A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.
In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants ...
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 ...
Avirmax Biopharma Inc. has begun IND-enabling studies of ABI-201, a gene therapy for dry age-related macular degeneration ...
The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL) is pleased to announce a Request for ...
Symbiosis Pharmaceutical Services has completed the latest inspection by the U.S. FDA of its facilities in Scotland.
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 ...
Pharmaceutical Technology on MSN5d
Inborn errors of metabolism elicit unique challenges for therapy developmentAs Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...
A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine ...
Solid Biosciences reported recent positive data in developing their gene therapy candidate SGT-003. The phase 1/2 INSPIRE ...
A study led by UMass Chan Medical School researchers has demonstrated that a gene therapy to correct a mutation that causes ...
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