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BioWorld - Friday, June 26, 2026
Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Lab sample and bone marrow illustration
Cancer

ALK5 inhibitors promote erythropoiesis in myelodysplasia

Jan. 23, 2025
Myelodysplastic syndromes (MDS) are marked by ineffective hematopoiesis and dysplasia, which elevate the risk of progression to acute myeloid leukemia (AML). The inflammatory environment in MDS alters the bone marrow niche, facilitating the growth of cancerous cells while inhibiting the healthy formation of blood cells.
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Scientist, microscope and dropper
Hematologic

Immvention, Novo Nordisk collaborate on BACH1 inhibitors

Jan. 23, 2025
Immvention Therapeutix Inc. has entered into a collaboration and license agreement with Novo Nordisk A/S to co-develop oral therapies for sickle cell disease and other chronic conditions.
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Test tubes, dropper and capsules
Neurology/psychiatric

Pheno gets clinical trial clearance for remyelination therapeutic

Jan. 15, 2025
Pheno Therapeutics Ltd. has received clinical trial authorization (CTA) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its lead candidate, PTD-802. The program will progress to a first-in-human phase I trial.
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Photo of older woman holding hand that's colored red
Inflammatory

mPGES-1 inhibitor AGU-654 is anti-inflammatory and analgesic

Jan. 8, 2025
Researchers from Gazi University and Friedrich-Schiller-Universität Jena presented the discovery and preclinical characterization of novel microsomal prostaglandin E2 synthase 1 (mPGES-1) inhibitors as potential anti-inflammatory and analgesic drug candidates.
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Drug R&D concept image.
Cancer

Rakovina advances synthesis of PARP1-selective inhibitors

Jan. 7, 2025
Rakovina Therapeutics Inc. has announced the successful synthesis of novel small-molecule drug candidates designed under the company’s artificial intelligence (AI) platform collaboration.
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Musculoskeletal

Epirium’s MF-300 cleared to enter clinic for sarcopenia

Dec. 24, 2024
Epirium Bio Inc. has obtained IND clearance from the FDA for MF-300, a first-in-class orally administered, 15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor.
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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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AI-generated illustration of spinal cord
Neurology/psychiatric

Dewpoint and Mitsubishi ink $480M ALS research collaboration

Dec. 10, 2024
By Tamra Sami
Dewpoint Therapeutics Inc. and Mitsubishi Tanabe Pharma Corp. have entered a research collaboration worth up to $480 million to advance Dewpoint’s novel TDP-43 small-molecule condensate modulator for amyotrophic lateral sclerosis. Under terms of the deal, Boston-based Dewpoint will receive an undisclosed up-front payment and is eligible to receive R&D-based milestone payments up to $480 million. Upon reaching those milestones, Osaka, Japan-based MTPC will have an exclusive option to license the program and assume responsibility for global clinical development and commercialization. Dewpoint will also receive tiered royalties on net sales.
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SARS-CoV-2 illustration turns from blue to red
Infection

Sunshine Biopharma’s PLpro inhibitor shows activity in SARS-CoV-2

Dec. 10, 2024
Sunshine Biopharma Inc. announced it has developed an orally active protease inhibitor that showed dose-dependent antiviral activity in mice infected with SARS-CoV-2.
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Esophageal cancer
Cancer

ML-385 enhances radiotherapy sensitivity in esophageal cancer models

Dec. 9, 2024
Chinese researchers described ML-385.
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