Scientists have discovered that the internal shape of tiny drug-delivery particles—called lipid nanoparticles—has a big ...

Researchers successfully developed a platform that utilizes microbubbles to deliver gene-targeted nanoparticles capable of ...

Sarepta’s shares crashed 41% in pre-market trading Monday morning to $21.01 after the biotech reported a second death from ...

Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

SpliceBio's lead in-house pipeline project is in Stargardt disease, an IRD with no approved therapies that is caused by ...

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

Boyer discusses a potential treatment for diabetic retinopathy and macular degeneration using suprachoroidal injections, ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...