Scientists from the Machine Intelligence from Cortical Networks (MICRONS) consortium have published the microconnectome of a cubic millimeter of the mouse brain. This is the most complete map of this organ to date at nanometer resolution for a mammal. It not only contains the structure and connections of each and every cell in that volume of tissue, but is also linked to the neuronal activity of that portion of the CNS, linking anatomy and function in the same cells.
Genflow Biosciences plc has announced a development program in ophthalmology focused on advancing a novel gene therapy leveraging its proprietary centenarian SIRT6 (cSIRT6).
Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.
Age-related macular degeneration (AMD) is the leading cause of blindness among older adults. The exudative or ‘wet’ form of the disease is characterized by the growth of abnormal blood vessels from the choroid into the subretinal space, known as choroidal neovascularization (CNV), leading to macular edema.
Hubble Therapeutics LLC has successfully closed a $7.3 million series A funding round to support progression of its lead candidate, HUB-101, into clinical trials.
Researchers from Senju Pharmaceutical Co. Ltd. presented results from preclinical studies that assessed the hyperthermic effects of SJP-0132, a selective antagonist of transient receptor potential vanilloid 1 (TRPV1), which is currently awaiting approval in Japan as an ophthalmic treatment to alleviate the signs and symptoms of dry eye disease.
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.
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.
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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