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BioWorld - Friday, December 12, 2025
Home » Topics » Disease categories and therapies » Ocular

Ocular
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Illustration demonstrating structure of the human eye and organization of retinal cells
Genetic/congenital

rAAV2-U1a-hELP1 rescues RGC function in familial dysautonomia

Oct. 20, 2025
No Comments
Familial dysautonomia (FD) is an autosomal recessive neurodevelopmental disorder.
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Ocular

Pyrotech Biotechnology describes new GSDME inhibitors

Oct. 16, 2025
Pyrotech (Beijing) Biotechnology Co. Ltd. has identified gasdermin-E (GSDME) inhibitors reported to be useful for the treatment of genetic diseases, cancer, viral infection, age-related macular degeneration, autoimmune diseases, neurological disorders, renal disorders and cancer therapy-associated disorders.
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Ocular

Guangzhou Runer Ophthalmic Biotechnology discloses RBP4/TTR interaction inhibitors

Oct. 8, 2025
Guangzhou Runer Ophthalmic Biotechnology Co. Ltd. has identified phenyl heterocyclic compounds acting as retinol‑binding protein 4 (RBP4)/transthyretin (TTR) interaction inhibitors.
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Eye, DNA double helix illustration.
Ocular

Complement Therapeutics’ CTx-001 gains IND clearance

Oct. 8, 2025
No Comments
Complement Therapeutics GmbH has obtained clearance for CTx-001, a gene therapy candidate.
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Eye and DNA
Ocular

Seed financing at Cirrus Therapeutics to develop ocular medicines

Oct. 3, 2025
No Comments
Cirrus Therapeutics Inc. has closed an $11 million seed financing to advance its pipeline of gene and cell therapies designed to preserve sight and extend ocular healthspan in patients with chronic blinding diseases.
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Illustration of pink and blue antibodies
Neurology/psychiatric

Chugai’s MR16-1 exerts neuroprotection in NMOSD

Oct. 2, 2025
No Comments
Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.
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Eye chart and teen with laser over eye

Magnum Opus: Street drinks in LCA5 gene therapy phase I/II data

Sep. 30, 2025
By Randy Osborne
No Comments
Opus Genetics Inc. will be sitting down with the U.S. FDA to talk about positive three-month data from the pediatric cohort of its ongoing phase I/II trial called OPGx-LCA5-1001 – partially funded by the agency – evaluating OPGx-LCA5, a gene augmentation therapy for ultra-rare Leber congenital amaurosis type 5 (LCA5). The affliction is a severe form of retinal dystrophy that renders babies blind in the first year of life.
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Ocular

Shenzhen Bay Laboratory describes new compounds for aldehyde metabolism disorders

Sep. 29, 2025
Shenzhen Bay Laboratory has identified compounds reported to be useful for the treatment of cancer, eye, cardiovascular, metabolic, autoimmune, inflammatory, neurological and dermatological disorders.
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Businesswoman pressing dollar sign on touchscreen
Ocular

Iveena’s eye drop for myopia receives NEI funding

Sep. 19, 2025
No Comments
Iveena Delivery Systems Inc. has been awarded a $2 million phase II Small Business Innovation Research (SBIR) grant from the National Eye Institute (NEI) to advance the development of novel topical eye drops to control pediatric myopia and other refractive disorders.
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Close up of eye and vision test

Cornea field growing nicely; harvest ahead for Kala?

Sep. 18, 2025
By Randy Osborne
No Comments
As Wall Street awaits phase IIb data from Kala Bio Inc. with KPI-012 in persistent corneal epithelial defect, odds are being weighed for that candidate – and others in development – against the lone approved treatment used in a market already worth more than $1 billion.
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