Precision BioSciences, Inc. (DTIL), a clinical-stage gene editing company, on Thursday, announced positive clinical efficacy data from its partner iECURE in the Phase 1/2 OTC-HOPE study for ECUR-506, an in vivo gene insertion therapy designed to treat Ornithine Transcarbamylase or OTC deficiency in neonates.
The first infant dosed with ECUR-506 showed a complete clinical response, with significant improvements in metabolic function maintained through six months post-treatment.
In the study, ECUR-506 utilizes Precision's ARCUS platform for gene editing, inserting a functional copy of the OTC gene to correct the genetic defect that causes ammonia buildup in the blood—a potentially life-threatening condition.
Following a single dose of ECUR-506, the infant showed a reduction in ammonia levels, and ammonia scavenger medication was discontinued.
Additionally, the infant's daily protein intake was increased to age-appropriate levels, which was well tolerated.
The therapy was generally well tolerated, with asymptomatic transaminitis noted at four weeks, which was successfully managed with immunosuppressive therapy.
No other significant safety concerns were reported.
The trial, which is ongoing in the United States, the United Kingdom, Australia, and Spain, expects to complete enrollment by 2025, with full data available in 2026.
Michael Amoroso, CEO of Precision BioSciences, expressed about ECUR-506 and the ARCUS platform, highlighting their transformative potential for OTC deficiency, a condition with no long-term treatment options except liver transplant.
The company is also advancing its gene editing pipeline, including PBGENE-HBV for chronic hepatitis B.
DTIL closed Wednesday's (Jan. 08, 2025) trading at $4.50, down 6.05 %. In after-hours trading, the stock was up 4.44% at $4.70.
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