Opus Genetics Inc. has entered a strategic partnership with the Global RDH12 Alliance to advance Opus’ gene therapy program for patients with vision loss due to retinol dehydrogenase 12 (RDH12) gene mutations.
Myopia, a leading cause of visual impairment globally, can progress to serious complications like retinal and choroidal degeneration. While atropine is commonly used to manage childhood myopia, its nonselective action on all five acetylcholine muscarinic receptor subtypes (M1-M5 receptors) can cause side effects such as pupil dilation and photophobia.
Graves disease (GD)-associated hyperthyroidism is an autoimmune disorder characterized by the presence of autoantibodies that stimulate the thyroid-stimulating hormone receptor (TSHR), leading to excessive production of thyroid hormones.
In a recent study published in Nature Genetics, a team of scientists used CRISPR-Cas9 gene editing to systematically analyze genetic weaknesses in uveal melanoma cells and comprehensively map monogenic and digenic dependencies.
The U.S. FDA has cleared Aavantgarde Bio Srl’s IND application for AAVB-039, the company’s gene therapy program for Stargardt disease, the most common inherited form of macular degeneration.
Diabetic retinopathy (DR) is a leading cause of vision loss in patients with diabetes, with limited therapeutic options. While current treatments focus on the proliferative stage, there is an urgent need to understand the underlying mechanisms of the early stages of DR to halt progression. Growing research suggests that activated microglia are key drivers of inflammation in DR.
Age-related macular degeneration (AMD) is a leading cause of vision loss in older adults. Current treatments mainly address advanced AMD, while early or intermediate stages rely only on micronutrient supplements. This highlights a critical gap in understanding the molecular drivers of early dry AMD and the need for strategies to prevent progression to geographic atrophy or choroidal neovascularization and vision loss.
Torrent Pharmaceutical Ltd. will buy J. B. Chemicals and Pharmaceuticals Ltd. (JB Pharma) from private equity firm KKR & Co. Inc. at an equity valuation of ₹256.89 billion (US$3 billion), which will then merge into one entity under Torrent Pharma.
Three months after dosing the first patient with its dual vector gene therapy, Splicebio SL has closed a $135 million series B to fund the phase I/II trial of SB-007 in the treatment of Stargardt’s disease to completion. Other adeno-associated viru gene therapies for the inherited retinal disorder have entered the clinic, but SB-007 is the first with the capacity to deliver a full version of the ABCA4 gene that underlies Stargardt’s.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have patented 5-[2,7-diazaspiro[3.5]nonan-7-yl]-5-[4-phenoxyphenyl]hexahydropyrimidine-2,4,6-trione derivatives acting as matrix metalloproteinase-9, (MMP-9) inhibitors potentially useful for the treatment of dry eye.
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