In a new experimental study, scientists from Juntendo University in collaboration with researchers from the University of Tokyo have developed an adeno-associated virus (AAV) vector-mediated ...

Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX ...

Siren said proceeds will go toward investigational new drug (IND)-enabling animal studies and clinical-grade production of therapy. Upon FDA approval of an IND application, Siren plans to start on ...

How to design and launch a successful Clinical Trial Manufacturing.

Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the R75W mutation. Moreover, this gene editing technology ...

The Viral Vector & Plasmid DNA Manufacturing Market Size accounted for USD 5.4 Billion in 2023 and is estimated to achieve a ...

D Molecular Therapeutics, Inc.’s FDMT share price has surged by 5.88%, which has investors questioning if this is right time ...

NEW YORK – In cell and gene therapies, the genetic machinery is key to producing the proteins missing or dysregulated in a disease, but whether the therapeutic cassette reaches target cells and how ...

Adeno-associated viral 'packages' carrying truncated dystrophin genes were injected into the calf muscle of mice unable to produce the dystrophin protein. Functional dystrophin protein was expressed ...