MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

Scientists Receive $25 Million to Develop a Vision-Restoring System that Connects to the Brain

Labels in English are (clockwise): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer

Foundation Fighting Blindness and 4D Molecular Therapeutics Partner to Boost Retinal Gene Therapy Development

Promising gene therapies for inherited retinal diseases (IRDs) — many of which have moved into clinical trials — use viral delivery systems (aka vectors) to get copies of the therapeutic gene to the retinal cells that need them. Viruses are well suited when used for gene delivery, because they’re adept at penetrating cells with their genetic cargo. While some viruses in their natural state can cause illness, therapy developers remove the native genes and replace them with those that are therapeutic.

FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

SparingVision Formed to Advance Sight-Saving Protein for RP

L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance

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