Precision BioSciences shares soar on promising gene editing trial results

Investing.com -- Shares of Precision BioSciences, Inc. (NASDAQ:DTIL ) soared 60% following the announcement of a complete clinical response in the first infant dosed in a Phase 1/2 clinical trial. The trial, conducted by its partner iECURE, is aimed at treating Ornithine Transcarbamylase (OTC) deficiency, a genetic disorder.

The clinical trial results have demonstrated the potential of Precision BioSciences' ARCUS® gene editing platform, which was utilized to insert a functional copy of the OTC gene. The successful outcome in the first patient has increased confidence in the ARCUS platform's ability to perform sophisticated gene edits in vivo, including gene insertion, excision, and elimination.

The infant, treated with ECUR-506, experienced no significant clinical safety concerns, with the exception of asymptomatic transaminitis, which was managed and resolved with immunosuppressive therapy. Twelve weeks post-treatment, the patient was able to discontinue ammonia scavenger medication and increase protein intake to normal levels for their age, without elevating ammonia levels. The positive response has been maintained for six months, indicating a complete clinical response.

The OTC-HOPE study is set to continue enrollment across multiple countries with the aim of completing in 2025 and providing full program data by the first half of 2026. Meanwhile, Precision BioSciences is also advancing its ELIMINATE-B trial for PBGENE-HBV, targeting chronic hepatitis B, with Phase 1 data expected in 2025.

In light of these developments, BMO Capital analyst Kostas Biliouris upgraded Precision BioSciences from Market Perform to Outperform, setting a price target of $34.00. Biliouris noted the promising nature of the iECURE data, stating, "iECURE data appear very promising," and highlighted the early clinical validation for the ARCUS platform. The analyst estimates a roughly 65% probability for DTIL's in vivo HBV program to show acceptable safety and strong efficacy, which could drive the stock up by 100-200%, similar to recent gains seen by other companies in the sector. Given Precision BioSciences' valuation at approximately one-third of its net cash position, with a runway extending into the second half of 2026, the risk/reward profile is viewed favorably.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Source: Investing.com