“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).
An oral Bruton’s tyrosine kinase (BTK) inhibitor that Sanofi SA acquired in 2020 through its $3.68 billion buyout of Principia Biopharma Inc. is headed toward regulatory filings in the U.S and EU by the end of the year, following phase III data in immune thrombocytopenia.
Agios Pharmaceuticals Inc.’s fourth-quarter and full-year 2023 financial report in mid-February highlighted further prospects with Pyrukynd (mitapivat), the pyruvate kinase activator for hemolytic anemia in adults with pyruvate kinase deficiency (PKD).
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