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Preclinical conference data for May 22, 2023: ASGCT

May 22, 2023
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: Epic Bio, Kalivir Immunotherapeutics, Rejuvenate Bio.
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Other news to note for May 22, 2023

May 22, 2023
Additional early-stage research and drug discovery news in brief, from: Navrogen, Noxopharm.
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Endocrine/Metabolic

Terns Pharmaceuticals divulges new GLP-1 receptor agonists for diabetes and liver diseases

May 22, 2023
Glucagon-like peptide 1 (GLP-1) receptor agonists have been reported by Terns Pharmaceuticals Inc. in a recent patent. They are described as potentially useful for the treatment of diabetes and liver diseases.
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Neurology/Psychiatric

Lundbeck and Vernalis jointly patent LRRK2 inhibitors

May 22, 2023
H. Lundbeck A/S and Vernalis (R&D) Ltd. have jointly patented leucine-rich repeat kinase 2 (LRRK2; Dardarin) and/or LRRK2 mutant inhibitors reported to be useful for the treatment of Lewy body dementia, multiple system atrophy and Parkinson’s disease.
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Cancer

Voronoi identifies new PDGFR/c-Kit inhibitors

May 22, 2023
A Voronoi Bio Inc. and Voronoi Inc. patent describes platelet-derived growth factor receptor (PDGFR), mast/stem cell growth factor receptor kit (KIT; c-Kit; CD117) and their mutant inhibitors reported to be useful for the treatment of cancer, fibrosis, neurological diseases, atherosclerosis, and pulmonary hypertension.
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Respiratory

SERT inhibitors detailed in Sensorium Therapeutics patent

May 22, 2023
Research at Sensorium Therapeutics Inc. has led to the identification of deuterated alkaloids acting as serotonin transporter (SERT) inhibitors reported to be useful for the treatment of asthma, chronic obstructive pulmonary disease, depression, rheumatoid arthritis, stress and anxiety disorders.
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Neurology/Psychiatric

Zywie patents polydeuterated analogues of ambroxol and bromhexine

May 22, 2023
Polydeuterated analogues of ambroxol and bromhexine have been reported in a Zywie LLC patent as potentially useful for the treatment of aging, Parkinson’s and Alzheimer’s diseases, dementia with Lewy bodies and frontotemporal dementia.
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Acute myeloid leukemia
Immuno-oncology

Pretreatment with decitabine enhances antileukemia activity of novel CD33 CAR T cells

May 22, 2023
Researchers from City of Hope discussed preclinical data for CD33-targeted chimeric antigen receptor (CAR) T cells being developed for the treatment of acute myeloid leukemia (AML).
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Neurology/Psychiatric

Solid Biosciences’ AVB-202 ameliorates symptoms in preclinical Friedreich’s ataxia

May 22, 2023
Friedreich’s ataxia (FA) is an autosomal recessive disorder caused by mutations in the FXN gene and characterized by cardiomyopathy, gait ataxia and sensory loss, with cardiac complications the main cause of death among patients with FA.
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Blood cell, test tubes, dropper
Hematologic

HMI-104 inhibits hemolysis in murine model of paroxysmal nocturnal hemoglobinuria

May 22, 2023
Homology Medicines Inc. has reported data on an adeno-associated viral (AAV) vector-based therapy, HMI-104, an AAV treatment intended to induce hepatic expression of a complement C5 monoclonal antibody, named as C5mAb, for the potential treatment of paroxysmal nocturnal hemoglobinuria (PNH) as well as other complement-driven pathologies. C5mAb is thought to bind to C5 and inhibit the C5-mediated hemolysis observed in PNH.
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