The FDA has accepted the IND application from Rest Therapeutics SAS for RST-101, the company’s lead investigational candidate for the early treatment of post-traumatic stress disorder (PTSD).
Recent findings are reshaping current understanding of the post-infection landscape of SARS-CoV-2. Although previous studies had already suggested that autoimmunity might underlie the persistent neurological symptoms seen in long COVID, researchers at Yale University and Mount Sinai now reinforce this hypothesis. SARS-CoV-2 infection appears to trigger an autoimmune mechanism that drives chronic pain, fatigue and cognitive impairment in some patients.
The “deep dive” April 8 by Ovid Therapeutics Inc. into potassium-chloride co-transporter 2 (KCC2) research included company officials and independent experts in the space, where a handful of drug developers are known to be working. New York-based Ovid owns a portfolio of potential first-in-class direct activators of KCC2, and the firm detailed pharmacodynamic results along with the translational and clinical development strategy.
Medtronic plc is strengthening its pain management portfolio with its $650 million move to buy SPR Therapeutics Inc. and bring the company’s peripheral nerve stimulation system for sustained pain relief into its neuromodulation offering. The deal marks Medtronic’s third tuck-in deal so far this year, following its $585 million purchase of Cathworks Ltd. and $550 million acquisition of Scientia Vascular Inc., and is also a sign of the momentum in M&A activity in the medtech sector with other players making strategic acquisitions.
Cavalon Therapeutics Inc. and Northwestern University have disclosed new voltage-gated calcium channel Cav1.3 blockers potentially useful for the treatment of Parkinson’s disease and aldosteronism.
AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload.
Pfizer Inc. has reported new triggering receptor expressed on myeloid cells 2 (TREM2) agonists potentially useful for the treatment of neurodegeneration.
Tau pathology, driven by MAPT, is central to Alzheimer’s disease (AD) and closely associated with cognitive decline. Supported by extensive preclinical evidence across tauopathies, reducing MAPT expression represents a promising disease‑modifying strategy for AD, frontotemporal dementia (FTD), primary progressive aphasia (PPA) and related disorders. Researchers at Aviadobio Ltd. presented the preclinical characterization of AVB‑406, an intravenously administered gene therapy developed using its proprietary vMiX RNAi gene silencing platform, designed to lower tau production and reduce neurofibrillary tangle formation.