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BioWorld - Saturday, May 16, 2026
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Home » Topics » BioWorld Science, Neurology/psychiatric

BioWorld Science, Neurology/psychiatric
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Illustration of man holding magnifying glass to human body model showing muscle anatomy
Musculoskeletal

In muscular dystrophies, gene therapies race the clock

Nov. 25, 2024
By Mar de Miguel
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.
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Immune

Seed funding at Autoimmunity Biosolutions to advance immuno-corrective therapy for autoimmune diseases

Nov. 22, 2024
Autoimmunity Biosolutions has closed a seed financing to support its work advancing a next-generation, immuno-corrective therapy for autoimmune diseases. The company’s approach targets a branch of the interleukin-7 (IL-7)/interleukin-7 receptor (IL-7R) pathway controlled by the soluble IL-7R (sIL-7R), a critical amplifier of autoimmune reactions.
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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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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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Neurology/psychiatric

Verge Analytics patents new PIKfyve inhibitors

Nov. 19, 2024
Verge Analytics Inc. (dba Verge Genomics) has prepared and tested new hydrazone and pyrazole 3H-imidazo(4,5-b)pyridine compounds acting as 1-phosphatidylinositol 3-phosphate 5-kinase (PIKfyve) inhibitors.
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