Qure's AMT-130 shows 80% Huntington's progress reduction, eyeing FDA approval. Click here to read why I rate QURE stock a Buy ...

Boehringer Ingelheim, IP Group, the UK Respiratory Gene Therapy Consortium (GTC) 1 and OXB, 1 today announce the start of LENTICLAIR TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, ...

Ionis relies on upfront payments and licensing fees from partners, as well as Spinraza royalties, to drive revenue today, but its rapidly advancing portfolio should help diversify revenue. 2025 is ...

Krystal Biotech KRYS reported fourth-quarter earnings per share of $1.52, which beat the Zacks Consensus Estimate of $1.29 as ...

Spotlight Therapeutics, a Bay Area biotech company that raised $66 million from investors, including GV, has shut down.

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their ...

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic deficiency. Scientists at UCL and Moorfields Eye Hospital successfully injected ...

Pfizer will end global development of its bleeding disorder gene therapy, Beqvez, the Nikkei reported on Thursday.

The evolution of cell and gene therapy is reshaping drug discovery, providing new hope for rare diseases and cancer through innovative gene editing approaches.

Researchers have identified a gene therapy that could prevent life-threatening arrhythmias by restoring the heart’s ...

Cardiac arrhythmias affect millions across the world and are responsible for a fifth of all deaths in the Netherlands.