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BioWorld - Saturday, April 11, 2026
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3D-rendered illustration of a synapse cross-section
Neurology/psychiatric

PAK1 inhibition confers synaptic spine resilience in Alzheimer‘s models

Aug. 5, 2025
No Comments
In Alzheimer’s disease (AD), degeneration of synapses, including synaptic spines, has been linked to cognitive losses.
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DNA double helix under a magnifying glass
Nephrology

Purespring gets go-ahead for first kidney-directed gene therapy

Aug. 5, 2025
No Comments
Purespring Therapeutics Ltd. has received the go-ahead for a phase I/II trial of its investigational gene therapy PS-002 from both the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and the National Health Service Health Research Authority and Research Ethics Committee.
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Cancer

ACLY connects metabolism to immunity in MASH-driven liver cancer

Aug. 5, 2025
No Comments
Researchers from McMaster University and Espervita Therapeutics Inc. have identified the enzyme ATP citrate lyase (ACLY) as a key metabolic regulator of tumor-immune interactions in hepatocellular carcinoma (HCC) driven by metabolic dysfunction-associated steatohepatitis (MASH).
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Illustration of magnifying glass inspecting brain
Neurology/psychiatric

Subtyping beats a path toward precision medicine in Alzheimer’s

Aug. 5, 2025
By Anette Breindl
No Comments
Subtyping is what made precision medicine in cancer a reality. And for successful drug discovery in all its stages, finding subtypes in Alzheimer’s disease is all but imperative. Prior to the approval of the modestly effective Lequembi (lecanemab, Biogen Inc./Eisai Co. Ltd.) Kisunla (donanemab, Eli Lilly and Co.), and the since-withdrawn Aduhelm (aducanumab, Biogen Inc./Eisai Co. Ltd.), more than a dozen failed phase III clinical trials were all that amyloid-targeting drugs had to show for themselves for decades of effort.
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NME Digest: Q2 2025

Aug. 4, 2025
No Comments
A quarterly dynamic table featuring new molecular entities (NMEs) revealed for the first time in current literature, at congresses and in company communications during the quarter. NMEs include compounds chosen for further pharmacological evaluation or as clinical candidates; new leads whose structural optimization could provide new therapeutic agents; new additions to the structural diversity of known mechanistic classes of drugs; and new pharmacological tools for investigating drug targets.
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Infection

Animol Discovery patents cyclic depsipeptides for parasitic infections

Aug. 4, 2025
Animol Discovery Inc. has prepared and tested cyclic depsipeptides reported to be useful for the treatment of parasitic infections.
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Neurology/psychiatric

Wavebreak Therapeutics discovers new α-synuclein aggregation inhibitors

Aug. 4, 2025
Wavebreak Therapeutics LLC has identified thiazole compounds acting as α-synuclein (SNCA) aggregation inhibitors reported to be useful for the treatment of amyloidosis, Creutzfeldt-Jakob disease, frontotemporal dementia, Gaucher disease, Lewy body dementia, multiple system atrophy, progressive supranuclear palsy and Parkinson’s disease, among others.
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Cancer

Systimmune presents ADCs targeting TROP2

Aug. 4, 2025
Systimmune Inc. has synthesized antibody-drug conjugates comprising antibodies targeting tumor-associated calcium signal transducer 2 (TACSTD2; TROP2) covalently linked to cytotoxic drugs through linker reported to be useful for the treatment of cancer.
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Cancer

Vrise Therapeutics identifies new USP1 inhibitors

Aug. 4, 2025
Vrise Therapeutics Inc. has discovered ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
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Cancer

Erasca discloses new GTPase KRAS mutant inhibitors

Aug. 4, 2025
Erasca Inc. has described GTPase KRAS (G12D mutant) and/or KRAS (G12V mutant) inhibitors reported to be useful for the treatment of cancer.
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