Topical Gene Therapy B-VEC Improves Vision in Compassionate Use Program
Krystal Biotech's B-VEC was well-tolerated and the treated patient experienced significant improvement in visual acuity.
Structural Changes Support Gene Therapy's Effect on Geographic Atrophy
New data from imaging analyses on RG6501 were presented at the ARVO 2023 Annual Meeting.
Advanced Retinitis Pigmentosa Optogenetic Therapy Demonstrates Vision Function Improvements
The data, presented at ARVO's 2023 conference, also showed a favorable safety profile.
Preclinical AMD Gene Therapy Shows Potential for Other Retinal Diseases
The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models.
NIH Awards Grant for Engineered Mechanosensitive Channel Glaucoma Gene Therapy
The grant goes toward developing an innovative approach to gene-agnostically and autonomously regulate pressure.
Streamlining Stem Cells to Treat Macular Degeneration
Scientist have proposed several methods for converting stem cells into RPE, but there is still a gap in our knowledge of how cells respond to these stimuli over time.
Gene Therapy Trial for Stargardt Disease Reaches Full Enrollment
Nanoscope Therapeutics’ Phase 2 STARLIGHT open-label trial enrolled 6 subjects with advanced vision loss due to a clinical or genetic diagnosis of Stargardt disease.
Dry AMD Gene Therapy Shows Promise in Preclinical Study
The therapy has demonstrated benefit in multiple models of dry AMD.
Novel Computational Platform scAAVengr Speeds Up Identification of Gene Therapy Viral Vectors
While focused on the retina, the researchers showed the platform works for the identification of AAVs that target tissues in various organs.
Stem Cell Model Developed to Study Albinism-Related Eye Conditions
Researchers will use the model to study how lack of pigmentation affects RPE physiology and function.
Stem Cell-Derived Retinal Implants Demonstrate Long-Term Viability
Investigators did not find any clinical signs of rejection of the mismatched donor cells.
X-Linked Retinitis Pigmentosa Trial Exceeds Enrollment
Applied Genetic Technologies is also developing AGTC-401 and AGTC-402 for the treatment of achromatopsia.
First Patient Dosed in Wet AMD Gene Therapy Trial
The clinical trial of 4D-150 expects to enroll around 60 participants with wet AMD.
First Patients Dosed in Gene Therapy Trial for Usher Syndrome, Retinitis Pigmentosa
Both the Sirius and Celeste phase 2/3 trials are evaluating QR-421a.
Second Pivotal Trial Initiated for Wet AMD Gene Therapy
The ASCENT trial is kicking off while the ATMOSPHERE trial continues to screen patients with wet AMD.
Blue Cone Monochromacy Gene Therapy Gets Orphan Drug Designation
ADVM-062 is designed to deliver a functional copy of the OPN1LW gene to the foveal cones of patients with BCM.
Photoreceptor Precursor Cells' Potential to Treat Inherited Retinal Diseases
Investigators are planning a first-in-man clinical trial for photoreceptor precursor transplant in Singapore.
Stem Cell-Derived Eye Drops Improve Outcomes in Dry Eye Disease
Researchers have developed a corneal epithelial stem cell-derived eye drop therapy for dry eye disease.
IND Accepted for Wet AMD Gene Therapy
4D Molecular Therapeuitcs plans to initiate a phase 1/2 trial of 4D-150 before the end of the year.
New Collaboration to Develop Gene Therapy for Chronic Retinal Diseases
RGX-314 is being developed to target wet AMD, diabetic retinopathy, and other chronic retinal diseases.
CaMKII Enzyme: A New Target for Vision Preservation?
Investigators found that reactivating the CaMKII enzyme could protect against further vision loss.
First Patient With Retinitis Pigmentosa Dosed in Trial of MCO-010
MCO-010 is designed to deliver multi-characteristic opsin to retinal cells.
Optogenetic Gene Therapy Restores Vision in 11 Retinitis Pigmentosa Patients
Nanoscope Therapeutics Inc. expects to advance the therapy by launching a late-stage Phase 2b trial this summer with gene therapy that delivers multi-characteristic opsin to retinal cells.
Optogenetic Treatment Restores Partial Vision in Retinitis Pigmentosa
An international research team has shown that optogenetic therapy has helped to partially regain visual function in a patient with retinitis pigmentosa. This is a milestone towards a gene therapy that could restore vision.
Biogen's BIIB112 Gene Therapy Falls Short in X-Linked Retinitis Pigmentosa
Although the XIRIUS study did not meet its primary end point, positive trends were observed across several clinically relevant prespecified secondary end points.
Collaboration formed to develop AAV-based gene therapies for eye disease
ViGeneron and Biogen this week have announced a global collaboration and licensing agreement in which the partners will develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors that treat inherited eye disease.
FDA grants fourth ODD for treatment of gene mutation-associated retinal diseases
The FDA this week granted the fourth Orphan Drug Designation for a novel gene therapy product candidate (OCU400, Ocugen Inc.) in the treatment of PDE6B gene mutation-associated retinal diseases.
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