Precision BioSciences Releases New Data on Pbgene-DMD
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 14 2026
0mins
Precision BioSciences announced new preclinical data from its Pbgene-DMD program in an oral presentation at the American Society of Gene & Cell Therapy, ASGCT, 2026 Annual Meeting in Boston, Massachusetts. The new data show that treatment with Pbgene-DMD in early-juvenile mice resulted in significantly higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. This new data further supports evaluating Pbgene-DMD in younger DMD patient populations, including the 2- to 3-year-old patients, who are a key demographic of the ongoing Phase 1/2 Function-DMD trial evaluating Pbgene-DMD in boys ages 2 to 7. Key data: New preclinical data show Pbgene-DMD drove up to a 3x higher dystrophin protein restoration in skeletal muscle and up to 12x higher in respiratory muscle in early-juvenile mice compared to late-juvenile mice; Findings further support evaluation of Pbgene-DMD in DMD patient populations as young as 2 years of age; Greater efficacy with earlier intervention further differentiates Pbgene-DMD from microdystrophin approaches
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Analyst Views on DTIL
Wall Street analysts forecast DTIL stock price to rise
2 Analyst Rating
2 Buy
0 Hold
0 Sell
Moderate Buy
Current: 7.150
Low
30.00
Averages
45.00
High
60.00
Current: 7.150
Low
30.00
Averages
45.00
High
60.00
About DTIL
Precision BioSciences, Inc. is a clinical-stage gene editing company dedicated to improving life (DTIL) with its proprietary ARCUS genome editing platform. Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver cures for a range of genetic and infectious diseases. The ARCUS platform is used to develop in vivo gene editing therapies for gene edits, including gene insertion, elimination, and excision. The Company's in vivo gene editing pipeline programs include PBGENE-HBV, PBGENE-3243, PBGENE-DMD, PBGENE-LIVER, PBGENE-CNS, PBGENE-NVS and iECURE-OTC. PBGENE-HBV is its lead in vivo gene editing program designed to eliminate cccDNA, the key source of replicating hepatitis B virus (HBV) and inactivating integrated HBV DNA in hepatocytes. Its PBGENE-3243 program is a first of its kind potential treatment for m.3243 associated mitochondrial disease. The Company's iECURE-OTC program is developed in partnership with iECURE, Inc.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Precision BioSciences to Host Webcast on Hepatitis B Clinical Data
- Webcast Announcement: Precision BioSciences will host an investor webcast on May 27, 2026, at 8:00 AM EDT to discuss new biopsy and clinical data from the ongoing ELIMINATE-B trial, which is expected to attract attention from investors and Hepatitis B experts, thereby enhancing the company's visibility in the biopharmaceutical sector.
- ELIMINATE-B Trial Progress: The trial is evaluating PBGENE-HBV as a potential curative treatment for chronic Hepatitis B and has received Fast Track designation from the FDA, indicating its innovative nature and market potential, which could present significant commercial opportunities for the company.
- Clinical Data Release: The webcast will showcase the latest clinical data and important biopsy results presented at the 2026 European Association for the Study of the Liver Congress, which will support the efficacy of PBGENE-HBV and further bolster investor confidence.
- Platform Advantages: PBGENE-HBV is the world's first gene editing program targeting the root cause of chronic Hepatitis B, cccDNA, aiming for a viral cure; if successful, it will establish Precision BioSciences as a leader in gene therapy, driving long-term growth.
See More
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- Crisis Rate Reduction: Following ECUR-506 treatment, the annualized rate of HACs decreased by approximately 52%, from about 3.12 to 1.49 events per year, providing better survival and developmental opportunities for patients and underscoring the clinical significance of the new therapy.
- Good Safety Profile: ECUR-506 was generally well tolerated across all dose cohorts, with no unexpected safety events observed, aligning with expected safety characteristics, suggesting optimistic prospects for its application in high-risk populations.
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See More
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- Revenue Growth: The company achieved Q1 revenue of $10.84M, a significant increase from $29K year-over-year, beating market expectations by $6.79M, demonstrating substantial progress in the commercialization of its gene editing technologies.
- Cash Reserves: As of March 31, 2026, Precision BioSciences holds a cash balance of $125.8M, including cash, cash equivalents, and restricted cash, providing financial stability for future clinical stage gene editing projects.
