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DNA illustration
Genetic/congenital

Prime Medicine presents data on PM-359 for chronic granulomatous disease

May 10, 2024
Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.
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Cancer

Targeting THOC7-AS1/OCT1/FSTL1 axis as therapeutic approach in cSCC

May 10, 2024
Researchers from Xiangya Hospital of Central South University published data from a study that explored potential treatment targets for cutaneous squamous cell carcinoma (cSCC) by investigating the regulatory mechanisms of follistatin-like 1 (FSTL1), which has been shown to be implicated in a variety of cancers.
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Vial and syringe with DNA
Ocular

ASGCT: ‘From darkness to light’ in ocular gene therapy

May 10, 2024
By Mar de Miguel
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
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Other news to note for May 9, 2024

May 9, 2024
Additional early-stage research and drug discovery news in brief, from: Modalis Therapeutics, Nanoviricides.
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Endocrine/metabolic

Maze Therapeutics discovers new SLC6A19 inhibitors

May 9, 2024
Maze Therapeutics Inc. has described sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria, metabolic syndrome, chronic kidney disease and metabolic diseases.
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Endocrine/metabolic

Biogen and Broad Institute present new GSK-3α inhibitors

May 9, 2024
Researchers at Biogen Inc. and Broad Institute Inc. have divulged glycogen synthase kinase-3α (GSK3A; GSK-3α) inhibitors reported to be useful for the treatment of cancer, neurological and metabolic diseases.
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Cancer

Norroy Bioscience describes new FAP-targeting conjugates for diagnosis and treatment of cancer

May 9, 2024
Norroy Bioscience Co. Ltd. has identified conjugates comprising fibroblast activation protein α (FAP)-targeting agents covalently linked to cytotoxic drugs or unlabeled or radiolabeled imaging agents through a linker acting as FAP ligands reported to be useful for diagnosis and treatment of cancer expressing FAP.
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Infection

Roche divulges new 3CLpro inhibitors

May 9, 2024
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized 3C-like proteinase (3CLpro, Mpro) (coronavirus) inhibitors reported to be useful for the treatment of coronavirus acute respiratory syndrome infections.
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Cardiovascular

Astrazeneca patents new CX3CR1 antagonists for heart failure

May 9, 2024
Astrazeneca AB has disclosed CX3C chemokine receptor 1 (CX3CR1; CMKBRL1; GPR13) antagonists reported to be useful for the treatment of heart failure, among others.
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Antibody-drug conjugate
Cancer

BL-M11D1 shows antitumor activity in AML xenograft models

May 9, 2024
Researchers from Sichuan Baili Pharmaceutical Co. Ltd. and Systimmune Inc. presented preclinical data for the novel CD33-targeting antibody-drug conjugate (ADC) being evaluated for the treatment of hematologic malignancies.
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