Genethon and Ampersand team up to engineer next-gen gene therapy vectors with precision

AAV vectors are widely used in gene therapy for their strong therapeutic potential

Rocket Pharma gets FDA nod for first pediatric gene therapy targeting severe LAD-I

The approval covers pediatric patients without a matched sibling donor for stem cell transplant

Lonza expands manufacturing pact with Genetix to meet rising demand for gene therapy ZYNTEGLO

Ultragenyx bags FDA priority review for groundbreaking GSDIa gene therapy

If approved, DTX401 would be the first treatment to address the disease at its root cause

Fresenius Kabi & TQ Therapeutics join forces to accelerate cell & gene therapy manufacturing

The goal of the agreement is to advance cell and gene therapy accessibility by enabling scalable and efficient cell therapy manufacturing

AGC Biologics powers commercial launch of its life-changing gene therapy for rare childhood immune disorder

Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector

Avista Therapeutics unveils AI-powered expansion of gene therapy platform

The company is rolling out ARTEMIS, a next-generation AI platform for capsid design

Complement Therapeutics’ gene therapy gains FDA fast-track for vision loss treatment

CTx001 targets Geographic Atrophy (GA) secondary to Age-related Macular Degeneration

Medera doses final patient in high-dose HFpEF gene therapy trial

Heart failure affects an estimated 64.3 million people worldwide

GenSight Biologics gets early access nod for candidate gene therapy in Israel

Applications include detailed scientific rationale and supporting clinical evidence