Precision Biosciences Stock Price, News & Analysis

Precision BioSciences (NASDAQ: DTIL) announced the acceleration of PBGENE-DMD, their first-in-class in vivo gene editing therapy for Duchenne Muscular Dystrophy (DMD). The therapy targets mutations in the 'hot spot' region between exons 45-55, which affects up to 60% of DMD patients. The company plans to submit an IND/CTA in 2025, with clinical data expected in 2026.

Preclinical data presented at ASGCT demonstrated significant and durable functional improvement in muscle function over 9 months in a humanized DMD mouse model. PBGENE-DMD uses two ARCUS nucleases delivered via AAV to restore dystrophin protein production. The company's current cash runway is expected to support both PBGENE-DMD and PBGENE-HBV through Phase 1 clinical readouts.

To focus resources, Precision will pause development of PBGENE-3243 for mitochondrial disease until after PBGENE-DMD enters the clinic.

Precision BioSciences (NASDAQ: DTIL), a clinical stage gene editing company, announced it will release its Q1 2025 financial results and provide a business update on May 15, 2025. The company specializes in developing in vivo gene editing therapies using its proprietary ARCUS® platform, focusing on diseases with high unmet medical needs.

Precision BioSciences (NASDAQ: DTIL) presented initial safety data from its Phase 1 ELIMINATE-B trial for PBGENE-HBV, a first-in-class gene editing therapy for chronic hepatitis B, at the 2025 EASL Congress. The trial's first cohort included three male participants (mean age 41) who received two of three planned doses at 0.2 mg/kg.

Key findings show PBGENE-HBV was well-tolerated with no serious adverse events. All adverse events were mild (grade 1 or 2) and transient, with no cumulative effects after the second dose. The data supports continued repeat dosing and dose escalation plans. Notably, the FDA granted Fast Track designation to PBGENE-HBV in April 2025. The trial is approved in the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom.

iECURE announced it will present initial clinical data from its OTC-HOPE trial evaluating ECUR-506, an in vivo gene editing therapy for ornithine transcarbamylase (OTC) deficiency, at two major medical conferences. The presentations will take place at the ASGCT Annual Meeting (May 12-17, 2025) in New Orleans and the ESHG Conference (May 24-27, 2025) in Milan.

The presentations will expand on previously reported data from January 2025, which showed a complete clinical response in the first infant treated in the study. The company will showcase these results through a poster presentation and symposium at ASGCT, and an oral presentation at ESHG, led by Chief Medical Officer Gabriel Cohn and Dr. Julien Baruteau.

Initial safety data from ELIMINATE-B trial for chronic hepatitis B (PBGENE-HBV), their first-in-human study. Updates on ECUR-506, a partnered program with iECURE for OTC deficiency. Preclinical results from two muscle programs: PBGENE-3243 for mitochondrial disease and PBGENE-DMD for Duchenne Muscular Dystrophy.

The presentations demonstrate ARCUS platform's versatility in gene elimination, insertion, and excision across complex genetic diseases. The company aims to develop durable, curative treatments through various gene editing approaches.

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company, has announced new employee inducement awards approved by its Board's Compensation Committee on April 22, 2025. The awards were granted under the company's 2021 Employment Inducement Incentive Award Plan.

The inducement package includes 11,339 restricted stock units (RSUs) distributed among two new employees. These RSUs were granted under Nasdaq Listing Rule 5635(c)(4) as employment incentives. The vesting schedule for the RSUs is structured over a three-year period, with equal annual installments, contingent upon continued employment with Precision.

The company leverages its proprietary ARCUS® platform to develop in vivo gene editing therapies targeting diseases with high unmet medical needs.

Precision BioSciences (DTIL) has received Fast Track designation from the FDA for PBGENE-HBV, its lead in vivo gene editing therapy program targeting chronic hepatitis B. The therapy aims to cure the disease by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes.

The company is currently conducting the global Phase 1 ELIMINATE-B trial across multiple countries including the United States, Moldova, Hong Kong, New Zealand, and the United Kingdom. Initial results have shown encouraging safety and antiviral activity. Updates on the full low-dose cohort and higher dose levels are expected throughout 2025.

The Fast Track designation may enable more frequent FDA communications, rolling review of marketing applications, and potential Priority Review eligibility.

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company, has announced its participation in the 24th Annual Needham Virtual Healthcare Conference. The company, known for its proprietary ARCUS® platform which develops in vivo gene editing therapies including gene elimination, insertion, and excision programs, will present on April 7, 2025, at 3:00 PM ET in Track 2.

A live webcast of the presentation will be available through the company's website in the Investors section under Events & Presentations at investor.precisionbiosciences.com. The webcast recording will remain accessible for approximately 30 days after the event.

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.