Investigators at Poseida Therapeutics Inc. developed P-KLKB1-101, a nonviral KLKB1 gene editing therapy, being developed for the treatment of hereditary angioedema (HAE).
Kalvista Pharmaceuticals Ltd. has disclosed coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of hereditary angioedema and thrombotic disorder.
The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.
Positive data from Novo Nordisk A/S’s pivotal phase IIIa study of once-weekly and once-monthly doses of its hemophilia treatment, Mim8, are prompting the company to say it will submit the first regulatory approval request toward the end of this year. It could challenge Roche Holding AG’s Hemlibra (emicizumab), a bispecific factor IXa- and factor X-directed antibody for hemophilia A, that was approved in 2017 by the U.S. FDA.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Killer immunoglobulin-like receptor (KIR)-chimeric antigen receptor (CAR) T-cell therapies have previously demonstrated superior performance and functional persistence in solid tumor models, and the mesothelin-specific KIR-CAR T cells, Synkir-110, are now being evaluated in phase I trials by Verismo Therapeutics Inc.
Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
Researchers from Sichuan Baili Pharmaceutical Co. Ltd. and Systimmune Inc. presented preclinical data for the novel CD33-targeting antibody-drug conjugate (ADC) being evaluated for the treatment of hematologic malignancies.
“Prenatal therapies are the next disruptive technologies in health care, which will advance and shape the future of patient care in the 21st century,” said Graça Almeida-Porada, a professor at the Fetal Research and Therapy Center of the Wake Forest Institute for Regenerative Medicine in Winston-Salem, North Carolina. At the American Society of Gene & Cell Therapy (ASGCT) annual meeting in Baltimore on May 5, 2024, Almeida-Porada introduced the first presentation of the scientific symposium “Prospects for Prenatal Gene and Cell Therapy.”
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