Industry News

  • Tuesday, November 21, 2017
    Based on the work of Riccardo Sangermano, Mubeen Khan, Stephanie Cornelis, PhD candidates in the group of Professor Frans Cremers in the Department of Human Genetics, Radboudumc, Nijmegen, the Netherlands, a new approach was recently published in the renowned journal Genome Research which enables the systematic and quick testing of putative causal sequence variants for their effect on RNA splicing and, thereby, on the protein.
  • Thursday, October 12, 2017
    Investigational LUXTURNA has the potential to be both the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United States First adeno-associated viral (AAV) vector investigational candidate to reach this stage of FDA review Investigational LUXTURNA clinical program includes up to four years of efficacy data, with observation ongoing
  • Monday, November 13, 2017
    ProQR Therapeutics N.V. today announced that the first patient has been dosed in the Phase 1/2 open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110.
  • Thursday, August 24, 2017
    A Symposium in honor of Dr. Gustavo Aguirre, Recipient of the 2017 Proctor Medal, Association for Research in Vision and Ophthalmology.
  • Thursday, August 10, 2017
    Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company's gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
  • Wednesday, January 25, 2017
    RAREDISEASEDAY.ORG With research, possibilities are limitless! Unfortunately, many people living with a rare disease are still unable to find the answers and medical solutions they are looking for. Often this happens because there is little or no research into the peson's particular condition. Those of us concerned with Inherited Retinal Diseases (IRDs) are lucky that so much work is on-going in this area. However, we must remember that with many genes yet to be discovered, support for basic research remains critical. On Rare Disease Day 2017 Retina International will work with its memebrs and collabroators gloably to highlight the importance of continued investment in retinal research.
  • Monday, June 13, 2016
    Arix Bioscience Limited (Arix Bioscience), a global healthcare and life science company supporting medical innovation, announced today it has provided development capital to OptiKira, LLC (OptiKira), an early stage company developing drugs to help prevent blindness.
  • Thursday, January 12, 2017
    Harrington Discovery Institute at University Hospitals in Cleveland, Ohio – part of The Harrington Project for Discovery & Development – has announced its 2016 partnership Scholars selected in collaboration with Alzheimer’s Drug Discovery Foundation (ADDF), and Foundation Fighting Blindness and University of Oxford.
  • Thursday, January 26, 2017
    Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596) committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide, announced today that on January 25, 2017, the first patient has enrolled in a study to evaluate Acucela’s leading drug candidate, emixustat hydrochloride (“emixustat”) in subjects with macular atrophy secondary to Stargardt disease.