Applied Genetic Technologies Corporation has announced that the first patient was dosed in its Phase 1/2 clinical trial evaluating the safety and efficacy of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP).
ProQR is a young biotech firm based in the Netherlands that is in the early stages of planning a number of clinical trials targeting vision loss in Usher 2A patients (due to mutations in Exon 13 of Ush2A gene).
Immune cells called microglia can completely repopulate themselves in the retina after being nearly eliminated, according to a new study in mice. The findings point to potential therapies for controlling inflammation and slowing progression of rare retinal diseases such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD).
Scientists in the U.S. say human trials of gene therapy for the inherited form of blindness known as vitelliform macular degeneration, or Best disease, could be less than two years away, following successful use of the treatment in a canine model of the disease.
Nightstar Therapeutics PLC, a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, has announced the initiation of the company’s STAR Phase III registrational trial to study the safety and efficacy of NSR-REP1 in patients with choroideremia.
MeiraGTx, a London and New York based gene therapy company, has announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to its gene therapy product candidate A002 for the treatment of patients with achromatopsia due to mutations in the CNGB3 gene.
In genome-editing, the challenge for CRISPR-wielding scientists is to edit only one of the two copies, or alleles, of every gene that people have, repairing the ever-so-slightly broken one and leaving the healthy one alone.
MeiraGTx, a London and New York-based gene therapy company, has announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted rare paediatric disease designation to the company’s gene therapy product candidate AAV2/8-hCARp.hCNGB3 (A002).
Researchers from Microelectronics Institute of Barcelona, Vall d’Hebron Research Institute, and Universitat Autònoma de Barcelona have developed a microfluidic chip that mimics the human blood-retinal barrier in the eye.
GenSight will start a clinical trial in the UK testing a combination of gene therapy and a wearable device to restore sight in patients with retinitis pigmentosa.