Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was and whether it warranted making the drug available for patients facing a fatal disease with few other options.
As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials. | As other gene editing programs ...
The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.
Funds will help the companies create a platform for the manufacturing and characterization of AAV-based gene therapy vectors.
Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.
NewBiologix, a technology innovation company pioneering efficient, cost-effective, and scalable production of viral vectors for gene and cell therapy, today released a scientific white paper outlining ...
EsoBiotec’s platform has the potential to eliminate the complex and time-consuming process for manufacturing, helping to make CAR-T cell therapies more accessible.
X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in ...
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
A recent study on T cell gene therapy trials shows that while secondary malignancies occur in a small percentage of patients, ...
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