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Home » Topics » Disease categories and therapies » Ocular

Ocular
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Ocular

Gene editing in iPSCs corrects inherited retinal dystrophies

Feb. 25, 2025
Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a recent publication, researchers from the Institute of Ocular Microsurgery applied clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and transcription activator-like effector nucleases (TALEN) gene-editing tools to precisely correct induced pluripotent stem cell (hiPSC) lines derived from IRD patients.
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Close-up of toddler eyes

Meiragtx’s gene therapy shows ‘exceptional’ data for blind children

Feb. 21, 2025
By Nuala Moran
Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods.
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Close up of eye and vision test
Ocular

Perceive Pharma secures Series A for ophthalmic diseases

Feb. 19, 2025
Perceive Pharma Inc., a spin out from Perceive Biotherapeutics Inc., has closed a $15 million series A funding round to help it advance first-in-class therapies to prevent vision loss in glaucoma, and additional undisclosed disease areas.
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Ocular

Fudan University patents new SMS2 inhibitors

Feb. 17, 2025
Fudan University has disclosed phosphatidylcholine:ceramide cholinephosphotransferase 2 (SGMS2; SMS2) inhibitors reported to be useful for the treatment of cancer, atherosclerosis, dry eye, pulmonary edema, fatty liver, obesity, type 2 diabetes and Alzheimer’s disease.
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Ocular

Horizon Therapeutics synthesizes new IGF-1R inhibitors for Graves’ ophthalmopathy

Feb. 13, 2025
Heterocyclic insulin-like growth factor 1 receptor (IGF-1R; CD221) inhibitors potentially useful for the treatment of thyroid-associated ophthalmopathy (Graves’ ophthalmopathy) have been disclosed in a Horizon Therapeutics Ltd. patent.
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Liver illustration

Olix wins $630M deal from Eli Lilly for phase I MASH asset

Feb. 11, 2025
By Marian (YoonJee) Chu
Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.
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Eye and DNA illustration

4D’s new data in wet AMD bolster its phase III study vision

Feb. 10, 2025
By Lee Landenberger
4D Molecular Therapeutics Inc.’s gene therapy, 4D-150, in wet age-related macular degeneration (wet AMD) produced positive phase IIb data as the company preps two phase III studies set to begin this year.
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Liver illustration

Olix wins $630M deal from Eli Lilly for phase I MASH asset

Feb. 10, 2025
By Marian (YoonJee) Chu
Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.
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Ocular

RBM47, a new target to watch in thyroid-associated ophthalmopathy

Feb. 10, 2025
Researchers from Huazhong University of Science and Technology have described the potential use of RBM47 as a therapeutic target for thyroid-associated ophthalmopathy, an autoimmune disorder characterized by proptosis, lid swelling, diplopia and compressive optic neuropathy.
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Close up of man's eye

Qlaris’ QLS-111 reduces eye pressure in two phase II trials

Feb. 5, 2025
By Jennifer Boggs
Touting its novel approach for treating glaucoma, Qlaris Bio Inc. reported promising top-line data from two phase II studies testing QLS-111 in patients with primary open-angle glaucoma and ocular hypertension, showing the drug met all primary and secondary endpoints, reducing intraocular pressure with a clean safety profile that could encourage patients to remain on treatment.
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