Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification. Read More

Heart disease caused by damage to blood vessels is the leading cause of death worldwide. Arteries become clogged with fats and cholesterol when certain proteins in the body, known as lipoproteins, combine with and transport fats in the blood to cells. Scientists have long believed that the LDL receptor molecule was responsible for the intracellular transport of LDL. But given that some individuals lacking the LDL receptor still have high levels of LDL, questions remain about the mechanism. Read More

At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD). Read More

Laekna Therapeutics Shanghai Co. Ltd. has received FDA clearance of its IND application to initiate clinical development with LAE-102 in the U.S. Read More

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. Read More

Trigeminal neuralgia (TN) is a chronic disorder caused by the hyperactive functioning of a damaged trigeminal nerve that provokes severe facial pain coming from the trigeminal nerve. Read More

Arrhythmogenic cardiomyopathy (ACM) is a devastating inherited disorder characterized by massive cardiomyocyte loss, fibrofatty infiltration and ventricular arrhythmias, among others. Most known genetic causes of ACM involve the gene PKP2, which encodes plakophilin-2. An unmet medical need exists regarding therapies that correct this PKP2 deficiency. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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. Read More

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). Read More

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. Read More

Additional early-stage research and drug discovery news in brief, from: Navrogen, Noxopharm. Read More

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. Read More