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BioWorld - Tuesday, December 23, 2025
Home » Topics » Disease categories and therapies » Neurology/psychiatric

Neurology/psychiatric
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Hands holding brain
Neurology/psychiatric

Rome Therapeutics’ LINE-1 RT inhibitors show neuroprotective effect in Parkinson’s disease models

Nov. 22, 2024
Rome Therapeutics Inc. has presented data demonstrating the potential of its LINE-1 reverse transcriptase (RT) inhibitors.
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Neurology/psychiatric

Gruenenthal describes new nociceptin receptor agonists for pain

Nov. 21, 2024
Gruenenthal GmbH has patented new cis-8-(3,5-difluorophenyl)-8-(dimethylamino)-1,3-diazaspiro[4.5]decan-2-one derivatives acting as nociceptin receptor (OPRL1; KOR3; ORL1) agonists and thus reported to be useful for the treatment of pain.
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Illustration of tau proteins in Alzheimer's disease
Neurology/psychiatric

Voyager Therapeutics to advance tau silencing gene therapy for Alzheimer’s disease

Nov. 21, 2024
Voyager Therapeutics Inc. has selected a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an IND application with the FDA and a clinical trial application (CTA) with Health Canada for VY-1706 in 2026.
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NMPA clears Alpha’s oral EGFR-TKI for NSCLC with brain metastases

Nov. 21, 2024
By Marian (YoonJee) Chu
China’s drug regulator approved Alpha Biopharma Ltd.’s EGFR tyrosine kinase inhibitor (TKI), Zorifer (zorifertinib hydrochloride tablets; AZD-3759), as a first-line treatment for EGFR-mutated non-small-cell lung cancer (NSCLC) with brain metastases.
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Novartis acquires gene therapy developer Kate Therapeutics for $1.1B

Nov. 21, 2024
By Karen Carey
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
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Sage shuts down Huntington’s program after phase II failure

Nov. 20, 2024
By Lee Landenberger
The bad news keeps piling up for Sage Therapeutics Inc. Having absorbed other study stumbles in the past few months, the company now has halted development of dalzanemdor in treating Huntington’s disease after top-line phase II data showed it missed a statistically significant difference compared to placebo on the primary endpoint.
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Neurology/psychiatric

Aeovian Pharmaceuticals patents new CD38 inhibitors

Nov. 20, 2024
Aeovian Pharmaceuticals Inc. has prepared tricyclic fused imidazole compounds acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of neurodegeneration, inflammation, fibrosis, systemic scleroderma and more.
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Illustration of siRNA structure
Neurology/psychiatric

Ractigen Therapeutics’ RAG-21 designated orphan drug for ALS

Nov. 20, 2024
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
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Neurology/psychiatric

Sangamo’s ST-503 receives IND clearance for idiopathic small fiber neuropathy

Nov. 20, 2024
Sangamo Therapeutics Inc. has received IND clearance from the FDA for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN).
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Degradation of motor neurons
Neurology/psychiatric

Lilly to develop ALS therapeutics against targets identified under Verge collaboration

Nov. 20, 2024
Eli Lilly & Co. has elected to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) from its collaboration with Verge Analytics Inc. (dba Verge Genomics).
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