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Home » Topics » Neurology/psychiatric, BioWorld Science

Neurology/psychiatric, BioWorld Science
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Art concept for gene therapy research
Neurology/psychiatric

Caspida’s CAP-004 exhibits potential as a best-in-class therapy for Friedreich’s ataxia

May 16, 2025
No Comments
Capsida Biotherapeutics Inc. has reported development of a systematically administered capsid, CAP-004.
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Illustration of human brain and dna
Neurology/psychiatric

Quralis signs agreements to further fragile X syndrome research

May 16, 2025
No Comments
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
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Cerebellum, brain stem, spinal cord
Neurology/psychiatric

Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3

May 15, 2025
No Comments
The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).
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Illustration of liver with DNA double helixes
Endocrine/metabolic

ASGCT 2025: Gene and cell therapies transform metabolic diseases

May 15, 2025
By Mar de Miguel
No Comments
Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.
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Neurology/psychiatric

Sevenless Therapeutics identifies new SOS1 inhibitors

May 14, 2025
Sevenless Therapeutics Ltd. has disclosed son of sevenless homolog 1 (SOS1) inhibitors reported to be useful for the treatment of cancer, pain, dementia, Noonan syndrome, Parkinson's disease and neurofibromatosis type 1.
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Neurology/psychiatric

BIN1 gene replacement therapy shows preclinical promise for myopathy

May 14, 2025
No Comments
A study published in Molecular Therapy has presented a promising gene therapy approach for treating centronuclear myopathy.
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3D illustration showing DNA inside adenovirus
Neurology/psychiatric

IND clearance for Mavrix Bio’s AAV gene therapy for Angelman syndrome

May 13, 2025
No Comments
Mavrix Bio has received IND clearance from the FDA for MVX-220, an investigational AAV gene therapy for the treatment of Angelman syndrome. The company expects to initiate its first-in-human study, ASCEND-AS, in the second half of this year.
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Pediatric brain illustration
Neurology/psychiatric

Capsida’s gene therapy for STXBP1 developmental and epileptic encephalopathy gains IND clearance

May 13, 2025
No Comments
Capsida Biotherapeutics Inc. has gained IND clearance from the FDA for CAP-002, its first-in-class, intravenously administered gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). Dosing in the phase I/IIa SYNRGY trial will begin in the third quarter of this year.
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Neurology/psychiatric

Fudan University identifies new polyphenol compounds for ischemic stroke

May 12, 2025
Fudan University has described new polyphenol compounds reported to be useful for the treatment of ischemic stroke.
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Neurology/psychiatric

Teijin Pharma describes new orexin OX2 receptor agonists

May 9, 2025
Teijin Pharma Ltd. has identified crystalline forms of oxazepine derivatives acting as orexin OX2 receptor agonists reported to be useful for the treatment of narcolepsy, hypersomnia and sleep apnea.
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