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BioWorld - Monday, April 6, 2026
Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Brain and encephalography
Neurology/psychiatric

Jazz gains rights to Saniona’s SAN-2355

Aug. 21, 2025
No Comments
Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications.
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Heart and lungs
Cardiovascular

VTB-10 gets IND clearance for pulmonary arterial hypertension

Aug. 20, 2025
No Comments
Vasthera Co. Ltd. has received IND clearance from the U.S. FDA, enabling it to initiate a phase I trial for VTB-10 for pulmonary arterial hypertension (PAH). Vasthera identified a deficiency of the enzyme peroxiredoxin (PRX) in PAH lesions and used its Redoxizyme platform to develop VTB-10, a small-molecule enzyme that precisely replicates PRX function.
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Handshake with dollar sign and checkmark
Neurology/psychiatric

Skyhawk’s small-molecule RNA efforts draw Merck KGaA in $2B deal

Aug. 19, 2025
By Jennifer Boggs
No Comments
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
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Illustration of cancer tumor
Immuno-oncology

Naphthalene and indane derivatives as selective USP7 inhibitors

Aug. 19, 2025
No Comments
Researchers from China Pharmaceutical University and collaborators have identified various naphthalene and indane derivatives as selective USP7 inhibitors.
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Test tube, dropper
Cancer

Chinese researchers unveil a potent eIF4E/eIF4G interaction disruptor

Aug. 11, 2025
No Comments
Overexpression of the eukaryotic translation initiation factor 4E (eIF4E) has been observed in a wide range of tumors, where it is associated with malignant transformation, tumor progression, poor prognosis, and resistance to therapy.
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Melanoma cells stained with an H & E stain and magnified to 320x.
Cancer

SYHA-1813: A dual inhibitor against melanoma

Aug. 11, 2025
No Comments
Melanoma is one of the most mutation-prone cancers, with 90% of cases involving the V600E mutation in BRAF kinase. Several therapies are available against melanoma, but each one is associated with substantial drawbacks. Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and collaborators reasoned that it might be effective to simultaneously inhibit both angiogenesis and immunosuppression in the tumor microenvironment.
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Illustration of DNA methylation by the DNA methyl transferase I
Cancer

NSD2 inhibitors close chromatin and silence aggressive oncogenes

Aug. 11, 2025
By Mar de Miguel
No Comments
Experimental drugs that directly inhibit the NSD2 enzyme have shown potential as an effective strategy against hard-to-treat cancers, such as lung and pancreatic tumors driven by KRAS mutations. The therapeutic mechanism involves reversing a histone H3 methylation that promotes open chromatin and the expression of oncogenes.
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Gregory Verdine, founder and CEO, Dovetree
Drug design, drug delivery & technologies

Xtalpi finalizes $6B AI drug discovery deal with Dovetree

Aug. 8, 2025
By Marian (YoonJee) Chu
No Comments
Quantumpharm Inc., known as Xtalpi Inc., announced receiving $51 million up front from a potential $5.99 billion deal with Dovetree LLC on Aug. 6. The collaboration, first inked through a letter of intent between the two parties on June 23, will combine Shenzhen, China-based Xtalpi’s AI-based and robotics-driven discovery platform with Dovetree’s “biological insights.” The goal will be to select and validate potential first-in-class candidates for Dovetree across five areas of oncology, immunology and inflammatory diseases, neurological disorders and metabolic dysregulation.
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Test tube, dropper, DNA illustration
Cancer

K36 Therapeutics gains IND clearance for NSD2 inhibitor KTX-2001

Aug. 8, 2025
No Comments
K36 Therapeutics Inc. has obtained IND clearance from the U.S. FDA for KTX-2001, a selective, oral, small-molecule inhibitor of nuclear receptor-binding SET domain protein 2 (NSD2), a histone methyltransferase and oncogene that activates gene expression in some cancers.
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Musculoskeletal

MSX2 plays role in postmenopausal osteoporosis, bone metabolism

Aug. 8, 2025
No Comments
Homeobox genes, a conserved family of transcription factors, are key regulators of embryogenesis, cell growth and differentiation, and have been linked to bone mass regulation and osteogenesis. However, their specific roles in postmenopausal osteoporosis and osteoclast development are still not well understood, with limited and fragmented knowledge on which genes are central to these processes.
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