Sarepta Launches ELEVIDYS Gene Therapy Commercially in Japan
– Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years – Company is eligible to receive...
Tag : gene therapy
Bio-Techne’s Simple Western Used in FDA Approval of ZEVASKYN™ Therapy
Bio-Techne Corporation (NASDAQ: TECH) today announced that its Simple Western™ Technology played a key role in supporting the FDA approval of ZEVASKYN™ (prademagene zamikeracel), the first...
Porton Advanced & EVA Pharma Sign MOU to Expand CAR T-Cell Access
A new era of personalized gene therapy for Leukemia patients in Egypt, Saudi Arabia, and beyond. Porton Advanced Solutions, a global leading cell and gene therapy CDMO...
BioMarin Presents 5-Year Phase 3 Data on ROCTAVIAN® at ISTH 2025
Data from longest and largest hemophilia gene therapy study demonstrates sustained factor VIII expression and bleed control, with 81.3% of individuals remaining off prophylaxis No...
AviadoBio Completes Second Cohort in ASPIRE-FTD Trial for AVB-101
AVB-101 Biomarker Data Readout Expected in 2026 Clinical Milestone Shared at AFTD Annual Caregiver Conference AviadoBio, a pioneering gene therapy company dedicated to developing and...