Category: Research news

ProQR Usher clinical development programme

April 18, 2018

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 …

Gene Therapy Trials for Best Disease could start in two years

March 27, 2018

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 …

Immune cells in the retina can spontaneously regenerate

March 26, 2018

Immune cells called microglia can completely repopulate themselves in the retina after being nearly eliminated, according to a new study in mice from scientists at the National Eye Institute (NEI). …

Nightstar Therapeutics announces initiation of STAR Phase III Registrational Trial for NSR-REP1 in Choroideremia

March 6, 2018

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 …

MeiraGTx Receives EMA PRIME Designation for Achromatopsia Gene Therapy Candidate

March 5, 2018

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 …

Precision CRISPR repairs blindness in mice

February 14, 2018

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 …

Gene therapy company receives Rare Paediatric Disease Designation for the treatment of Achromatopsia

February 7, 2018

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 …

Blood-Retina Chip research

January 25, 2018

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. The …

GenSight to start clinical testing combination of gene therapy and wearable device

January 11, 2018

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. The UK Medicines …

Agreement formed for development of AAV vectors for ocular gene therapy

January 9, 2018

Sirion Biotech GmbH, a virus-based technology and gene delivery company, announced that it has signed a two-year development agreement with Acucela, a clinical-stage ophthalmology company. Recombinant adeno associated virus (rAAV) …

FDA approves Luxturna gene therapy treatment

December 20, 2017

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 …

jCyte Presents Results of Clinical Testing in RP

December 14, 2017

Cell therapy company jCyte has announced results from a phase 1/2a clinical trial for its investigational product, jCell, in retinitis pigmentosa. Presented by David S. Boyer, MD, at the annual …