The company’s intein-based technology is initially aimed at Stargardt disease, a type of macular degeneration.

The financing will support the ongoing interventional Phase I/II ASTRA trial and the observational POLARIS study of SB-007.

Frontline issues #5 to #8, cover-dated February through May 2003, "Icebound" saw the Joe team trapped inside an Arctic ...

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

Spain’s SpliceBio will use $135 million from a series B round backed by Sanofi and Roche to advance its lead gene editing ...

Researchers at the Johns Hopkins Kimmel Cancer Center and Department of Radiation Oncology and Molecular Radiation Sciences ...

Spanish biotech SpliceBio has secured $135 million in series B financing to accelerate development of its experimental gene ...

Ultimately, Stoke Therapeutics is that classic “intriguing biotech” --hurdling to one of those proverbial “moments of truth” ...

This is a developing news story. Please check back soon for updates. The FDA granted VG801 rare pediatric disease designation ...