Capsida Biotherapeutics Inc. presented preclinical data for a new next-generation gene supplementation therapy candidate, CAP-003, for Parkinson’s disease (PD) patients with GBA1 mutations (PD-GBA).
Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.
Borvo Medical Inc., a neurointerventional med-tech startup, reported U.S. FDA 510(k) clearance of its Endoport Vacuum Assisted Collection (EVAC) system, an alternative to traditional subdural hematoma (SDH) drainage methods. The EVAC system is expected to be available in early 2025 the company said.
In an effort to shore up its position in a rapidly developing market for brain-computer interface (BCI) technologies, Onward Medical NV signed an exclusive license with France’s CEA-Clinatec to use its BCI chip as part the Arc-BCI system, which restores direct communication from the brain to the spinal cord.
Researchers at McGill University and Neurasic Therapeutics Inc. have described substituted thiophene fused cyclohexanone derivatives acting as acid-sensing ion channel 1 (ASIC1) blockers reported to be useful for the treatment of pain, particularly neuropathic and inflammatory pain.
Researchers from Nitrase Therapeutics Inc. recently presented preclinical findings on NTX-101, a murine chimeric antibody designed to specifically bind to nitrated tyrosine on α-synuclein (α-Syn). It was demonstrated that NTX-101 bound both chemically and synthetically nitrated α-Syn (n-Syn), with no binding to non-nitrated α-Syn or an irrelevant nitrated protein observed.
Graft Polymer (UK) plc has announced a research agreement between its partner, Awakn Life Sciences Corp., and the University of Nottingham. The agreement covers in vivo research to evaluate the potential of Graft Polymer and Awakn’s aminoindane NCE co-lead series to enhance social cognition and pro-social behaviors.
Researchers from Harvard Medical School and the Icahn School of Medicine at Mount Sinai found two rare, recurrent patterns of mutation in the post-mortem brain tissue of individuals with schizophrenia that could reflect prenatal mutational processes.
To recreate in the laboratory the formation of Lewy bodies as they would occur in a Parkinson’s patient, two ingredients are required: the protein α-synuclein and the participation of the immune system. The results could prevent the development and progression of this neurodegenerative disorder and help in the search for new therapies.
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