Investigating CRB1 Mutations in Retinitis Pigmentosa 12
Sang Yoon Moon, PhD, postdoctoral Research Associate, Lions Eye Institute, discussed research with CRISPRa in human fibroblast cultures.
Reexamining the Outcome Measures Used in Ophthalmic Gene Therapy Trials
Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed the need to reevaluate the goal posts used to determine whether gene therapies for eye indications come to market.
Gene Therapy Voretigene Neparvovec Increases Light Sensitivity in RPE65-IRD via Peripheral Cone Function Improvements
Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed new real world data regarding Spark Therapeutics’ Luxturna that was presented at ARVO’s 2023 conference.
Kenji Fujita, MD, on Clinically Meaningful FST and MLMT Improvements in LCA1 With ATSN-101
The chief medical officer at Atsena Therapeutics discussed updated, 6-month data from a phase 1/2 trial presented at ARVO 2023.
Arshad Khanani, MD, on Future Plans for anti-VEGF Gene Therapy 4D-150
The director of clinical research at Sierra Eye Associates discussed 4D Molecular Therapeutics’ plans for 4D-150 in both wet AMD and diabetic macular edema.
Real World Data Confirm RPE65-IRD Gene Therapy Voretigene Neparvovec Efficacy in Improving Light Sensitivity
Bart P. Leroy, MD, PhD, head, Department of Ophthalmology, Center for Medical Genetics, Ghent University Hospital, discussed the ongoing efforts to verify the efficacy of Spark Therapeutics’ Luxturna.
Arshad Khanani, MD, on 4D-150’s Ability to Address Treatment Burden in Wet AMD
The director of clinical research at Sierra Eye Associates discussed the latest results from a clinical trial evaluating 4D Molecular Therapeutics’ gene therapy, 4D-150, that were presented at ARVO’s 2023 conference.
Corneal Endothelial Cell Therapy Has Potential to Address Unmet Needs for Patients With Corneal Dystrophies
Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech, discussed AURN001, a corneal endothelial cell therapy that was recently approved in Japan, and had a nonclinical data read out at ARVO 2023.
Bart P. Leroy, MD, PhD, on Aligning IRD Gene Therapy Outcome Measures With Activities of Daily Living
The head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital discussed the need to reevaluate requirements for regulatory approval for ophthalmology gene therapies.
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.
Bart P. Leroy, MD, PhD, on Improving Light Sensitivity, Quality of Life With Luxturna
The head of the Department of Ophthalmology at the Center for Medical Genetics, Ghent University Hospital, discussed the main takeaways of a post-marketing study on the gene therapy.
Jay Barth, MD, on Ascidian Therapeutics' Developments in Gene Therapy
CGTLive's sister publication spoke with Barth, the chief medical officer at Ascidian, about the company’s attempts to develop a new method of administration for gene therapy at ARVO 2023.
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.
Luxturna Demonstrates Improvements on Multiple Vision Outcome Measures in Real-World Setting
The study found significant improvements in BCVA, in contrast to several other studies evaluating Luxturna.
Advanced Retinitis Pigmentosa Optogenetic Therapy Demonstrates Vision Function Improvements
The data, presented at ARVO's 2023 conference, also showed a favorable safety profile.
Arnaud Lacoste, PhD, on AURN001‘s Immunomodulatory Properties
The chief scientific officer of Aurion Biotech discussed the properties of the cell therapy for corneal dystrophies, which was recently approved in Japan.
CRISPR/Cas9 Therapy Shows Some Visual Improvements in LCA10
GenSight Biologics’ Lumevoq Demonstrates Clinically Meaningful Improvements in Early Access Programs
The new data builds upon positive long-term follow-up data from clinical trials.
Bart P. Leroy, MD, PhD, on Continuing to Verify Luxturna’s Efficacy
The head of the Department of Ophthalmology at the Center for Medical Genetics, Ghent University Hospital, discussed the results of a post-marketing study presented at ARVO’s 2023 conference.
MT-ND4 LHON Gene Therapy Continues to Yield Vision Improvements
The safety profile of lenadogene nolparvovec at 5-years post treatment was similar to 2-year data.
LCA1 Gene Therapy Improves Sensitivity, BCVA at Highest Dose
No serious TEAEs have been related to ATSN-101 with data up to 6 months after treatment.
Arnaud Lacoste, PhD, on Addressing Unmet Needs in Corneal Dystrophies
The chief scientific officer of Aurion Biotech discussed the investigational cell therapy AURN001.
PDE6A-associated Retinitis Pigmentosa Gene Therapy May Cause Loss of Vision in Some Patients
STZ eyetrial’s treatment rAAV8.hPDE6A was otherwise deemed well-tolerated.
Luxturna’s Safety and Efficacy Profile Supported by Retrospective Data in Patients With RPE65-Associated Inherited Retinal Dystrophy
The commercially available gene therapy, developed by Spark Therapeutics, was evaluated in multiple studies presented at ARVO’s 2023 conference.
Spark Therapeutics’ Luxturna Improves Light Sensitivity in Postmarketing Study of Inherited Retinal Dystrophy
The new positive data on the AAV based gene therapy builds upon real-world data presented at ARVO’s conference in 2022.
Real-World Luxturna Data Shows Good Efficacy, New Adverse Reaction in Retinal Dystrophy
Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.
AGTC-501 Efficacy Maintained in 18-Month Follow Up Data for X-linked Retinitis Pigmentosa
Updated data at ARVO show that AGTC-501 had sustained efficacy and safety at 18 months for patients with X-linked retinitis pigmentosa caused by RPGR mutations.
Advancements in Gene Therapy for Retinal Diseases
Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.
Treatment With OpRegen Shows Improvement in Visual Acuity, Cell Persistence in Dry AMD
New data from cohort 4 showed outer retinal restoration following treatment with OpRegen.
Neutralizing Antibodies to AAV Vector for AMD Gene Therapy May Boost Therapeutic Effect
The post-hoc analysis of the OPTIC trial explored the effects of neutralizing antibodies on patients treated with ADVM-002.
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