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BioWorld - Tuesday, May 5, 2026
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Neurology/psychiatric

Innovstone Therapeutics divulges new LRRK2 inhibitors for Parkinson’s disease

April 4, 2025
Innovstone Therapeutics Ltd. has synthesized leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
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DNA testing illustration
Policy

With ‘wholesale assault’ on research, bipartisan alarm at institutional failure

April 4, 2025
By Anette Breindl
Biomedical research seems like it should be the ultimate bipartisan issue. But under the Trump administration, unless and until Congress regains its will to make use of its constitutional powers, bipartisan support for research seems to be a thing of the past. On March 3, members of the National Academies of Science, Engineering and Medicine warned that the second Trump administration has been waging a “wholesale assault” on American research.
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Neurology/psychiatric

Lilly licenses rights to use Sangamo’s neurotropic AAV capsid in genomic medicines

April 4, 2025
Sangamo Therapeutics Inc. has entered into a license agreement with Eli Lilly and Co., allowing Lilly to use Sangamo’s novel proprietary neurotropic AAV capsid, STAC-BBB, to deliver intravenously administered genomic medicines to treat certain diseases of the central nervous system.
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Cancer

Moma Therapeutics patents new WRN inhibitors

April 4, 2025
Moma Therapeutics Inc. has disclosed tricyclic derivatives acting as Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
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Colorectal cancer illustration
Immuno-oncology

Targeting the arachidonic acid pathway sensitizes ARID1A-deficient colorectal tumors to ICIs

April 4, 2025
AT-rich interactive domain-containing protein 1A (ARID1A) encodes a switch/sucrose nonfermentable (SWI/SNF) complex and is mutated in around 10% of colorectal cancers. ARID1A deficiency damages DNA damage repair increasing tumor mutation burden in ovarian and gastrointestinal cancers.
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Photo of magnifying glass inspecting moles on person's back
Biomarkers

INSM1 is highly sensitive marker in primary and metastatic Merkel cell carcinoma

April 4, 2025
The detection of metastases in Merkel cell carcinoma (MCC) requires additional testing, such as pancytokeratin (panCK) or CK20, with no guarantee of success. Recent findings have identified transcription factor SOX11 and insulinoma-associated protein 1 (INSM1) as sensitive nuclear neuroendocrine markers, but their usefulness in MCC has not been proven.
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3D rendering of skin cells and elastin with collagen layer
Dermatologic

Resvita Bio’s RVB-003 designated orphan drug for Netherton syndrome

April 4, 2025
Resvita Bio Inc.’s RVB-003 has been awarded orphan drug designation by the FDA for the skin disorder Netherton syndrome. RVB-003 was previously granted rare pediatric disease designation.
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Parkinson's disease illustration showing neurons containing alpha-synuclein
Neurology/psychiatric

Preclinical results of SPK-10005 as a potential miRNA-based therapeutic for synucleinopathies

April 4, 2025
Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying cause of synucleinopathies.
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Neurology/psychiatric

DYRK1A inhibitors for treating Alzheimer’s disease presented at ACS Spring

April 4, 2025
Dual-specificity tyrosine phosphorylation-regulated kinase 1A (DYRK1A) is an attractive therapeutic target due to its involvement in cancer and neurodegenerative diseases. Researchers from the National Health Research Institutes and their collaborators have presented a series of DYRK1A inhibitors for reducing neurofibrillary tangle formation  in Alzheimer’s disease.
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Enterobacteriaceae
Infection

Arrepath’s antibiotic targeting Enterobacterales receives CARB-X funding

April 4, 2025
Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) will award Arrepath Inc. $3.7 million to execute a lead optimization workplan for its first-in-class antibiotic targeting a clinically novel target for the treatment of complicated urinary tract infections caused by multidrug-resistant Enterobacterales.
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