Gene Therapy

In a presentation at ARVO 2023 in New Orleans, the company noted data in a nonhuman primate model of non-arteritic anterior ischemic optic neuropathy demonstrates the ability to restore visual function after delivery of a novel gene therapy.

Physician outlines the latest emerging technologies to treat the condition.

A team of researchers has uncovered a mechanism that causes blindness, could lead to targeted therapies.

According to National Eye Institute researchers, the variants generate malformed proteins that alter the stability of the membrane attack complex, which may drive a chronic inflammatory response in the retina.

Ophthalmologist looks ahead amid company’s advances over past 18 months.

According to the company, it is the first approval in the world for allogeneic cell therapy to treat corneal endothelial disease.

Researchers are using a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.

Data for IG-002 show for the first time that a single subretinal administration of a DNA payload encoding the human ABCA4 gene resulted in durable expression of human ABCA4 protein.

The researchers from Indiana University School of Medicine found neurons use mitochondria for a steady source of energy, and restoring mitochondrial homeostasis in the diseased neurons can protect the optic nerve cells from being damaged.

The researchers focused on photoreceptor cells (PRCs), which are light-detecting cells found in the retina.

A look at what’s in the therapeutic delivery pipeline for these disorders.

The 2023 Harrington Prize for Innovation in Medicine recognizes Jean Bennett, MD, PhD, and Albert M. Maguire, MD, for their work in retinal gene therapy for genetic diseases.

Key is to use stem cell treatments to ensure optimal outcomes for patients.

Despite its promise, RGC replacement represents a more formidable challenge.

Three of Frontera’s gene therapy product candidates have entered clinical trials since the beginning of the year.

The results are consistent with those observed, and previously published by the same group, showing benefit in age related macular degeneration models and highlights the potential value of ophNdi1 for multiple eye diseases.

According to the company, ONL1204 is a first-in-class small molecule Fas inhibitor with a mechanism of action designed to provide neuroprotection for retinal cells.

The technique could provide researchers with samples for studying AMD and other eye diseases.

A University of Houston researcher has been awarded a $1.6 million grant to help restore vision loss in patients with the rare disease that causes blindness.

Researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.

Study results show evidence for augmentation, offering hope to patients.

A collaborative team of researchers with Oregon Health & Science University and Oregon State University are using lipid nanoparticles to target light-sensitive cells in the eye.

Viridian Therapeutics unveils positive data from its ongoing Phase 1/2 trial evaluating low dose VRDN-001 in patients diagnosed with TED.

According to the company, gene and protein expression of cytokines and chemokines after latanoprost+benzalkonium chloride treatment administered via the company’s microdose formulation decreased inflammation from preserved glaucoma solutions compared to drops.

FT-001 is administered by a one-time injection into the subretinal space of the eye that delivers a functional copy of the human RPE65 gene to the nuclei of the patient’s retinal cells.