News

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 ...
The American Journal of Managed Care4d
FDA Approves Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa
The main treatment strategies being explored are gene therapy, fibroblast cell therapy, bone marrow stem cell therapy, and protein therapy, with gene therapy appearing the most promising for RDEB.
pharmaphorum4d
bluebird claims first approval for gene therapy Zynteglo in EU
bluebird bio has its first ever product approval, getting a green light from the EMA for Zynteglo, its ex vivo gene ... bone marrow of cells before the cells are reinfused. The one-shot therapy ...
pharmaphorum6d
Regenxbio expands MPS I gene therapy trial after first look at data
Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited ... chemotherapy to destroy the patient's bone marrow. The latest readout is a boost for ...
First patient to receive gene therapy treatment developed at Children's Hospital of Philadelphia living pain-free
The first patient to receive a new gene therapy at the Children's Hospital of Philadelphia is now living pain-free for the ...
Reuters4d
US FDA approves first cell-based gene therapy for rare genetic skin disorder
April 29 (Reuters) - The U.S. Food and Drug Administration approved Abeona Therapeutics' (ABEO.O), opens new tab gene therapy for a rare skin disorder on Tuesday. The gene therapy, called Zevaskyn ...
Express & Star5d
Bone marrow tests ‘double survival time for blood cancer patients’, trial finds
A highly sensitive test that detects traces of disease in the bone marrow of patients with a rare and aggressive type of blood cancer could help double their chances of survival, a first-of-its ...
MedCity News4d
FDA Approves Abeona Gene Therapy for Healing Wounds From Rare, Inherited Skin Disease
In RDEB, the most severe form of EB, both copies of the COL7A1 gene are mutated. Cell therapy first reached the market as highly personalized cancer treatments made by engineering a patient’s ...
manilatimes4d
Shape Therapeutics Announces Multiple Oral and Poster Presentations at the American Society of Gene & Cell Therapy 28th Annual Meeting
Data support potential of innovative gene therapy using novel RNA editing, paired with targeted AAV.ai delivery and scalable manufacturing platforms SEATTLE, April 28, 2025 (GLOBE NEWSWIRE) -- Shape ...
manilatimes4d
ElevateBio Announces Presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
April 28, 2025 (GLOBE NEWSWIRE) -- ElevateBio, a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced presentations at the upcoming ...
Business Wire5d
Neurogene Announces Upcoming Oral Presentation on Monitoring and Treatment to Reverse Rare Complication of High Dose Gene Therapy at the ASGCT Annual Meeting
“Given the limited published research on HLH related to gene therapy, we believe it is important to provide information to the AAV gene therapy community on effective approaches to ...