The gene therapy regimen seems to be working wonders, including restoring nearly all hearing to a seven-year-old patient ...

AstraZeneca’s rare disease unit Alexion has penned a $825 million deal for adeno-associated virus (AAV) capsids from existing ...

The Hopewell site is equipped to support clinical-stage development, process-development, and scaleup for cell and gene therapy programs.

Through the program, gene therapies are developed to treat patients with retinitis pigmentosa caused by pathogenic variants ...

In the July 2 release, 4DMT explained that the 25% layoffs are part of a move to “streamline operations to offset additional ...

JCR Pharmaceuticals has signed a licence agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV ...

Beacon Therapeutics has completed the enrolment in its randomised, registrational Phase II/III VISTA trial of laru-zova ...

Technical Report Published: February 2001 Direct delivery of leptin to the hypothalamus using recombinant adeno-associated virus vectors results in increased therapeutic efficacy Cecilia Lundberg ...

Klotho is advancing its ALS gene therapy candidate KLTO‑202 to the manufacturing phase, and is planning the first human ...

Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe ...