uniQure Appoints Kylie O'Keefe as Chief Customer and Strategy Officer to Lead AMT-130 Commercialization for Huntington's Disease
uniQure has appointed Kylie O'Keefe as Chief Customer and Strategy Officer to lead the global commercialization strategy for AMT-130, the company's investigational gene therapy for Huntington's disease.
Kennedy Pledges Accelerated FDA Approvals for Rare Disease Therapies to Maintain US Biotech Leadership
Health Secretary Robert F. Kennedy Jr. announced plans to fast-track FDA approvals for rare disease treatments and remove regulatory obstacles during a meeting focused on cell and gene therapies.
uniQure's Gene Therapy AMT-260 Achieves 92% Seizure Reduction in First Drug-Resistant Epilepsy Patient
uniQure's investigational gene therapy AMT-260 demonstrated a 92% reduction in seizure frequency in the first patient treated for drug-resistant mesial temporal lobe epilepsy during five months of follow-up.
AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR
The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine.
uniQure Launches Public Offering to Advance Gene Therapy Pipeline
uniQure N.V., a pioneer in gene therapy, has initiated an underwritten public offering of ordinary shares and pre-funded warrants, with Leerink Partners serving as the lead bookrunner.
uniQure's Fabry Disease Gene Therapy AMT-191 Advances as First Patient Cohort Completes Enrollment
uniQure has completed enrollment for the first cohort in its Phase I/IIa trial of AMT-191 gene therapy for Fabry disease, with safety review showing no significant concerns.
uniQure's AMT-162 Gene Therapy for SOD1-ALS Advances to Second Dose Cohort
uniQure's AMT-162, a gene therapy for SOD1-ALS, has received the green light from the IDMC to proceed with enrollment in the second dose cohort of its Phase I/II EPISOD1 trial.
Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years
Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B.
Huntington's Disease: Optimism Surrounds Potential Breakthroughs in 2025
uniQure's gene therapy trial has shown promising results, with treated patients exhibiting a significant reduction in the rate of Huntington's disease progression.
FDA Lifts Clinical Hold on uniQure's Hemophilia B Gene Therapy
The FDA lifted the clinical hold on uniQure's etranacogene dezaparvovec (AMT-061) after reviewing a liver cancer case in the HOPE-B trial.