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Preclinical conference data for May 18, 2023: ASGCT

May 18, 2023
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: 2seventy Bio, Dyne Therapeutics, In8bio, Kelonia Therapeutics, Siren Biotechnology, Umoja Biopharma.
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Gastrointestinal

LPA1 receptor agonist improves NASH-related outcomes in preclinical models

May 18, 2023
Currently, there is no FDA-approved drug for nonalcoholic steatohepatitis (NASH), which has evolved into the second leading cause of liver transplantation in the U.S. Researchers from Children’s Hospital Los Angeles and Epigen Biosciences Inc. disclosed preclinical data on EPGN-2154, a novel lysophosphatidic acid LPA1 receptor agonist that has already demonstrated antifibrotic activity in preclinical kidney and liver models.
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Infection

Immunic presents XAVI-101, a novel DHODH inhibitor with broad-spectrum antiviral properties

May 18, 2023
Dihydroorotate dehydrogenase (DHODH) is an enzyme ubiquitously expressed in all tissues whose inhibition has proven effective in preventing viral replication. Previous knowledge on DHODH inhibitor vidofludimus calcium demonstrated that this compound inhibits viral replication of DNA, RNA and retroviruses and induces innate immune responses that lead to an antiviral state thus inhibiting virus reactivation.
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Antibodies and red blood cells
Immune

Hummingbird Bioscience HMBD-011 shows promise for autoimmune disorders

May 18, 2023
VH4-34 is an autoreactive antibody heavy chain variable region; the autoantigens it recognizes...
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Illustration of pancreas
Endocrine/Metabolic

Gene therapy to reverse T2D shows proof of concept at ASGCT

May 18, 2023
By Mar de Miguel
The most ambitious objective of any treatment is to eradicate the disease, acting on its origin to cure it instead of treating its symptoms. This is the purpose of the gene therapy against type 2 diabetes (T2D) and obesity that Fractyl Health Inc. is developing. Scientists from the Lexington, Mass.-based company have designed a strategy based on glucagon-like peptide-1 (GLP-1) to transform pancreatic cells and reverse the disease.
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Light micrograph and 3D illustration of small intestine villi
Gastrointestinal

Researchers present data from murine studies of PAM-369, a muscarinic M3 receptor PAM for enteropathy

May 17, 2023
Researchers from Kyushu University and Mochida Pharmaceuticals Co. Ltd. disclosed recent data along with the chemical structure of PAM-369, a novel muscarinic acetylcholine M3 receptor positive allosteric modulator (PAM), being developed for the potential prevention or treatment of NSAID-induced enteropathy.
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Microscope and coronavirus illustration
Infection

Preclinical efficacy of ALG-097558 in SARS-CoV-2 hamster model presented

May 17, 2023
Researchers from Aligos Therapeutics Inc. and the Katholieke Universiteit Leuven reported on the preclinical activity of ALG-097558, a novel oral available 3CL protease inhibitor with pan-coronavirus antiviral activity.
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Dermatologic

PARP14 inhibitor RBN-3143 shows efficacy in inflammation models

May 17, 2023
PARP14 is an ADP-ribosyltransferase that transfers ADP-ribose from NAD+ onto the appropriate protein target. PARP14 selectively binds to STAT6 and promotes expression of IL-4-regulated genes, thus modulating inflammatory signaling pathways. PARP14 is overexpressed in tissues from patients with dermatomyositis, atopic dermatitis, psoriasis and other inflammatory disorder cells compared to normal tissues.
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Preclinical conference data for May 17, 2023: ASGCT

May 17, 2023
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: Abeona Therapeutics, Arsenal Biosciences, Cimeio Therapeutics, Homology Medicines, Locanabio, Precision Biosciences, Wave Life Sciences.
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3D rendering conceptualizing theranostics
Cancer

Bicycle’s radionuclide conjugate [212Pb]BCY-20603 shows potent preclinical antitumor activity

May 16, 2023
Targeted alpha therapy allows the selective delivery of potent, alpha particle-emitting...
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