Malaria remains a significant global health challenge, with approximately 249 million cases reported annually. Despite being ...

RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.

Genezen, a best-in-class gene therapy contract development and manufacturing organization (CDMO), is pleased to announce Dr. Pam Stetkiewicz and Stuart Auerbach have joined its Board of ...

Regenxbio Inc (RGNX) reports strong progress in late-stage programs and maintains a robust cash position to fund operations ...

The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis.

ABBV-RGX-314, administered once within an office setting, can significantly improve disease severity in patients with NPDR.

RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic ...

LentiGlobin (marketed under the name Lyfgenia) uses the same BB305 viral vector as Zynteglo to deliver a working gene to a patient’s blood cells. In July 2021, the European Medicines Agency’s safety ...