Optogenetics: Hope for Vision Restoration for Advanced Retinal Diseases
Early, encouraging results from two human studies — trials launched by Bionic Sight and GenSight — are putting optogenetic therapies in the spotlight for patients with advanced vision loss (i.e., only...
View ArticleOcugen to Launch Clinical Trial for Cross-Cutting RP Gene Therapy
Ocugen, a developer of gene therapies targeting eye diseases as well as a vaccine for COVID-19, has received authorization from the US Food & Drug Administration to launch an 18-participant, Phase...
View ArticleProQR Doses First Patients in Phase 2/3 Clinical Trials for its USH2A-Exon 13...
ProQR, a developer of RNA therapies in the Netherlands, has dosed the first patients in its Phase 2/3 Sirius and Celeste trials for QR-421a, an RNA therapy for people with mutations in the exon 13...
View ArticleReNeuron Not Continuing Clinical Development of Cell-Based Therapy for RP
ReNeuron, a developer of cell-based therapies for retinal diseases and other conditions, has announced that it will not further develop its emerging human retinal progenitor cell (hRPC) therapy for...
View ArticleAldeyra Launches Phase 2 Clinical Trial of Methotrexate Intravitreal...
The biotechnology company Aldeyra Therapeutics has launched a Phase 2 clinical trial of ADX-2191, an intravitreal injection of methotrexate, for people with autosomal dominant retinitis pigmentosa...
View ArticleWebinar for the X-Linked Retinitis Pigmentosa (XLRP) Community
The EL-PFDD meeting will be held on June 7th, 2022. The goal of the June 7th meeting will be to educate FDA staff and other key stakeholders such as biotech and pharmaceutical companies about what it...
View ArticleEndogena Launches Clinical Trial of Therapy to Activate Stem Cells in RP...
Endogena Therapeutics has dosed the first patient in its Phase ½ clinical trial for E-2353, its small molecule designed to activate dormant retinal stem cells in the eyes of patients with retinitis...
View ArticleNanoscope Doses First Patient in Phase 2 Clinical Trial of its Optogenetic...
Nanoscope Therapeutics, a biotechnology company developing gene therapies for retinal degenerative diseases, has dosed the first participant in the Phase 2 STARLIGHT clinical trial of its optogenetic...
View ArticleSparingVision Receives Authorization to Launch US Clinical Trial for its...
SparingVision, a French company developing therapies for ocular conditions including inherited retinal diseases, has received authorization from the US Food & Drug Administration (FDA) to launch a...
View ArticleOpus Genetics Acquires Rights to Gene Therapies for BEST1 and RP (RHO)
Opus Genetics, a company developing gene therapies for inherited retinal diseases (IRDs), has acquired the rights from Iveric Bio for emerging gene therapies targeting autosomal dominant retinitis...
View ArticleVision Improvements Reported for RP Patients in Phase 2B Clinical Trial of...
Nanoscope Therapeutics has reported that 16 of 18 patients with advanced vision loss from retinitis pigmentosa (RP) in the RESTORE Phase 2b clinical trial for its optogenetic therapy were able to...
View ArticleBionic Sight Reports Meaningful Vision Improvements for RP Patients Receiving...
All 12 patients dosed thus far in a Phase ½ dose-escalation clinical trial for Bionic Sight’s emerging optogenetic treatment have demonstrated significant vision improvements. Those receiving the...
View ArticleBudd Tucker Honored with ARVO’s Cogan Award for Advancements in Retinal...
Budd Tucker, PhD, a highly innovative retinal-disease therapy developer and manufacturing expert from the University of Iowa, received the prestigious Cogan Award on April 26 at the 2023 annual...
View ArticleCoave Reports Encouraging Phase 1/2 Clinical Trial Results for PDE6B Gene...
Coave Therapeutics, a French biotechnology company, announced that its gene therapy for people with retinitis pigmentosa (RP) caused by mutations in the gene PDE6B improved visual function at 12...
View ArticleBeacon Therapeutics to Advance XLRP, Cone-Rod Dystrophy, and Dry AMD Gene...
Beacon Therapeutics, a company focused on development of gene therapies for retinal diseases, has been launched with £96 million ($120 million) in investments from Syncona Limited, Oxford Sciences...
View ArticlePYC Doses First Patient in Clinical Trial of RNA Therapy for RP11 (PRPF31...
PYC Therapeutics, an Australia-based developer of RNA therapies, has launched a Phase 1 clinical trial for its RNA therapy known as VP-001 for people with retinitis pigmentosa 11 (RP11) which is...
View ArticleSparingVision Recruiting for Clinical Trial of Cone-Preserving Gene Therapy...
SparingVision, a French company developing therapies for ocular conditions including inherited retinal diseases, has dosed the first cohort of patients at the low dose in its Phase ½ clinical trial...
View ArticleRetinitis Pigmentosa Research Advances
FDA APPROVES SPARK’S VISION-RESTORING GENE THERAPY Spark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first...
View ArticleNew Report: Vitamin A Supplementation Provides No Vision Benefit to RP Patients
A Mass Eye and Ear research team led by Eric Pierce, MD, PhD, director of the Ocular Genomics Institute, and Jason Comander, MD, PhD, director of the Inherited Retinal Disorders Service, has...
View ArticleKiora Reports Vision Restoration in Phase 1/2 Clinical Trial for Photoswitch...
Kiora Pharmaceuticals has announced some vision restoration for participants in ABACUS-1, its Phase ½ clinical trial in Australia for KIO-301, a molecule designed to bestow light sensitivity to retinal...
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