Sarepta Therapeutics said on Tuesday that a 16-year-old boy died from acute liver failure months after receiving the company's U.S-approved gene therapy for a rare muscular dystrophy. The patient ...
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...
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.
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.
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...
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 ...
Funds will help the companies create a platform for the manufacturing and characterization of AAV-based gene therapy vectors.
Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit that the Swiss pharma ...
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 ...
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