A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this threat, surgeons operate inside the uterus in an open procedure that partially exposes the baby to remove the tumor and give the baby a chance to survive until birth. According to scientists presenting at the American Society of Gene & Cell Therapy's special meeting on Breakthroughs in Targeted In Vivo Gene Editing, this could be avoided.
The U.S. FDA said postmarketing reports of neutralizing antibodies to ADAMTS13, including one reported patient death, have prompted an investigation into Takeda Pharmaceutical Ltd.’s Adzynma (apadamtase alfa), a recombinant protein product approved in 2023 for use in adults and pediatric patients with congenital thrombotic thrombocytopenic purpura.
The field of gene therapy is experiencing major advances driven by precise editing technologies, such as base and prime editing, and by the design of increasingly sophisticated vectors to deliver payloads that could reverse the effects of diseases. However, in the transition to in vivo applications many approaches still fail in their attempt to effectively reach target tissues or cells.
“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints.
Fifteen years since the first patient was treated, and after being ditched by two companies, the EMA is recommending approval of Waskyra (etuvetidigene autotemcel), the first gene therapy for treating Wiskott-Aldrich syndrome.
Metagenomi Inc. has reported new dose-range-finding data from its MGX-001 hemophilia A program. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children with hemophilia A.
City Therapeutics Inc. has submitted a clinical trial application (CTA) to the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a phase I study of CITY-FXI, an investigational RNAi therapeutic targeting factor XI (FXI) for the treatment of thromboembolic diseases.
Simcere Pharmaceutical Co. Ltd. has described stachybotrys microspore triprenyl phenols (SMTPs) acting as plasminogen activators reported to be useful for the treatment of thromboembolism.
Just as investors were looking ahead to news by year-end on Prelude Therapeutics Inc.’s SMARCA2-targeted degraders, the firm said work in the space will be halted, with efforts shifting toward the mutant selective JAK2V617F JH2 inhibitor program by way of a new deal with Incyte Corp.
Alethio Therapeutics (formerly known as Alethiomics Ltd.) has emerged from stealth with two antibody-drug conjugate (ADC) programs for the treatment of myeloproliferative neoplasms (MPNs).
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