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BioWorld Science

Oct. 27, 2021

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VY-NPC101, a potential NPC1-based gene therapy strategy for Niemann-Pick disease type C

Discovery of novel VMAT2 inhibitor with potential for the treatment of tardive dyskinesia

New tyrosine-protein kinase CSK inhibitors identified at Plexxikon

Stealth BioTherapeutics describes compounds for treating mitochondrial disease

Global Blood Therapeutics patents pyruvate kinase activators

Vertex Pharmaceuticals discovers alpha-1-antitrypsin (Z-mutant) polymerization inhibitors

New TYK2 inhibitors synthesized at Eternity Bioscience

Ec1-LoPE DARPin-toxin fusion protein evaluated with trastuzumab for ovarian cancer

188Re-Rituximab shows promise for treating CD20-overexpressing cancers

Vyne reports findings with BET inhibitor VYN-201 in animal model and ex vivo human tissue study

CRISP/Cas9 modulation of utrophin as a strategy to treat DMD

Diverse Biotech's DBT-12733 shows efficacy against glioblastoma tumor cells

New mouse model shows possibility of reversing autosomal dominant polycystic kidney disease

Study casts light on DNA damage in esophageal cancer

Novel split-intein-based gene therapy strategy to treat Wilson disease

mLB-001 GeneRide vector restores hepatocyte function in MMA mice

RFN-ALFQ nanoparticle vaccine elicits immunity against SARS-CoV-2 in macaques

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