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.
German-based company, SIRION Biotech has signed a two-year development agreement with a clinical-stage ophthalmology company, Acucela, to establish optimised adeno associated virus (AAV) vectors for clinical applications in ocular gene therapy.
The U.S. Food and Drug Administration has approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness.
Cell therapy company jCyte has announced results from a phase 1/2a clinical trial for its investigational product, jCell.
Scientists have shown that a medicine prescribed for severe pain can help preserve vision in a model of severe, blinding retinal degeneration. Now they want to know more about how it works.
Based on the work of Riccardo Sangermano, Mubeen Khan, Stephanie Cornelis, PhD candidates in the group of prof. Frans Cremers in the Department of Human Genetics, Radboudumc, Nijmegen, The Netherlands, a new approach was recently published in the renown journal Genome Research.
ProQR Therapeutics N.V. has announced that the first patient has been dosed in the Phase I / II open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110.
Horama has raised a second fundraising round that will support its lead candidate, a gene therapy for retinitis pigmentosa, through clinical trials.