for rare genetic skin disorder
The U.S. Food and Drug Administration approved Abeona Therapeutics' gene therapy for a rare skin disorder on Tuesday. The ...
Fierce Biotech20h
Vertex discontinues AAV research for gene therapies
Amid a reckoning for the gene therapy field, Vertex Pharmaceuticals has joined a growing list of companies paring back their ...
Gene-editing therapy shows early success in fighting advanced gastrointestinal cancers
Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing ...
First Cell-Based Gene Therapy Approved for Rare Skin Disorder
The US Food and Drug Administration (FDA) has approved the first cell-based gene therapy for a rare genetic skin disorder.
PharmExec2d
Asembia 2025: Therapy Advancements Highlight Growing Cost and Access Concerns
Fran Gregory, VP, emerging therapies, Cardinal Health, discusses the evolving cell and gene therapy landscape, highlighting ...
pharmaphorum3d
Abeona prices rare cell disorder gene therapy at $3.1m
Abeona Therapeutics' Zevaskyn, a cell therapy based on engineered keratinocytes harvested from patients, has been approved in ...
Investigational gene therapy gives children with rare immune disorder a new lease on life
An investigational gene therapy has successfully restored immune function in all nine children treated with the rare and life ...
Fierce Pharma4d
Abeona enters commercial gene therapy arena with FDA nod for Zevaskyn to treat rare genetic skin disease
Persistence has paid off for Abeona Therapeutics and its gene therapy for recessive dystrophic epidermolysis bullosa (RDEB).
Rolling Out3d
Wunmi on life after gene therapy at Sickle Cell Consortium
Wunmi Bakare was born in Nigeria, and growing up, she suffered from severe complications from sickle cell. In her youth, she ...
DutchNews.nl3d
QIAGEN Expands Its Digital PCR Portfolio With New Lentivirus Solutions to Strengthen Cell and Gene Therapy Quality Control
QIAcuity digital PCR kits, assays and protocols for lentivirus-based therapies support quality control in cell and gene ...
In a first for St. Louis, patient completes new gene therapy to cure sickle cell disease
Martin Mwita, 20, is the first patient in St. Louis and among the first across the country to receive a new gene therapy for ...