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.
Researchers from Tongji University School of Medicine and collaborating institutions have discovered that NP65 plays a crucial role in maintaining normal visual function in mice. The study, published in Frontiers in Cellular Neuroscience, found that mice lacking NP65 exhibit impaired visual responses.
Among the most significant genetic risk factors for age-related macular degeneration is the Y402H variant of complement factor H, which, along with its splice isoform FHL-1, impairs binding to the retinal surface.
Individuals with autoimmune diseases affecting the thyroid can suffer eye complications in the form of Graves’ orbitopathy, in which inflammation in and around the orbit leads to eyelid retraction, eye bulging and other symptoms.
The progression of neurodegeneration during experimental glaucoma is tied to the early degradation of anterograde transport along retinal ganglion cells to central brain targets, which is followed by frank optic nerve degeneration.
RSS
