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

Small molecule, BioWorld Science
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3D dollar sign
Neurology/psychiatric

Augustine Therapeutics closes $85M series A for HDAC6 inhibitors

March 26, 2025
By Nuala Moran
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
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Inflammatory

Novartis adds Kyorin’s preclinical MRGPRX2 antagonist in $777M deal

March 5, 2025
By Tamra Sami
Kyorin Pharmaceutical Co. Ltd. has out-licensed its preclinical candidate, KRP-M223, and its back-up compounds to Novartis Pharma AG in a deal worth up to $777.5 million. Under the terms, Novartis gains an exclusive worldwide license to develop, manufacture and commercialize Kyorin-discovered KRP-M223.
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3D illustration of cancer in crosshairs
Cancer

Laekna’s USP1 inhibitor LAE-120 gains IND clearance for solid tumors

March 4, 2025
Laekna Inc. has obtained FDA clearance of its IND application for LAE-120, an allosteric and highly potent USP1 inhibitor, for the treatment of advanced solid tumors.
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AI-generated depiction of lungs affected by cystic fibrosis
Respiratory

‘Remarkable’ progress in cystic fibrosis means more work needed

Feb. 28, 2025
By Nuala Moran
The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer.
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Endocrine/metabolic

Septerna to change candidate for hypoparathyroidism

Feb. 19, 2025
Septerna Inc. has announced its decision to discontinue a healthy volunteer phase I trial of SEP-786, an oral small-molecule agonist of the parathyroid hormone 1 receptor being developed for the treatment of hypoparathyroidism.
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Close up of eye and vision test
Ocular

Perceive Pharma secures Series A for ophthalmic diseases

Feb. 19, 2025
Perceive Pharma Inc., a spin out from Perceive Biotherapeutics Inc., has closed a $15 million series A funding round to help it advance first-in-class therapies to prevent vision loss in glaucoma, and additional undisclosed disease areas.
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Lungs
Infection

Bioversys identifies optimized lead compounds in BV500 NTM program

Feb. 12, 2025
Bioversys AG has announced that its BV500 NTM program has reached its second milestone with the identification of up to five optimized lead compounds, under the CF AMR Syndicate collaborative discovery program agreement.
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Kidneys
Nephrology

HIF-prolyl hydroxylase inhibitor MK-8617 promotes renal tubule regeneration

Jan. 27, 2025
Acute kidney injury (AKI) may progress to chronic kidney disease, and there is an urgent need for approaches that improve the limited regeneration of the renal tubules after AKI.
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Closeup of red eye
Immune

AMG-487 dampens inflammation in autoimmune uveitis model

Jan. 24, 2025
Researchers from Sun Yat-Sen University and affiliated organizations presented data from a study that aimed to investigate mechanisms by which the CXC chemokine receptor 3 (CXCR3) antagonist, AMG-487, acts on autoimmune uveitis.
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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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