Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Data show SPR301 lowered α-synuclein, a hallmark of Parkinson’s disease, in cellular models, indicating potential for efficacy at doses with favorable safety Demonstrated greater α-synuclein ...

Voyager struggles with a negative enterprise value, financial risks, and limited catalysts in a competitive Alzheimer's ...

In this video, Jay Chhablani, MD, professor of ophthalmology at University of Pittsburgh School of Medicine, discusses a preliminary study of combination gene therapy targeting neovascular age-related ...

Final Written Decisions in Pfizer’s IPR2021-00925 and IPR2021-00926 finding all challenged claims of uniQure’s U.S. Patent No. 9,982,248 (“the ’248 patent”) unpatentable (CAFC 23-1404 and 23-1405), ...

Bionova Scientific opened its plasmid DNA production and development facility on May 29 in an effort to provide gene and cell ...

The HHS secretary said people should have access to experimental therapies including unregulated uses of stem cells. But some ...

Scientists have made a key breakthrough for treating respiratory diseases by developing a new drug delivery system that ...

Though some cancer patients lack sufficient enzymes to metabolize capecitabine or 5-FU, many patients are not warned about the risks associated with these treatments.

A Clifton Heights baby treated for a life-threatening genetic disorder with a pioneering gene editing therapy is out of the ...

We have never seen anything close to as good as what we are seeing, in terms of how well we are able to reveal this virus.' ...