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.”
Researchers have identified enzymes in gut microorganisms that could cleave A and B antigens from red blood, transmuting them to O negative cells. This is “a decisive step forward” in the quest to develop a universal donor blood that can be administered to people of any blood group without eliciting a harmful immune response, according to Maher Abou Hachem of the Technical University of Denmark, who co-led the research.
Amidst a slew of end-of-week, positive EMA Committee for Medicinal Products for Human Use (CHMP) opinions is Takeda Pharmaceutical Co. Ltd.’s Fruzaqla (fruquintinib). The selective inhibitor of vascular endothelial growth factor receptors-1, -2 and -3 is for adults with previously treated metastatic colorectal cancer.
The FDA has cleared Prime Medicine Inc.’s IND application for PM-359 for the treatment of chronic granulomatous disease (CGD), enabling initiation of a phase I/II trial in the U.S.
Amidst a slew of end-of-week, positive EMA Committee for Medicinal Products for Human Use (CHMP) opinions is Takeda Pharmaceutical Co. Ltd.’s Fruzaqla (fruquintinib). The selective inhibitor of vascular endothelial growth factor receptors-1, -2 and -3 is for adults with previously treated metastatic colorectal cancer.
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
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