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BioWorld - Sunday, December 21, 2025
Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Brain and DNA
Neurology/psychiatric

Grant supports early studies of SBT-589 for Friedreich’s ataxia

Sep. 12, 2024
Stealth Biotherapeutics Inc. has been awarded the Kyle Bryant Translational Research Award from the Friedreich’s Ataxia Research Alliance (FARA) to evaluate mitochondria-targeted molecule SBT-589 in Friedreich’s ataxia (FA).
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Drug design, drug delivery & technologies

Gilead and Genesis collaborate on drug discovery

Sep. 12, 2024
Gilead Sciences Inc. and Genesis Therapeutics Inc. have entered into a strategic collaboration to discover and develop novel small-molecule therapies across multiple targets.
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Kidneys
Nephrology

Keap1-Nrf2 interaction inhibitors show activity in rat kidneys

Sep. 12, 2024
Researchers from Japan Tobacco Inc. presented the discovery and preclinical characterization of novel highly potent Keap1-Nrf2 protein-protein interaction (PPI) inhibitors.
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Neurology/psychiatric

MK-4334 ameliorates cognition in Alzheimer’s disease models

Sep. 12, 2024
Neuroinflammation underlies neurodegeneration in Alzheimer’s disease (AD) and other brain disorders. The stimulation of nicotinic α7 receptors, involved in the regulation of neuroinflammation, has shown efficacy in improving memory and learning in AD models.
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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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