- Project Milestones: The company anticipates utilizing its cash reserves to support data milestones for its two wholly owned clinical stage gene editing programs, PBGENE-HBV and PBGENE-DMD, through 2028, reflecting the sustainability of its long-term strategic vision.
See More
Precision BioSciences to Announce Q1 Earnings on May 5
- Earnings Announcement Schedule: Precision BioSciences is set to release its Q1 2023 earnings report on May 5 before market open, with a consensus EPS estimate of -$0.81, reflecting a 63.3% year-over-year improvement, indicating efforts towards enhancing profitability.
- Revenue Expectations: Analysts forecast revenue of $4.05 million for the quarter, although the company has only beaten revenue estimates 25% of the time over the past year, suggesting ongoing challenges in revenue growth.
- Historical Performance Review: Over the past year, Precision BioSciences has exceeded EPS estimates 75% of the time, demonstrating relative stability in earnings forecasts, which may bolster investor confidence ahead of the upcoming report.
- Market Reaction Anticipation: As the earnings report approaches, investors will closely monitor the company's performance, particularly in the current economic climate, where any results exceeding expectations could positively impact the stock price.
See More
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- Trial Design and Objectives: The FUNCTION-DMD study aims to enroll ambulatory patients aged 2-7 with mutations between exons 45 and 55, representing approximately 60% of boys with DMD, to evaluate safety, tolerability, and efficacy, potentially providing durable muscle function improvements.
- Multidisciplinary Care Center: Arkansas Children's Hospital, recognized as a PPMD-certified Duchenne Care Center, delivers specialized multidisciplinary care, ensuring high standards in clinical services while providing comprehensive support for patients participating in the FUNCTION-DMD study.
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See More
Precision BioSciences Grants RSUs to New Employee
- Employee Incentive Plan: On April 20, 2026, Precision BioSciences approved the grant of 7,094 restricted stock units (RSUs) to a new employee as an inducement award, aimed at attracting high-quality talent to the company.
- Grant Conditions: Under Nasdaq Listing Rule 5635(c)(4), the RSUs will vest in substantially equal annual installments on each of the first three anniversaries of the employee's start date, ensuring continued service and contribution to the company.
- ARCUS Platform Advantages: Precision BioSciences' ARCUS gene editing platform, with its unique cutting mechanism and smaller structure, is dedicated to developing gene editing therapies for high unmet need diseases, showcasing significant market potential.
- Diverse Treatment Approaches: The platform supports various gene editing strategies, including gene elimination, defective gene excision, and gene insertion, aimed at providing lasting cures for a broad range of genetic and infectious diseases, further solidifying the company's position in the industry.
See More
Precision BioSciences to Host Webcast on Hepatitis B Clinical Data
- Webcast Announcement: Precision BioSciences will host an investor webcast on May 27, 2026, at 8:00 AM EDT to discuss new biopsy and clinical data from the ongoing ELIMINATE-B trial, which is expected to attract attention from investors and Hepatitis B experts, thereby enhancing the company's visibility in the biopharmaceutical sector.
- ELIMINATE-B Trial Progress: The trial is evaluating PBGENE-HBV as a potential curative treatment for chronic Hepatitis B and has received Fast Track designation from the FDA, indicating its innovative nature and market potential, which could present significant commercial opportunities for the company.
- Clinical Data Release: The webcast will showcase the latest clinical data and important biopsy results presented at the 2026 European Association for the Study of the Liver Congress, which will support the efficacy of PBGENE-HBV and further bolster investor confidence.
- Platform Advantages: PBGENE-HBV is the world's first gene editing program targeting the root cause of chronic Hepatitis B, cccDNA, aiming for a viral cure; if successful, it will establish Precision BioSciences as a leader in gene therapy, driving long-term growth.
See More
Preliminary Data on ECUR-506 Treatment for OTC Deficiency
- Significant Treatment Effects: As of April 20, 2026, 71% of the 7 participants treated with ECUR-506 experienced no hyperammonemic crises (HACs), indicating the therapy's potential to improve quality of life, particularly in neonates with OTC deficiency.
- Crisis Rate Reduction: Following ECUR-506 treatment, the annualized rate of HACs decreased by approximately 52%, from about 3.12 to 1.49 events per year, providing better survival and developmental opportunities for patients and underscoring the clinical significance of the new therapy.
