Another brick in the ambitious Human Cell Atlas initiative has been put into place with the publication of the largest and most comprehensive cell map of the human lung. The open and freely available atlas catalogs the diversity of cells in the lung, including rare and previously undescribed cell types.
Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Currently, there are no treatments to reverse or prevent genetic hearing loss, which affects 1 in 500 newborns. Several gene replacement and overexpression preclinical studies targeting genetic hearing loss have shown success, as the inner ear can be accessed safely by local injection. However, all these gene therapy studies have been performed in neonatal animals, except one in the Otof gene; therefore, the suitability of the approach in the fully mature adult inner ear remains to be elucidated.
Lantern Pharma Inc. has entered into a research collaboration with Bielefeld University to develop antibody-drug conjugates (ADCs) with therapeutic and antitumor potential. Using Lantern’s proprietary artificial intelligence (AI) platform, RADR, the collaboration will leverage insights from the recently developed RADR AI ADC module in combination with research from Bielefeld.
Pipeline Therapeutics Inc. has received FDA clearance to initiate a phase I trial of PIPE-791 in healthy volunteers, with dosing expected to begin in the second half of this year. PIPE-791 is advancing toward clinical development for remyelination and neuroinflammation, with a leading indication of multiple sclerosis (MS).
Wigen Biomedicine Technology (Shanghai) Co. Ltd. researchers have identified mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer.
AC Immune SA has patented 4h-imidazo[1,5-b]pyrazole derivatives and labeled compounds targeting α-synuclein (SNCA). They are reported to be useful for diagnosis of multiple system atrophy, Parkinson’s dementia, Lewy body dementia, Alzheimer’s and Parkinson’s disease, among others.
Cerevance Inc. has described N-(4-aminocyclohexyl)pyrimidine-4-carboxamide derivatives acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors. As such, they are reported to be useful for the treatment of aging, diabetic nephropathy, glaucoma, inflammatory disorders, metabolic syndrome, obesity and rheumatoid arthritis.
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