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BioWorld - Friday, June 19, 2026
Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Illustration of motor neuron connecting to muscle fiber
Neurology/psychiatric

Grant supports study of NRG Therapeutics’ mPTP inhibitors for ALS

Sep. 5, 2024
NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).
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Man clutching heart
Cardiovascular

Pfizer presents data on PF-07328948 for the treatment of heart failure

Sep. 5, 2024
Essential branched-chain amino acids (BCAA) such as leucine, isoleucine or valine, cannot be synthesized by mammals, making them an obligate part of the diet.
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Heart scientific overlay
Cardiovascular

AZD-0780 lowers LDL-C plasma levels by stabilizing the PCSK9 C-terminal domain

Sep. 5, 2024
Researchers from Astrazeneca plc presented the structure and preclinical characterization of a novel PCSK9 inhibitor, AZD-0780, being developed for the treatment of cardiovascular disease. A new potentially druggable functional binding pocket was identified on the PCSK9 C-terminal domain (CTD), and multiple fragment screens generated a single CTD-binding hit.
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3D dollar sign
Cancer

Cumulus raising $50M, spinning GPR68 small molecules into GIO

Sep. 2, 2024
By Nuala Moran
Cumulus Oncology Ltd. is in the thick of raising a $50 million series A round as its model of sourcing novel drug targets emerging from academia, shaping them up for clinical development, and spinning them into startups, gathers pace. At the same time, Nodus Oncology Ltd., the first spinout created around an acquired asset, has just reached in vivo proof of concept with its lead DNA damage response inhibitor, and it, too, is looking to raise a series A to take the program through to the end of phase I.
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Neurology/psychiatric

Vandria’s series A funds spur development of mitophagy inducer

Aug. 22, 2024
By Nuala Moran
After sparking further interest from investors after the close of its series A, Vandria SA has extended the round and now has the means to advance its lead mitophagy inducer program as far as phase Ib/IIa development in the treatment of mild cognitive impairment.
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Pill in immersive interface
Immune

Deepcure selects DC-9476 for autoimmune diseases

Aug. 13, 2024
Deepcure Inc. has selected its first artificial intelligence (AI)-generated candidate, DC-9476, for autoimmune disease.
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Ischemic stroke
Cardiovascular

Esculetin promotes stroke rehabilitation by inhibiting CKLF1

Aug. 13, 2024
Esculetin, also known as 6,7-dihydroxycoumarin, is a coumarin-derived phytochemical that has shown effects against ischemic stroke. Additionally, chemokine-like factor 1 (CKLF1) has been revealed in previous findings to be a target for stroke when using coumarin-derived compounds. The effects of esculetin and the potential involvement of CKLF1 were tested in photothrombotic stroke in mice.
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Man holding hand up to ear
Ear, nose & throat

Piplartine counteracts antibiotic-induced hearing loss

Aug. 13, 2024
Piplartine, also called piperlongumine, had protective effects against hearing loss induced by aminoglycoside antibiotics in zebrafish and mouse models of kanamycin-induced hair cell loss. Kanamycin is a member of the aminoglycosides, a group of antibiotics that have broad-spectrum activity against gram-negative bacteria, including multidrug resistant strains.
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Doctor points at molecular structure
Cancer

Halda nabs $126M, publishes proof of concept

Aug. 12, 2024
Halda Therapeutics Inc. announced it has secured an additional $126 million in funding in a series B extension, bringing its total funding to date to $202 million. The company expects its lead molecule HLD-0915 to enter a phase I trial in the first half of 2025 for the treatment of patients with metastatic castration-resistant prostate cancer (mCRPC).
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3D illustration of a nerve cell
Neurology/psychiatric

Negative allosteric modulator of TrkA has efficacy in pain models

Aug. 9, 2024
Researchers from Alzecure Pharma AB presented preclinical data for ACD-137, a negative allosteric modulator (NAM) of tropomyosin receptor kinase A (TrkA), being developed for the treatment of pain.
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