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BioWorld - Sunday, June 21, 2026
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Brain and DNA
Neurology/Psychiatric

PPMD funding supports Kinea’s AAV-mediated midi-dystrophin gene replacement therapy for DMD

Feb. 14, 2024
Parents Project Muscular Dystrophy (PPMD) has provided $500,000 in funding to Kinea Bio Inc. 
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Endocrine/Metabolic

Autologous B-cell therapy WFX-001 corrects systemic α-GAL enzyme deficiency in vivo

Feb. 14, 2024
Walking Fish Therapeutics Inc. presented a new first-in-class autologous B-cell therapy being developed for the treatment of Fabry disease.
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Muscular dystrophy
Neurology/Psychiatric

SAT-3247 improves skeletal muscle function in mouse model of facioscapulohumeral muscular dystrophy

Feb. 14, 2024
Satellos Bioscience Inc. has released promising preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of...
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Neuron
Neurology/Psychiatric

GABA-A receptor potentiator KRM-II-81 blocks pain-like behaviors in rats

Feb. 14, 2024
Respirerx Pharmaceuticals Inc.’s lead GABA-A receptor potentiator, KRM-II-81, has advanced to the next level of evaluation within the National...
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Neurology/Psychiatric

VST Bio presents data on novel anti-syndecan-2 antibody

Feb. 14, 2024
It has been shown that vascular endothelial growth factor A (VEGF-A) induces blood-brain barrier disruption and vasogenic edema and it is up-regulated in stroke. When bound to its receptor, VEGF promotes angiogenesis and neuroprotection, in addition to inducing vasogenic edema. VST Bio Ltd. and Yale University have presented data on their monoclonal antibody against syndecan-2, named VST-002, that completely blocks VEGF-driven vasogenic edema while preserving neuroprotective effects.
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Man holding head in pain, brain inset
Neurology/Psychiatric

Degrading damaged mitochondria improves concussion

Feb. 14, 2024
By Mar de Miguel
In repeated concussions, removing damaged mitochondria could prevent the neurodegeneration that occurs when pathology progresses in some patients. The key would be in the role of the p17 protein in restoring mitophagy, according to scientists from the Medical University of South Carolina (MUSC). “Brain injury is an extrinsic disease. It is not idiopathic. When the primary injury occurs, the secure mechanism only relies on an endogenous protection of the brain. If you have a good neuroprotective mechanism, then after the primary injury, basically you don’t see any symptomatic effect,” Onder Albayram told BioWorld.
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Masayo Takahashi, CEO, Vision Care
Ocular

Newco news: Vision Care’s iPS retinal cells primed for global market

Feb. 13, 2024
By Tamra Sami
Vision Care Group CEO Masayo Takahashi led the world's first clinical study of a retinal cell transplant derived from induced pluripotent stem cells (iPS cells) in 2014 when she led the Laboratory for Retinal Regeneration at Japan’s Riken Center for Biosystems Dynamics Research. In 2019, she founded Vision Care and subsequently founded two subsidiary companies dedicated to developing cell and gene therapies.
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Immuno-oncology

Sutro Biopharma discovers new antibody-drug conjugates for diagnosis and treatment of cancer

Feb. 13, 2024
Sutro Biopharma Inc. has described antibody-drug conjugates (ADCs) comprising a cytotoxic drug linked to an antibody or polypeptide residue through a linker reported to be useful for the diagnosis and treatment of cancer.
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Cancer

Jiangsu Tasly Diyi Pharmaceutical presents new Wee1 inhibitors

Feb. 13, 2024
Jiangsu Tasly Diyi Pharmaceutical Co. Ltd. has divulged Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of inflammation, infection, cancer and autoimmune disease.
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Endocrine/Metabolic

Jnana Therapeutics describes new SLC6A19 inhibitors

Feb. 13, 2024
Jnana Therapeutics Inc. has identified sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatohepatitis, phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others.
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