Centogene NV and Evotec SE have announced the discovery of a new small molecule with potential to treat patients with type 2 and type 3 Gaucher disease, or neuronopathic Gaucher disease.
Ascidian Therapeutics Inc. recently provided preclinical data for ACDN-01, an AAV-encoded RNA exon editor targeting ABCA4, being developed for the treatment of ABCA4-related retinopathies, including Stargardt disease.
Arvinas Operations Inc. has synthesized proteolysis targeting chimera (PROTACs) compounds comprising an E3 ubiquitin ligase-binding moiety coupled to a B-cell lymphoma 6 protein (BCL6)-targeting moiety through a linker.
Merck Sharp & Dohme LLC has patented potassium voltage-gated channel subfamily C member 1 (Kv3.1; KCNC1) activators reported to be useful for the treatment of neurological and psychiatric disorders.
Scorpion Therapeutics Inc. has found compounds acting as cellular tumor antigen p53 (TP53) (Y220C mutant) activators reported to be useful for the treatment of cancer.
A new adeno-associated viral vector-driven CR2-CR1 fusion protein, named KRIYA-825, was tested in vivo in mice with sodium iodate-induced geographic atrophy.
Researchers from Vividion Therapeutics Inc. and partner Roche Holdings AG have published the discovery and preclinical characterization of the novel covalent allosteric inhibitor of WRN, VVD-133214 (RO-7589831).
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