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BioWorld - Saturday, December 27, 2025
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Neurology/psychiatric

PN-6047 overcomes unwanted side effects of conventional opioids

Dec. 20, 2024
The regular use of agonists of the μ-opioid receptor for acute pain relief usually leads to tolerance, respiratory depression, constipation and importantly, abuse potential.
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Inflammatory

ATYR-0101 exerts anti-fibrotic activity in lung and kidney

Dec. 20, 2024
Researchers from Atyr Pharma Inc. presented preclinical efficacy data of ATYR-0101, a potential therapeutic biologic based on a domain appended to aspartyl-tRNA synthetase (DARS) in models of lung and kidney fibrosis. By targeting latent-transforming growth factor beta-binding protein 1 (LTBP-1), ATYR-0101 induced apoptosis in myofibroblasts which play a central role in fibrosis and tissue remodeling.
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AI-generated art of brain connections
Neurology/psychiatric

New nonhallucinogenic neuroplasticity-promoting therapeutics reported

Dec. 19, 2024
Researchers from Engrail Therapeutics Inc. recently described the pharmacological characterization of ENX-105, a new class of molecule that combines 5-HT1A/2A receptor agonism with D2/3 receptor antagonism, to be used for its antianhedonic and antipsychotic effects.
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Macrophage releasing cytokines as a part of the body's immune response.
Immune

POLB-001 reduces cytokine release syndrome in mice

Dec. 19, 2024
Cancer immunotherapies are highly effective treatments for treating hematological malignancies, but usually tied to systemic toxicity, including cytokine release syndrome (CRS).
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Cancer

SKY-1214 overcomes nonresponse in B-cell malignancies

Dec. 19, 2024
Skyhawk Therapeutics Inc. presented preclinical data on SKY-1214, a splicing modulator of the Fanconi anemia pathway.
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Neurology/psychiatric

JNJ-69095897 attenuates stress and anxiety in rodents

Dec. 18, 2024
The endocannabinoid system is involved in pain perception, cognition and mood regulation among other functions. The dysregulation in synthesis and degradation of endocannabinoids results in anxiety, depression or degenerative diseases.
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Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia
Cancer

AUTX-703, a KAT2A/KAT2B degrader with efficacy in acute myeloid leukemia models

Dec. 18, 2024
KAT2A is a histone acetyltransferase that functions as a transcriptional activator which, together with its paralogue KAT2B, is markedly overexpressed in acute myeloid leukemia (AML) compared to in hematopoietic progenitor cells.
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Cancer

Simcha’s DR-18 shows efficacy in preclinical hematological malignancies

Dec. 18, 2024
Interleukin-18 (IL-18) is a pro-inflammatory cytokine that modulates innate and adaptive immune responses. Decoy-resistant IL-18, DR-18, from Simcha Therapeutics Inc., is an engineered IL-18 cytokine able to interact with the IL-18 receptor but resistant to IL-18-binding protein (IL18BP), which is a negative regulator of IL-18 signaling, thus overcoming the antitumoral efficacy limitation seen with recombinant IL-18.
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Acute myeloid leukemia
ASH 2024

Kura, Kyowa highlight positive combo data for ziftomenib

Dec. 17, 2024
By Tamra Sami
Kura Oncology, Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor ziftomenib showed encouraging data across multiple studies, the most encouraging of which were in combination with other standard of care therapies in patients with NPM1-mutant and KMT2A-rearranged  acute myeloid leukemia.
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Immuno-oncology

Crossbow reports preclinical data on first development candidate

Dec. 17, 2024
Cathepsin G (CTSG) is overexpressed and aberrantly localized for antigen presentation on acute myeloid leukemia blasts and stem cells compared to normal hematopoietic progenitors. Earlier this year, Crossbow Therapeutics Inc. announced the nomination of its first development candidate, CBX-250, a TCR-mimetic (TCRm) bispecific T-cell engager (TCE) antibody targeting a CTSG peptide-human leukocyte antigen (pHLA) complex and CD3.
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