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.

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.

Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.

Alltrna Inc. has presented new preclinical data on the company’s first transfer RNA (tRNA) development candidate, AP-003.

In one of the largest private rounds raised by an Italian biotech, Genespire Srl has closed a €46.6 million (US$51.88 million) series B, enabling it to lay the ground for a phase I/II clinical trial of its lead program, Gene-202, and to further develop its proprietary lentiviral vectors. The vectors are designed to be applicable to a range of liver-related metabolic disorders and, as its first indication, the company intends to treat methylmalonic acidemia, a serious genetic condition that results in impaired metabolism of certain amino acids and lipids.

The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on a clinical hold. So far, four patients have been dosed in the study and two have had serious adverse events related to the candidate, the company’s lead asset.