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BioWorld - Wednesday, April 29, 2026
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3D illustration and light micrograph of lung cancer.
Cancer

KO-2806 demonstrates potential as combination partner in models of treatment-resistant NSCLC and CRC

Nov. 14, 2024
The next-generation farnesyl transferase inhibitor KO-2806 is currently in phase I clinical development at Kura Oncology Inc. for the treatment of patients with solid tumors, including non-small-cell lung cancer (NSCLC) and colorectal cancer (CRC).
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Eye and DNA
Biomarkers

Iron levels in tears for diagnosis of WDR45-related optic atrophy

Nov. 14, 2024
WDR45 is located on the X chromosome. Its pathogenic variants are associated with various neurodegenerative disorders that are predominantly reported in females and present a wide range of clinical phenotypes, from early-onset developmental delay to neurodegeneration and multiple epileptic syndromes.
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Immuno-oncology

Bolt presents immune-stimulating antibody-drug conjugate

Nov. 14, 2024
Claudin (CLDN) 18.2 is a transmembrane tight junction protein overexpressed in multiple cancers, including gastric, esophageal and pancreatic cancers.
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Endocrine/metabolic

Islet-targeted gene therapy RJVA-001 leads to durable weight loss in obese mice

Nov. 14, 2024
Researchers from Fractyl Health Inc. presented preclinical data for RJVA-001, a novel pancreatic gene therapy.
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Adenovirus cells
Neurology/psychiatric

Preclinical data on SPK-10001 AAV-based gene therapy released

Nov. 14, 2024
Spark Therapeutics Inc. has presented preclinical data on SPK-10001, an engineered adeno-associated virus (AAV).
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3D illustration of tumor
Cancer

CK1α-selective degrader has activity in multiple tumor models

Nov. 13, 2024
CK1α serves as an upstream regulator of the p53 pathway, and its degradation may facilitate cell death by preventing MDM2 and/or MDMX mediated inactivation of p53.
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Mouth open wide showing cleft palate
Biomarkers

GRHL2, GRHL3 variants tied to orofacial clefting

Nov. 13, 2024
Orofacial clefting is a common birth defect that affects 1 in 700 newborns, and includes cleft lip, cleft palate and cleft lip and palate, with a strong genetic component, thus being highly heritable. Researchers have identified new gene variants tied to orofacial clefting.
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Immuno-oncology

Sonata’s SNT-3012 shows antitumor activity in preclinical models

Nov. 13, 2024
The immunological targeting of cold tumors still remains a challenge. Cytokine therapy is promising but has modest efficacy and significant toxicity.
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3D representation of tumor microenvironment
Immuno-oncology

Trispecific antibody CS-2009 has in vivo activity

Nov. 13, 2024
Cstone Pharmaceuticals Co. Ltd. presented preclinical data of CS-2009, a trispecific antibody targeting PD-1, CTLA-4 and VEGF-A, for cancer immunotherapy.
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Genetic/congenital

GTF2H1 de novo genetic variants tied to developmental delay

Nov. 13, 2024
At the annual meeting of the American Society for Human Genetics, scientists presented three new cases of Kabuki syndrome presented with developmental delay, autism and myopia as the common traits.
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