Precision Biosciences Stock Price, News & Analysis

Precision BioSciences (NASDAQ: DTIL) reported significant clinical progress in Q4 and FY2024, highlighting two successful in vivo gene editing programs. The company's lead program PBGENE-HBV, targeting Hepatitis B, demonstrated safety and substantial antiviral activity in initial trials. The first patient cohort showed HBsAg reduction in two of three patients at the lowest dose level.

Their partner iECURE reported a complete clinical response in the first infant treated for Ornithine Transcarbamylase Deficiency. The company received IND and CTA approvals for ELIMINATE-B trials across multiple countries, including the US.

Financial highlights include $108.5M in cash and equivalents as of December 31, 2024, with runway extended into H2 2026. Q4 revenues were $0.6M compared to $7.0M in Q4 2023. Net loss increased to $17.8M from $13.4M year-over-year.

iECURE presented encouraging data from its OTC-HOPE Phase 1/2 clinical trial of ECUR-506 for ornithine transcarbamylase (OTC) deficiency at the 2025 ACMG Annual Clinical Genetics Meeting. The first infant patient, dosed at 6.5 months old with 1.3 x 1013 GC/kg of ECUR-506, showed promising results after six months of treatment.

Key findings include:

• Complete clinical response achieved per study protocol
• Successful discontinuation of ammonia scavenger medication at 12 weeks post-treatment
• Increased protein allowance to age-appropriate levels
• Higher blood urea nitrogen (BUN) levels post-treatment, suggesting improved OTC enzyme function
• Normal plasma ammonia levels maintained after medication removal

While treatment was generally well-tolerated, a temporary Grade 3 transaminitis was observed at four weeks post-exposure, which resolved by week eight with immunosuppressive therapy.

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company, has announced it will release its fourth quarter and fiscal year 2024 financial results on March 26, 2025. The company, which develops in vivo gene editing therapies using its proprietary ARCUS® platform, will also provide a business update. Their platform technology focuses on gene elimination, gene insertion, and gene excision programs.

Precision BioSciences (NASDAQ: DTIL) presented preclinical data for PBGENE-HBV, the first clinical-stage gene editing therapy for chronic hepatitis B, at the Global Hepatitis Summit 2025. The data supports repeat dosing in the ongoing ELIMINATE-B Phase 1 trial.

Key findings show that in non-human primates, repeated PBGENE-HBV administration was safe and well-tolerated, with no systemic accumulation. Previous data demonstrated up to 99% viral eradication with two doses. Initial results from cohort 1 showed the first 0.2 mg/kg dose was safe, with substantial HBsAg reduction in two of three participants.

The ELIMINATE-B study is actively enrolling HBeAg-negative chronic Hepatitis B patients in Moldova, Hong Kong, and New Zealand, with planned expansion to U.S. sites. The trial will evaluate up to three dose administrations at each dose level, with the company planning to share detailed clinical data throughout 2025.

Precision BioSciences (DTIL) presented preclinical data for PBGENE-DMD, a novel gene editing therapy for Duchenne muscular dystrophy (DMD) at the 2025 MDA Conference. The therapy uses ARCUS® platform to excise exons 45-55 of the dystrophin gene, potentially benefiting up to 60% of DMD patients.

Key findings from the preclinical study in humanized DMD mouse models showed:

• Restoration of functional dystrophin protein across multiple muscles including heart, diaphragm, and skeletal muscles
• 66% improvement in muscle resilience against eccentric injury
• Maximum force output reaching 93% of healthy control mice, with improvements observed between 3-6 months
• Evidence of dystrophin gene correction in muscle satellite stem cells, suggesting potential long-term therapeutic benefits

Precision BioSciences (NASDAQ: DTIL) has received FDA clearance for its Investigational New Drug (IND) application for PBGENE-HBV, marking a significant milestone as the first-ever in vivo gene editing therapy cleared for clinical trials in treating chronic hepatitis B in the United States.

The company will expand its Phase 1 ELIMINATE-B study to the U.S., specifically at the Liver Center at Massachusetts General Hospital, adding to existing trial sites in Moldova, Hong Kong, and New Zealand. The trial's first cohort is currently receiving doses at 0.2 mg/kg, with plans to escalate to a higher dose level in Cohort 2.

PBGENE-HBV, developed using Precision's proprietary ARCUS® platform, aims to cure chronic hepatitis B by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes. The company plans to expand the study to the UK and will share detailed clinical data throughout 2025. The development addresses a significant medical need, as up to 2.4 million people in the U.S. live with chronic hepatitis B, with numbers remaining largely unchanged over the past 15 years despite existing treatments.

iECURE announced it will present initial data from its Phase 1/2 OTC-HOPE clinical trial for ECUR-506, an in vivo gene editing treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency, at the 2025 ACMG Annual Clinical Genetics Meeting in Los Angeles.

The presentation will expand on previously reported data from January 2025, which showed that the first infant treated in the trial achieved a complete clinical response according to study protocol. Dr. Julien Baruteau from Great Ormond Street Hospital for Children, London, will present the findings on March 21, 2025.

Precision BioSciences (NASDAQ: DTIL) has announced an upcoming oral presentation of preclinical data from their PBGENE-DMD program at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference. The presentation, scheduled for March 19, 2025, at 8:00 AM CT, will focus on the treatment of Duchenne muscular dystrophy (DMD) using their proprietary ARCUS® platform.

The presentation will detail how ARCUS-mediated excision of exons 45-55 leads to functional Del45-55 dystrophin and restoration of skeletal muscle function in DMD treatment. The company's ARCUS platform is being developed for various in vivo gene editing therapies, including gene elimination, insertion, and excision programs.

Precision BioSciences (NASDAQ: DTIL) announced initial results from the ELIMINATE-B Phase 1 trial of PBGENE-HBV, their novel gene editing therapy for chronic Hepatitis B. The first cohort of three patients received the lowest dose level (0.2 mg/kg) of PBGENE-HBV, which uses ARCUS-encoding mRNA in lipid nanoparticles.

Key findings include:

• The treatment was safe and well-tolerated with no Grade ≥2 treatment-related adverse events
• Two of three participants showed substantial reduction in Hepatitis B surface antigen (HBsAg)
• The trial is designed for three dose administrations per dose level

The study is currently enrolling HBeAg-negative chronic Hepatitis B patients in Moldova, Hong Kong, and New Zealand, with plans to expand to the U.S. and U.K. Precision plans to share detailed clinical data throughout 2025.