Gene Therapy
9 important updates for World Sickle Cell Day
Experimental gene-based COVID-19 vaccine holds promise for fast development
An experimental gene-based vaccine candidate against SARS-CoV-2 is currently under development at Massachusetts Eye and Ear and Massachusetts General Hospital.
VIDEO: Khanani delves into data from OPTIC trial
Arshad M. Khanani, MD, discusses results from the phase 1 OPTIC trial of ADVM-022 intravitreal injection gene therapy (Adverum Biotechnologies) for the treatment of wet age-related macular degeneration presented at the virtual Association for Research in Vision and Ophthalmology meeting.
Gene therapy appears to reduce sickle cell disease-related complications
LentiGlobin gene therapy led to reductions in sickle cell disease-related complications and hemolysis, according to results of a phase 1/phase 2 trial presented at the virtual American Society of Gene and Cell Therapy Annual Meeting.
Adverum Biotechnologies reports positive interim data from three cohorts of OPTIC phase 1 trial
New interim data for the OPTIC phase 1 dose-ranging clinical trial of ADVM-022 intravitreal injection gene therapy for the treatment of wet age-related macular degeneration showed robust efficacy in each cohort, long-term durability in cohort 1 and the potential for fewer adverse events in cohort 3, according to a press release from Adverum Biotechnologies.
First patient dosed in cohort 4 of Adverum OPTIC trial
Regenxbio gene therapy delivers long-term treatment effect in wet AMD
Anticancer gene therapy regimen confers survival benefit in recurrent glioblastoma
Continued VB-111 monotherapy priming after progression with bevacizumab combination treatment improved survival outcomes among patients with recurrent glioblastoma, according to results of a phase 1/phase 2 study published in Neuro-Oncology.
X-linked retinitis pigmentosa gene therapy granted PRIME, ATMP designations by EMA
The European Medicines Agency has granted priority medicines and advanced therapy medicinal product designations for AAV-RPGR, an investigational gene therapy treatment for X-linked retinitis pigmentosa, according to press releases from MeiraGTx Holdings and Janssen Pharmaceuticals, which are jointly developing the therapy.