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BioWorld - Thursday, May 28, 2026
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Illustration of kidney with DNA structures
Biomarkers

Mutation in the UMOD gene tied to autosomal dominant tubulointerstitial kidney disease

April 18, 2024
Unknown etiology is commonly encountered in the kidney pre-transplant routine program. A screening program was performed to detect patients and study recipients that meet the following features: hypertension with no clear etiology and biopsies that do not match with clinical features of classical glomerulopathies.
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Acute myeloid leukemia
Immuno-oncology

Bivictrix’s BVX-001 awarded US orphan drug designation for AML

April 18, 2024

Bivictrix Therapeutics plc’s BVX-001 has been awarded orphan drug designation by the FDA for acute myeloid leukemia (AML).


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mRNA on digital background
Drug Design, Drug Delivery & Technologies

Circio establishes in vivo proof of concept for Circvec circular RNA platform

April 18, 2024
Circio Holding ASA has established technical in vivo proof of concept for its proprietary Circvec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression.
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Cancer

Preclinical characterization of AZD-8421, a potent and selective CDK2 inhibitor

April 18, 2024
Researchers from Astrazeneca plc reported preclinical data for AZD-8421, a selective cyclin-dependent kinase 2 (CDK2) inhibitor, currently being evaluated in early-phase clinical trials as a treatment for solid tumors.
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UVA and UVB radiation penetrate into the skin layer vector on white background.
Cancer

Derm-Biome compound shows promise to prevent and treat squamous cell carcinoma in UV-induced skin cancer model

April 18, 2024
Derm-Biome Pharmaceuticals Inc. has developed a topical anticancer treatment for patients with or at high risk of developing multiple actinic keratoses.
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Medical vector illustration showing cross section of an eyeball with close up of the macula
Ocular

Saliogen nominates development candidate for Stargardt disease

April 18, 2024
Saliogen Therapeutics Inc. has nominated a development candidate, SGT-1001, for the treatment of Stargardt disease. SGT-1001 is being developed as a one-time, nonviral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
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Cardiovascular

Study investigates how PCSK9 leads to atherosclerosis progression independently of LDL receptor

April 18, 2024
Proprotein convertase subtilisin/kexin type-9 (PCSK9) is highly expressed in adult hepatocytes. PCSK9 binds to and promotes the degradation of the low-density lipoprotein (LDL) receptor, thereby increasing LDL cholesterol levels. PCSK9 inhibition has emerged as a promising strategy for cardiovascular diseases. However, it is still unclear whether PCSK9 can trigger blood vessel inflammation directly modulating monocytes or endothelial cells independently of LDL receptor.
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Illustration of muscle anatomy
Musculoskeletal

Human muscle aging atlas unveils damage and repair systems

April 18, 2024
By Mar de Miguel
The first cellular human and mouse map focused on muscle fibers and their microenvironment has revealed both the mechanisms of deterioration of this tissue over time and its adaptive capacity for regeneration. “We intended to map the skeletal muscle, isolating all the cell types, and characterizing how they change with age,” first author Veronika Kedlian from the Wellcome Sanger Institute in Cambridge told BioWorld.
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Other news to note for April 17, 2024

April 17, 2024
Additional early-stage research and drug discovery news in brief, from: 20med Therapeutics, Athira Pharma, Telomir Pharmaceuticals.
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Neurology/Psychiatric

Biointervene discovers new adenosine A3 receptor agonists

April 17, 2024
Biointervene Inc. has described adenosine analogues acting as adenosine A3 receptor (ADORA3) agonists reported to be useful for the treatment of mild cognitive impairment, diabetic neuropathy, irritable bowel syndrome, neurodegeneration, ototoxicity, neuropathic pain, chemotherapy-induced peripheral neuropathy and spinocerebellar ataxia, among others.
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