- Good Safety Profile: ECUR-506 was generally well tolerated across all dose cohorts, with no unexpected safety events observed, aligning with expected safety characteristics, suggesting optimistic prospects for its application in high-risk populations.
- Clinical Trial Progress: Preliminary results from the OTC-HOPE trial support continued evaluation of ECUR-506's efficacy, with iECURE planning to further validate its effectiveness in upcoming pivotal trials, offering hope for infants affected by OTC deficiency.
See More
Precision BioSciences Q1 Earnings Report Analysis
- Earnings Performance: Precision BioSciences reported a Q1 GAAP EPS of -$0.75, missing expectations by $0.09, indicating challenges in profitability that may affect investor confidence.
- Revenue Growth: The company achieved Q1 revenue of $10.84M, a significant increase from $29K year-over-year, beating market expectations by $6.79M, demonstrating substantial progress in the commercialization of its gene editing technologies.
- Cash Reserves: As of March 31, 2026, Precision BioSciences holds a cash balance of $125.8M, including cash, cash equivalents, and restricted cash, providing financial stability for future clinical stage gene editing projects.
- Project Milestones: The company anticipates utilizing its cash reserves to support data milestones for its two wholly owned clinical stage gene editing programs, PBGENE-HBV and PBGENE-DMD, through 2028, reflecting the sustainability of its long-term strategic vision.
See More
Precision BioSciences to Announce Q1 Earnings on May 5
- Earnings Announcement Schedule: Precision BioSciences is set to release its Q1 2023 earnings report on May 5 before market open, with a consensus EPS estimate of -$0.81, reflecting a 63.3% year-over-year improvement, indicating efforts towards enhancing profitability.
- Revenue Expectations: Analysts forecast revenue of $4.05 million for the quarter, although the company has only beaten revenue estimates 25% of the time over the past year, suggesting ongoing challenges in revenue growth.
- Historical Performance Review: Over the past year, Precision BioSciences has exceeded EPS estimates 75% of the time, demonstrating relative stability in earnings forecasts, which may bolster investor confidence ahead of the upcoming report.
- Market Reaction Anticipation: As the earnings report approaches, investors will closely monitor the company's performance, particularly in the current economic climate, where any results exceeding expectations could positively impact the stock price.
See More
Precision BioSciences Launches DMD Clinical Trial at Arkansas Children's Hospital
- Clinical Trial Launch: Precision BioSciences has activated the FUNCTION-DMD clinical trial at Arkansas Children's Hospital, now enrolling patients, marking a significant advancement in the treatment of Duchenne muscular dystrophy (DMD) and potentially offering new therapeutic options for patients.
- Trial Design and Objectives: The FUNCTION-DMD study aims to enroll ambulatory patients aged 2-7 with mutations between exons 45 and 55, representing approximately 60% of boys with DMD, to evaluate safety, tolerability, and efficacy, potentially providing durable muscle function improvements.
- Multidisciplinary Care Center: Arkansas Children's Hospital, recognized as a PPMD-certified Duchenne Care Center, delivers specialized multidisciplinary care, ensuring high standards in clinical services while providing comprehensive support for patients participating in the FUNCTION-DMD study.
- FDA Special Designations: PBGENE-DMD has received Orphan Drug Designation and Fast Track designation from the FDA, highlighting its potential in treating DMD and likely leading to significant functional improvements for patients, thereby enhancing Precision BioSciences' leadership in the gene editing sector.
See More
Precision BioSciences Grants RSUs to New Employee
- Employee Incentive Plan: On April 20, 2026, Precision BioSciences approved the grant of 7,094 restricted stock units (RSUs) to a new employee as an inducement award, aimed at attracting high-quality talent to the company.
- Grant Conditions: Under Nasdaq Listing Rule 5635(c)(4), the RSUs will vest in substantially equal annual installments on each of the first three anniversaries of the employee's start date, ensuring continued service and contribution to the company.
- ARCUS Platform Advantages: Precision BioSciences' ARCUS gene editing platform, with its unique cutting mechanism and smaller structure, is dedicated to developing gene editing therapies for high unmet need diseases, showcasing significant market potential.
- Diverse Treatment Approaches: The platform supports various gene editing strategies, including gene elimination, defective gene excision, and gene insertion, aimed at providing lasting cures for a broad range of genetic and infectious diseases, further solidifying the company's position in the industry.
See More
