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.
HER2-targeted antibody-drug conjugates (ADCs) have significantly improved cancer therapy in recent decades. However, ADCs can cause toxicity and resistance, while T-cell engagers (TCEs) show promising antitumor activity in solid tumors but are limited by substantial adverse effects.
Researchers from Synthekine Inc. presented preclinical efficacy data on STK-012, a first-in-class IL-2 partial agonist engineered for preferential binding to the IL-2Rαβγ receptor.
High-grade gliomas, including glioblastomas, are aggressive cancers of the brain that usually recur despite standard treatment, including radiation therapy. These mechanisms conferring persistence to tumor cells may be explained by DNA damage response pathways, especially those relying on ataxia-telangiectasia mutated (ATM) and DNA-dependent protein kinase (DNA-PK), which induce the repair of radiation-induced DNA damage. The effects of the ATM/DNA-PK inhibitor XRD-0394 were evaluated in in vitro models of glioma.
Pancreatic cancer is among the most aggressive cancer types, with survival rates being very low and current treatment being quite ineffective. To address this unmet medical need, HCW Biologics Inc. has developed and presented preclinical data for their T-cell engager approach – HCW11-018.
M-3554 is an antibody-drug conjugate (ADC) targeting GD2; it has shown strong antitumor activity in neuroblastoma xenograft models and has been engineered to reduce anti-GD2 antibody-associated pain.
Vivace Therapeutics Inc. reported preclinical efficacy data of VT-3989, a TEAD inhibitor, in in vitro and in vivo models of aggressive meningioma. VT-3989 is in early clinical development for the treatment of patients with advanced solid tumors.
Researchers from Tango Therapeutics Inc. presented preclinical efficacy data on TNG-456, a selective MTA-cooperative PRMT5 inhibitor under development for the treatment of glioma and other tumors that frequently metastasize to the brain, such as non-small-cell lung cancer.
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.
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.
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