ACY-957 / Regenacy - LARVOL DELTA

Modeling Therapeutic Targets: β-Globin Disorders and Beyond. (PubMed, Adv Exp Med Biol) - "Comparative analyses of existing interventions identify MS-275 and ACY-957 as the most effective drug-based inducers of -globin, while CHD4-targeting RNAi emerges as the most potent among established gene therapies. Both strategies produce significantly greater -globin induction than currently known treatments. Overall, the chapter demonstrates how hybrid PNs can serve as a powerful computational tool for mechanistically guided target discovery in -globin disorders and related genetic diseases."

Journal • Review • Beta-Thalassemia • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CHD4 • GATA1 • HDAC1

Differential effects of HDAC inhibitors in the RhoI255d mouse model for autosomal dominant retinitis pigmentosa. (PubMed, Cell Death Discov) - "Treatment with the HDAC class I/II inhibitor Trichostatin A and the HDAC class III inhibitor nicotinamide (NAM) suggested that most HDAC activity detected in RhoI255d/+ photoreceptors was related to class I/II isoforms...HDAC inhibitors tested included SAHA (Vorinostat), MPT0G211, ACY-957, and NAM...Our study highlights the complexity and ambiguity of HDAC activity during photoreceptor neurodegeneration and cautions against the use of unspecific inhibitors. At the same time, it showcases important differences between rod and cone photoreceptors and suggests especially HDAC-6 as a potential target for future therapy development."

Journal • Preclinical • CNS Disorders • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • CAPN2 • CASP3