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.
Biocytogen Pharmaceuticals (Beijing) Co. Ltd. and Nanjing Chia Tai Tianqing Pharmaceutical Co. Ltd. have announced IND clearance by China’s National Medical Products Administration (NMPA) for NTB-003 (formerly BCG-009) for thyroid eye disease.
Inmed Pharmaceuticals Inc. has divulged cannabinoid CB1 and/or CB2 receptor modulators reported to be useful for the treatment of neurodegeneration and age-related macular degeneration.
Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, progressive retinal degeneration resulting in vision loss or blindness, and renal abnormalities that may progress to renal failure. Mutations in the BBS10 gene are the second most prevalent cause of BBS, accounting for over 20% of cases.
Age-related macular degeneration (AMD) is the primary cause of blindness in people aged 65 and older in developed nations. Recent estimates suggest that, globally, the number of individuals affected by AMD could rise to approximately 288 million by 2040.
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