Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 gene, obtaining sustained efficacy as well as good safety for clinical translation for the treatment of BBS.
Grifols SA has entered into a partnership with the U.S. Biomedical Advanced Research and Development Authority (BARDA) to test investigational ocular surface immunoglobulin (OSIG) eye drops for their ability to treat ocular damage from sulfur mustard exposure.
Shanghai Meiyue Biotech Development Co. Ltd. has synthesized spiro compounds acting as complement factor B (CFB) inhibitors reported to be useful for the treatment of age-related macular degeneration, glomerulonephritis, atypical hemolytic uremic syndrome, membranous nephropathy, myasthenia gravis, paroxysmal nocturnal hemoglobinuria, diabetic retinopathy and rheumatoid arthritis, among others.
Protein homeostasis is disrupted in response to an uncontrolled stress condition, a hallmark of many diseases, such as cataracts. Lens protein accumulation and aggregates are a cause of cataract development.
A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work.
A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work. By using electron microscopy and three-dimensional reconstruction supported by AI tools, the researchers have revealed the neural wiring of the Drosophila melanogaster brain, a connectome of 140,000 neurons with 50 million synaptic connections. In the future, researchers could possibly use this map as an artificial in silico model to study the brain as a simulator through its connections, though a lot of work remains to be done for this.
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune demyelinating disorder of the optic nerves and the spinal cord that is linked to the presence of anti-aquaporin (AQP4) autoantibodies. Satralizumab, a humanized anti-IL-6 receptor (IL-6R) antibody, has been approved for the treatment of NMOSD but the effects of IL-6R inhibition in myelitis severity have not been fully explored.
Uveitis is a disease involving the eye, affecting the iris, ciliary body, retina and their associated blood vessels, among others. The non-infectious type of uveitis is linked to immune-related factors and its pathogenesis remains poorly understood.
Surrozen Inc. has announced that Boehringer Ingelheim Pharma GmbH & Co. KG will further develop SZN-413 to advance the compound and prepare it for clinical testing. Boehringer decided to move forward with development based on the completion of the initial period of joint research under the companies’ strategic partnership for the research and development of SZN-413 for the treatment of retinal diseases.
For once, the EMA appears to have pipped the U.S. FDA to the post, with Pfizer Inc.’s hemophilia A and B therapy Hympavzi (marstacimab) recommended for approval in Europe on Sept. 20, while the U.S. PDUFA date is set for the fourth quarter of the year.
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