Researchers at Iama Therapeutics Srl and Italian Institute of Technology have disclosed solute carrier family 12 member 2 (SLC12A2; NKCC1) inhibitors reported to be useful for the treatment of Down syndrome.
Researchers from Iama Therapeutics Srl and the Italian Institute of Technology have prepared and tested solute carrier family 12 member 2 (SLC12A2; NKCC1) inhibitors reported to be useful for the treatment of complex regional pain syndromes, Alzheimer’s disease, Down syndrome, glioma, Rett syndrome, temporal lobe epilepsy, traumatic brain injury and obsessive-compulsive disorder.
Neurona Therapeutics Inc. has completed an upsized, oversubscribed $102 million financing round, the third company to do so in a month that’s only four days old. The privately held company’s lead asset is NRTX-1001, a regenerative cell therapy derived from human pluripotent stem cells.
Growing evidence exists on regulation of the chloride importer solute carrier family 12 member 2 (SLC12A2), also known as NKCC1, as a therapeutic approach to treat neurological disorders. Altered expression of NKCC1 leads to impaired intracellular chloride levels in neurons and imbalance in the excitatory-inhibitory axis in the brain.
Iama Therapeutics Srl has obtained clinical trial application (CTA) clearance from the Italian Medicines Agency (AIFA) to initiate a first-in-human phase I study of IAMA-6, an orally administered small-molecule therapeutic targeting NKCC1 for the treatment of autism and epilepsy.
Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and neurologic conditions, including mesial temporal lobe epilepsy (MTLE), among others, leading to an imbalance between excitatory and inhibitory signals.
Iama Therapeutics srl and Psychogenics Inc. have entered into a new services agreement to identify the efficacy of novel drug candidates in preclinical animal models of Dravet syndrome.
Iama Therapeutics Srl has sought new, selective inhibitors of solute carrier family 12 member 2 (NKCC1) to treat autism, Down syndrome and brain disorders featuring chloride (NKCC1/solute carrier family 12 member 5 [KCC2]) imbalance.