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Home » Topics » Disease categories and therapies » Ocular

Ocular
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Transparent capsule with DNA and cells

Gene therapy efforts active, despite low Bluebird, 2seventy bids

March 14, 2025
By Karen Carey
Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value that gene therapies bring to patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field.
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Genetic/congenital

CHK2 inhibition alleviates photoreceptor degeneration in preclinical Bardet-Biedl syndrome

March 13, 2025
Bardet-Biedl syndrome (BBS) is a ciliopathy that leads to progressive blindness due to degeneration of cone photoreceptors, but the mechanisms behind this are not well understood. The syndrome is associated with obesity, polydactyly, cardiovascular disease and skeletal abnormalities, among others, and it is caused by genetic variants in the BBS10 gene in about 40% of the cases.
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Neurotech ECT illustration

Neurotech’s CNTF implant approved for macular telangiectasia

March 6, 2025
By Jennifer Boggs
The first treatment for macular telangiectasia type 2 is set to enter the market following U.S. FDA approval of NT-501 (revakinagene taroretcel) from Neurotech Pharmaceuticals Inc., a privately held company that has been quietly advancing its encapsulated cell therapy platform for more than two decades.
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Close up of eye and vision test
Ocular

Avirmax begins IND-enabling studies with gene therapy for dry AMD

March 3, 2025
Avirmax Biopharma Inc. has begun IND-enabling studies of ABI-201, a gene therapy for dry age-related macular degeneration (AMD). ABI-201 is an AAV vector that delivers three genes to correct the dysregulation of complement activation, to protect retinal pigment epithelia and photoreceptors, as well as to block retinal neovascularization.
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Ocular

Caregen describes new PAC1R agonists for dry eye

March 3, 2025
Caregen Co. Ltd. has identified peptides acting as ADCYAP receptor type I (PAC1R) agonists reported to be useful for the treatment of dry eye.
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vaporized ophthalmic drug delivery
Patents

Verily developing vaporized ophthalmic drug delivery device

Feb. 28, 2025
By Simon Kerton
A recent patent filing from Verily Life Sciences LLC seeks protection for the development of an ophthalmic drug delivery device with sensors and a camera which allow the delivery of a specific or controlled dose of a therapeutic agent in vapor form to a patient’s eye.
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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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