The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).
At first blush, the brain’s extracellular matrix (ECM) seems like the opposite of synaptic plasticity. Plasticity is the ability to change; the ECM is stable, to the point that it is often described as a scaffold – something to lend stability. “ECM proteins have some of the longest lifetimes of any protein in the brain,” Anna Molofsky told her audience at the XVII Meeting on Glial Cells in Health and Disease, which is being held in Marseille this week.
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged bifunctional epoxide hydrolase 2 (EPHX2; sEH) inhibitors reported to be useful for the treatment of pain, diabetic retinopathy and neurodegeneration. An exemplified compound (Ex 23 pg 78, claim 18) inhibited human sEH activity (IC50=0.5 nM) using Rapidfire mass spectrometry (RFMS) assays (WO 2025125135).
Idorsia Pharmaceuticals Ltd. has synthesized aryl sulfone and sulfanone derivatives acting as orexin OX2 receptor agonists reported to be useful for the treatment of eating disorders, fatigue, Kleine-Levin syndrome, narcolepsy, obesity, pain, and psychiatric and inflammatory disorders, among others.
Neuroinflammation is a common hallmark in several neurodegenerative disorders, including Alzheimer’s and Parkinson’s diseases, among others, where TREM2 (triggering receptor expressed on myeloid cells 2) is a crucial member involved in this biological process and is mainly expressed in microglial cells, being thus an attractive target for diagnostic imaging.
Sensorium Therapeutics Inc. has reported that the U.S. FDA has cleared the IND application for SNTX-2643 (SENS-01), its lead anxiety program. First-in-human dosing begins in Q3 2025.
Insightec Ltd. received U.S. FDA approval for use of its Exablate Neuro device to address severe motor symptoms in patients with Parkinson’s disease, offering a new option for patients who have not found adequate relief from medications. Exablate Neuro uses focused ultrasound to create lesions in the brain without requiring an incision or implant.
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