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BioWorld - Thursday, January 22, 2026
Home » Topics » Disease categories and therapies » Ocular

Ocular
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Senior eye exam

Medicare administrative contractors take dim view of several glaucoma technologies

June 12, 2023
By Mark McCarty
Beneficiaries in the U.S. Medicare program have access to several technologies and procedures for treatment of glaucoma, but Medicare administrative contractors (MACs) seem to be looking sideways at some of these offerings. Both Wisconsin Physician Services and Palmetto GBA have floated draft local coverage proposals that deem procedures such as goniotomy and the combination of canaloplasty and trabeculectomy to be investigational, suggesting that claims for these and other services and devices will not be paid by these MACs.
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Eye and DNA illustration
Ocular

Launch of Beacon Therapeutics with focus on gene therapies for retinal diseases

June 12, 2023
Beacon Therapeutics Holdings Ltd. has launched with a focus on developing a new generation of...
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AAVantgarde Bio secures $65M series A round for large gene delivery in AAV vectors

June 6, 2023
By Cormac Sheridan
AAVantgarde Bio SrL raised €61 million (US$65 million) in series A funding to take forward two novel approaches to gene therapy that aim to overcome the packaging limits of adeno-associated virus (AAV) vectors. The company plans to move its lead program, in retinitis pigmentosa associated with Usher syndrome type 1b, into the clinic later this year. A second program, in Stargardt disease, is a couple of years behind it.
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Diagram showing parts of the eye
Ocular

IL-17A as a new therapeutic target in glaucoma

June 1, 2023
It has been previously demonstrated that IL-17A plays a proinflammatory role in autoimmune diseases, and it has also been reported that IL-17A may take part in the occurrence and development of neurodegenerative disorders. Due to its association with both immunomodulation and inflammation, in a recent study, researchers from Shanghai Jiao Tong University aimed to investigate the role of IL-17A in the pathological process of glaucoma neuropathy.
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Eye and DNA

Signs of efficacy seen in Coave's gene therapy trial in RP

May 31, 2023
By Cormac Sheridan
Coave Therapeutics aims to move its lead gene therapy program, CTx-PDE6b, for a form of retinitis pigmentosa (RP) into a pivotal trial in 2025 on the back of a phase I/II study, which uncovered a preliminary efficacy signal in one patient subgroup.
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Central nervous system
Biomarkers

MYO9B gene mutations tied to CMT2 and optic atrophy, study finds

May 31, 2023
Charcot-Marie-Tooth disease (CMT) is a group of neuropathies characterized by sensory and motor dysfunction that progress with aging. It is considered that about 60% of the axonal forms of the disease, such as CMT2, remain genetically undiagnosed.
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Gene editing illustration
Ocular

NGGT-001 gene therapy shows efficacy in cell and animal models of Bietti’s crystalline dystrophy

May 30, 2023
Bietti’s crystalline corneoretinal dystrophy (BCD) is an autosomal recessive inherited disease caused by mutations in the cytochrome P450 (CYP) family 4 subfamily V member 2 (CYP4V2) gene, which encodes a polyunsaturated fatty acid (PUFA) hydroxylase dominantly expressed in retinal pigment epithelium (RPE) cells.
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Ocular

OT-004 delivers RPGRIP1 gene and stabilizes visual loss in murine model

May 30, 2023
Mutations in the RPGRIP1 gene are associated with rare retinal dystrophies and most commonly with Leber congenital amaurosis (LCA) type 6, which is characterized by vision loss, among other symptoms.
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Eye and financial charts

Annexon sights BCVA benefit in missed-endpoint GA phase II

May 25, 2023
By Randy Osborne
On the strength of upside in best corrected visual acuity (BCVA) in patients with geographic atrophy (GA), Annexon Inc. officials will meet with the U.S. FDA to talk about what’s next for ANX-007, after the C1q inhibitor missed the phase II trial’s primary endpoint: mean rate of change, or slope, in the GA lesion area compared to sham.
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Illustration of the inside of an eye with macular degeneration
Ocular

SKG-0106 shows durable efficacy in preclinical models of neovascular AMD

May 25, 2023
Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD).
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