After a landmark clinical trial revealed a full clinical response in the first infant dosage, Precision BioSciences, Inc. (NASDAQ: DTIL) shares are rocketing this morning. DTIL stock jumped 36.70% to $6.15 as of the most recent pre-market check, indicating that investors are becoming more confident in the company’s cutting-edge gene-editing capabilities.
Breakthrough in Neonatal Gene Therapy
Precision BioSciences (DTIL) revealed that its partner, iECURE, has reported encouraging clinical efficacy and safety data from the first patient dosed with ECUR-506 in the Phase 1/2 OTC-HOPE study. ECUR-506 is a pioneering in vivo gene insertion therapy targeting neonatal-onset Ornithine Transcarbamylase (OTC) deficiency.
This therapy employs a PCSK9-specific ARCUS nuclease, licensed from Precision BioSciences, to insert a functional copy of the OTC gene, offering hope for children born with this severe genetic disorder.
The trial results demonstrated that ECUR-506 achieved a complete clinical response in the first infant, marking a significant milestone in the field of in vivo gene editing. Precision BioSciences lauded iECURE for these groundbreaking results, highlighting their potential to transform treatment options for OTC deficiency and similar genetic diseases.
Safety and Tolerance Data Bolster Confidence
According to the report, the infant’s treatment with ECUR-506 was generally well tolerated, with no significant safety concerns aside from asymptomatic transaminitis observed at four weeks post-dosing.
This condition was effectively managed using immunosuppressive therapy, resolving within the same period. After 12 weeks, the infant’s reliance on ammonia-scavenging medications ceased, and daily protein intake increased to age-appropriate levels.
The mean ammonia levels remained stable within normal limits, representing a marked improvement from pre-treatment levels. The patient maintained these favorable outcomes over six months, further supporting the efficacy of ECUR-506.
OTC-HOPE Study Progress and Future Goals
The OTC-HOPE study, currently underway in the United Kingdom, the United States, Australia, and Spain, is expected to complete enrollment by 2025. iECURE anticipates presenting comprehensive program data by the first half of 2026, potentially paving the way for broader applications of DTIL’s ARCUS-based gene therapies in treating genetic disorders.