The Recombinant DNA Advisory Committee (RAC) meeting in Washington, D.C., Dec. 8-10 delved into every aspect of Jesse Gelsinger's death. Gelsinger, a teenager from Arizona, had ornithine ...

This was the case with 18-year-old Jesse Gelsinger, who in September ... undergo gene therapy (for the rare condition ornithine transcarbamylase deficiency) but who died as a result.

The recent tragic and widely publicised death of Jesse Gelsinger in a gene therapy trial has ... Jesse was an 18-year-old man with a mild form of ornithine transcarbamylase (OTC) deficiency, a ...

In January, our partner iECURE announced news from the OTC-HOPE study, where first-in-human data demonstrated a complete clinical response in severe neonatal OTC deficiency using an ARCUS nuclease ...

Furthermore, the team succeeded in editing genes to replicate ornithine transcarbamylase deficiency, a genetic disorder disrupting the urea cycle. This achievement advances methods for modeling ...

The corporation’s gene therapy pipeline includes promising candidates such as UX111 (for Mucopolysaccharidosis Type IIIA), DTX301 (for Ornithine Transcarbamylase deficiency), DTX401 (for ...

Furthermore, the team succeeded in editing genes to replicate ornithine transcarbamylase deficiency, a genetic disorder disrupting the urea cycle. This achievement advances methods for modeling ...

evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. In January 2025, iECURE reported clinical efficacy and safety data in the first patient ...

Furthermore, the team succeeded in editing genes to replicate ornithine transcarbamylase deficiency, a genetic disorder disrupting the urea cycle. This achievement advances methods for modeling ...

Furthermore, the team succeeded in editing genes to replicate ornithine transcarbamylase deficiency, a genetic disorder disrupting the urea cycle. This achievement advances methods for modeling ...

Now three distinct programs have entered Phase 3 trials: UX111 for Sanfilippo syndrome type A, DTX401 for glycogen storage disease type Ia, and DTX301 for ornithine transcarbamylase deficiency.