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 Biotech15h
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 ...
Managed Healthcare Executive2d
More Innovative Cell and Gene Therapy Payment Strategies Needed, Expert Says | Asembia's AXS25 Summit
Fran Gregory, PharmD, MBA, VP of emerging therapies at Cardinal Health, talks about the importance of market access strategies in the cell and gene therapy space.
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.
Boy with ‘death sentence’ disease living a normal life after breakthrough gene therapy
A four-year-old boy with a life-threatening immune disease is now living a normal life thanks to a pioneering gene therapy ...
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 ...
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 ...
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).
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 ...