Analysts did not seem very concerned by the treatment-related serious adverse event, noting that NGN-401 was well-tolerated ...

An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics ...

Diamond-Blackfan anemia (DBA) is a potentially life-threatening condition that can cause severe anemia and other abnormalities. It's a rare genetic blood disorder that occurs when the bone marrow ...

Ocugen, Inc. (NASDAQ: OCGN) has reported its financial results for the third quarter of 2024, highlighting significant ...

Neurogene shares tumbled 35% last night after it disclosed that a pediatric patient with Rett syndrome experienced a severe ...

New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through regulated GATA1 expression. Notably, it’s one of the first ...

These preclinical findings could lead ... Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding ...

Its lead gene therapy candidate has the potential to be faster, less expensive, and more widely available than current CAR T ...

A 16-month-old boy from a low-income family in Kolkata receives a rare gene therapy for spinal muscular atrophy free of ...

ASTCT® and CIBMTR® Bring Leading Experts Together to Share Groundbreaking Advancements in Hematopoietic Cell Transplantation, Cellular Therapy and Gene Therapy.

To improve long-term persistence and reduce the risk of immune rejection, researchers are exploring ways to diminish the ...

Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture ...