Neurology/psychiatric

Janssen Pharmaceutica reports new TMEM175 activators

Tue, 02 Jun 2026 09:00:00 -0400

Janssen Pharmaceutica NV has identified new endosomal/lysosomal proton channel TMEM175 activators that are potentially useful for the treatment of Parkinson’s disease.

MJFF grants supports Lysoway’s TMEM175 agonist program

Tue, 02 Jun 2026 09:00:00 -0400

Lysoway Therapeutics Inc. has been awarded an additional research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support development of its TMEM175 agonist program for Parkinson’s disease.

Voyager’s tau-targeted gene therapy VY-1706 gains IND approval

Tue, 02 Jun 2026 09:00:00 -0400

Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.

Edgewise sells muscular dystrophy assets to Servier for $2.65B

Mon, 01 Jun 2026 12:00:00 -0400

Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten (EDG-5506), to Servier SA. The deal is potentially worth up to $2.65 billion when including a potential $1.1 billion in milestone payments.

Sharp Therapeutics identifies lead chemical series for Niemann-Pick disease

Fri, 29 May 2026 09:00:00 -0400

Sharp Therapeutics Corp. has reported new preclinical data supporting its novel therapeutic approach for Niemann-Pick disease type C (NPC).

FDA accepts IND for Rest Therapeutics’ early PTSD candidate

Fri, 29 May 2026 09:00:00 -0400

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).

New evidence links autoimmunity to long COVID symptoms

Thu, 28 May 2026 09:00:00 -0400

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.

Will Ovid’s golden age of KCC2 metamorph CNS?

Wed, 27 May 2026 12:00:00 -0400

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 bolsters pain portfolio in $650M SPR Therapeutics buy

Tue, 26 May 2026 12:00:00 -0400

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 and Northwestern patent new Cav1.3 blockers

Tue, 26 May 2026 09:00:00 -0400

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.

Gemma Biotherapeutics’ GB-703 shows promise for DMD

Tue, 26 May 2026 09:00:00 -0400

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.

TREM2 agonists detailed in Pfizer patent

Fri, 22 May 2026 09:00:00 -0400

Pfizer Inc. has reported new triggering receptor expressed on myeloid cells 2 (TREM2) agonists potentially useful for the treatment of neurodegeneration.

Ignis Therapeutics synthesizes new muscarinic M4 receptor PAMs

Fri, 22 May 2026 09:00:00 -0400

Ignis Therapeutics (Suzhou) Co. Ltd. has patented new muscarinic M4 receptor positive allosteric modulators (PAMs) potentially useful for the treatment of schizophrenia, bipolar disorder, Huntington’s, Alzheimer’s, inflammatory bowel disease, drug-induced dyskinesia, pain and skin lesions.

AVB-406: AAV-miRNA targeting MAPT in Alzheimer’s disease

Fri, 22 May 2026 09:00:00 -0400

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.

Sangamo presents primate data for prion suppressor ST-506

Fri, 22 May 2026 09:00:00 -0400

Sangamo Therapeutics Inc. discussed gene regulation approaches for neurodegenerative diseases when presenting findings on their clinical candidate ST-506 for the treatment of prion disease.

Unmasking the X: EPAC2 shifts the fragile X landscape

Thu, 21 May 2026 09:00:00 -0400

Researchers at UCLA have shown that divergent neuronal signaling in fragile X mice converges on EPAC2, a druggable target whose inhibition restores circuit activity and alleviates core behavioral impairments.

Roche reports new TREM2 agonists

Wed, 20 May 2026 09:00:00 -0400

F. Hoffmann-la Roche Ltd. and Hoffmann-La Roche Inc. have identified new triggering receptor expressed on myeloid cells 2 (TREM2) agonists potentially useful for the treatment of rheumatoid arthritis, amyotrophic lateral sclerosis, frontotemporal dementia, multiple sclerosis, prion disease, stroke, Parkinson’s and Alzheimer’s disease.

HSPCs delivering tissue-penetrating frataxin ameliorate Friedreich’s ataxia symptoms

Wed, 20 May 2026 09:00:00 -0400

Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients.

‘Detargeted’ targeted gene therapy improves activity in Pompe

Wed, 20 May 2026 09:00:00 -0400

A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.

Fosun secures rights to Aribio’s AR-1001 in potential $4.7B deal

Tue, 19 May 2026 12:00:00 -0400

Shanghai Fosun Pharmaceutical (Group) Co. Ltd. is paying $60 million up front for an option to secure exclusive rights to Aribio Co. Ltd.’s oral phase III-stage Alzheimer’s disease (AD) therapy, AR-1001. The option fee plus license agreement has potential to tally $4.7 billion for Aribio, marking the largest deal for an AD asset inked by a Korean biotech company.

Nxera Pharma prepares and tests new GPR17 antagonists

Mon, 18 May 2026 09:00:00 -0400

Nxera Pharma UK Ltd. has identified new uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-Like) antagonists potentially useful for the treatment of multiple sclerosis.

AAV-encoded dimeric PICK1 inhibitors reduce inflammatory, neuropathic pain in mice

Mon, 18 May 2026 09:00:00 -0400

Researchers from the University of Copenhagen and collaborating institutions aimed to develop a therapy for chronic neuropathic pain based on gene therapy delivered with adeno-associated viral (AAV) vectors.

Sonomind raises €20M for ultrasound neuromodulation technology

Fri, 15 May 2026 12:00:00 -0400

Sonomind SAS raised €20 million (US$23 million) in a series A funding round for its ultrasound-based neuromodulation technology for depression. The funds will be used for clinical trials of the non-invasive device, which uses a custom-made acoustic lens to precisely target deep regions within the brain to bring relief to patients suffering from treatment-resistant depression.

NTX-819, a novel mGlu7 NAM for psychiatric disorders

Fri, 15 May 2026 09:00:00 -0400

Increasing evidence supports metabotropic glutamate mGlu7 receptor as a promising target in psychiatric disorders. Neurosterix Pharma Sarl has presented data on NTX-819, a potential first-in-class, potent and selective negative allosteric modulator (NAM) of mGlu7. NTX-819 was tested in vivo in rats using behavioral tests that are relevant to disorders such as obsessive-compulsive disorder (OCD), agitation and mania/psychosis.

Camp4 studies CMP-002 in SYNGAP1 haploinsufficient mouse model

Fri, 15 May 2026 09:00:00 -0400

Camp4 Therapeutics Corp. has highlighted new preclinical data for CMP-002, the company’s lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder. CMP-002 administration resulted in a statistically significant improvement in seizure phenotypes and parameters in a SYNGAP1 haploinsufficient mouse model.

Biogen AD drug shows tau, cognition benefit, despite trial miss

Thu, 14 May 2026 12:00:00 -0400

Biogen Inc. has decided to advance diranersen (BIIB-080) into registrational trials for early Alzheimer’s disease, even though the antisense oligonucleotide therapy, originally discovered by Ionis Pharmaceuticals Inc., missed its phase II primary endpoint.

Fosun secures rights to Aribio’s AR-1001 in potential $4.7B deal

Thu, 14 May 2026 12:00:00 -0400

Sironax patents new SARM1 inhibitors

Thu, 14 May 2026 09:00:00 -0400

Sironax Ltd. has disclosed new NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors potentially useful for the treatment of Parkinson’s disease, multiple sclerosis, traumatic brain injury, diabetic neuropathy, Alzheimer’s disease, Huntington’s disease, hereditary neuropathies, and stroke.

Vivozon reports new dual 5-HT2A/dopamine D2 receptor antagonists

Thu, 14 May 2026 09:00:00 -0400

Vivozon Inc. has identified new heterocyclic dual 5-HT2A/dopamine D2 receptor antagonists potentially useful for the treatment of schizophrenia.

Andzonbio2 enters neuroinflammation collaboration

Thu, 14 May 2026 09:00:00 -0400

Andzonbio2 has signed agreements with the Alborada Drug Discovery Institute (ADDI) at the University of Cambridge and Cambridge Enterprise to advance a new class of therapeutics targeting neuroinflammation, a central driver of multiple neurodegenerative and neurological conditions.