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 ...

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 ...

CSL will consolidate its R&D teams around six sites located in biotech hubs as the company looks to boost external ...

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

JCR Pharma signs agreement with Alexion for proprietary JUST-AAV capsids to develop genomic medicines: Hyogo, Japan Thursday, July 10, 2025, 10:00 Hrs [IST] JCR Pharmaceuticals Co ...

The biotech will add a pivotal Phase III cohort to its ongoing Phase I/II trial evaluating ATSN-201 in X-linked retinoschisis ...

The Whitrod family launched Genetic Cures for Kids in the hopes of finding a treatment for their daughter with hereditary ...

While gene therapy clinical trials for peripheral artery disease ( PAD) using plasmid DNA or adenoviral vectors show no clear benefit in amputation-free survival, amputation, and all-cause mortality, ...