INO-1001 / Rocket Pharma, Roche - LARVOL DELTA

Poly(ADP-ribose) polymerase regulates transient receptor potential channel M2-dependent calpain activation in rd1 mouse retinal degeneration. (PubMed, Neural Regen Res) - "We also characterized INO1001 as potentially more beneficial for inherited retinal degeneration treatment than Olaparib. Our study details the complexity of poly(ADP-ribose) polymerase-signaling in photoreceptors and identifies poly(ADP-ribose) glycohydrolase and transient receptor potential channel M2 as new targets for inherited retinal degeneration therapy development."

Journal • Preclinical • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • CAPN2 • PARP1

PARP Activity Is Essential for Retinal Photoreceptor Survival in the Human Homologous RhoI255del Mouse Model for Autosomal Dominant Retinitis Pigmentosa. (PubMed, J Neurochem) - "The PARP inhibitors used -olaparib, saruparib, INO1001, and nicotinamide-target different PARP isoforms, and their potentially differential effects were evaluated in organotypic retinal explants cultivated under entirely defined conditions. On the other hand, cone photoreceptors were apparently unaffected by PARP inhibition. The present study thus demonstrates the importance of PARP activity for rod photoreceptor viability in a dominant rhodopsin mutant, highlights the need for a deeper understanding of the mechanisms underlying photoreceptor degeneration in different RP forms, and cautions against the indiscriminate use of PARP inhibitors for the treatment of RP."

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

Selective Sparing of Striatal Interneurons after Poly (ADP-Ribose) Polymerase 1 Inhibition in the R6/2 Mouse Model of Huntington's Disease. (PubMed, Front Neuroanat) - "Moreover, INO-1001 promoted CBP localization into the nuclei of the R6/2 mouse. The sum of our data corroborates the previous observations indicating PARP inhibition as a possible therapeutic tool to fight HD."

Journal • Biosimilar • Cardiovascular • CNS Disorders • Ophthalmology • Reperfusion Injury

PARP-1 Inhibition Is Neuroprotective in the R6/2 Mouse Model of Huntington's Disease. (PubMed) - "INO-1001 was effective in significantly increasing activated CREB and BDNF in the striatal spiny neurons, which might account for the beneficial effects observed in this model. Our findings show that PARP-1 inhibition could be considered as a valid therapeutic approach for HD."

Journal • Biosimilar

PARP-1 inhibition protects the diabetic heart through activation of SIRT1-PGC-1α axis. (PubMed, Exp Cell Res) - "PARP1 inhibitor INO1001 attenuated cardiomyopathic features in diabetic mice through the activation of SIRT1 and its downstream antioxidant defence mechanisms. The results of this study suggest a pivotal role of PARP-1 inhibition in treating diabetic and AT-induced cardiomyopathy."

Journal • PARP Biomarker