Precision BioSciences, Inc.

https://precisionbiosciences.com

Precision BioSciences, Inc. is a clinical-stage gene editing company headquartered in Durham, North Carolina, United States. Founded in 2006, the company is dedicated to improving life through its novel and proprietary ARCUS® genome editing platform, focusing on developing therapies for high unmet medical needs, including genetic and infectious diseases.

The company's core technology is the ARCUS genome editing platform, which utilizes sequence-specific DNA-cutting enzymes (nucleases) to precisely insert, remove, or repair DNA within living cells and tissues. Precision BioSciences is developing in vivo gene editing therapies for various conditions, including genetic disorders like Duchenne muscular dystrophy (DMD) and infectious diseases such as Hepatitis B (HBV). Key programs in their pipeline, PBGENE-HBV and PBGENE-DMD, are currently in clinical trials.

Michael Amoroso serves as the President and CEO, having been appointed in October 2021. As of late April 2026, Precision BioSciences is actively advancing its pipeline, with recent announcements including the activation of the first clinical trial site and patient enrollment for its Phase 1/2 FUNCTION-DMD study for Duchenne Muscular Dystrophy. The company is also expanding its ELIMINATE-B trial for chronic Hepatitis B in Europe and presented late-breaking data for PBGENE-HBV at the European Association for the Study of the Liver (EASL) Congress 2026.

Latest updates

Precision BioSciences Initiates DMD Gene Editing Trial, Securing Early Momentum

Event summary

  • Precision BioSciences has activated the first clinical trial site, Arkansas Children’s Hospital, for its Phase 1/2 FUNCTION-DMD study.
  • The trial will enroll ambulatory DMD patients aged 2-7 with mutations between exons 45 and 55, representing approximately 60% of DMD cases.
  • PBGENE-DMD utilizes Precision's ARCUS® platform to excise exons 45-55, aiming to restore a functional dystrophin protein.
  • The program received Orphan Drug Designation in July 2025 and is eligible for a Priority Review Voucher (PRV) and Fast Track designation.

The big picture

Precision BioSciences' entry into the Duchenne muscular dystrophy gene editing space represents a significant development, as current treatment options offer limited functional benefit. The company's ARCUS platform aims to address the underlying genetic cause of DMD, potentially offering a more durable therapeutic effect. Securing the Priority Review Voucher (PRV) could expedite regulatory approval and significantly increase the program's value, but clinical success remains the primary driver.

What we're watching

Enrollment Pace
The speed of patient enrollment across additional trial sites will be a key indicator of trial efficiency and potential for timely data readouts, given the unmet need in DMD.
Efficacy Signals
Early data on dystrophin protein expression and functional outcomes will be critical to assess the therapeutic potential of PBGENE-DMD and its differentiated approach compared to microdystrophin therapies.
PRV Utilization
The potential to secure and utilize the Priority Review Voucher (PRV) will significantly impact the commercialization timeline and value proposition of PBGENE-DMD, contingent on positive clinical data.

Precision BioSciences Data Bolsters DMD Gene Editing Approach

Event summary

  • Precision BioSciences' ARCUS platform preclinical data for PBGENE-DMD will be presented orally at the ASGCT 2026 Annual Meeting on May 14th.
  • The data demonstrate efficacy in early-juvenile mice, suggesting potential benefit from earlier intervention in DMD patients.
  • PBGENE-DMD aims to treat ~60% of DMD patients by excising exons 45-55, restoring a near full-length dystrophin protein.
  • The program has received Orphan Drug Designation, Fast Track designation, and is eligible for a Priority Review Voucher (PRV).

The big picture

Precision BioSciences' PBGENE-DMD program represents a significant effort to address the unmet need in Duchenne Muscular Dystrophy, a market with limited treatment options and a substantial patient population. The company's ARCUS platform, combined with the potential for a PRV, positions it favorably, but the clinical trial results will be the ultimate determinant of its success. The broader gene editing space is attracting significant investment, and Precision's ability to demonstrate a durable and safe therapeutic effect will be crucial for maintaining investor confidence.

What we're watching

Clinical Translation
The success of preclinical data in mice does not guarantee similar results in human clinical trials, and the Phase 1/2 FUNCTION-DMD trial will be critical in assessing PBGENE-DMD's true potential.
Regulatory Pathway
The eligibility for a Priority Review Voucher (PRV) could significantly accelerate approval timelines, but hinges on successful clinical outcomes and FDA acceptance.
Competitive Landscape
While Precision BioSciences' approach aims for a more complete dystrophin restoration, the company faces competition from other gene editing and microdystrophin therapies, and the relative efficacy and safety will be key differentiators.

Precision BioSciences Data Suggests Potential HBV Cure in Early Trial

Event summary

  • Precision BioSciences will present Phase 1 ELIMINATE-B trial data at EASL Congress 2026 (Barcelona, May 27-30).
  • The presentation (LB26-51656, LBP-043) focuses on biopsy data showing elimination and inactivation of cccDNA in Hepatitis B patients.
  • PBGENE-HBV is designed to eliminate cccDNA, the root cause of chronic Hepatitis B, and inactivate integrated HBV DNA.
  • The FDA has granted PBGENE-HBV Fast Track designation and indicated sustained loss of HBV DNA as an approvable endpoint.

The big picture

Precision BioSciences' PBGENE-HBV represents a novel approach to treating chronic Hepatitis B, a global health challenge affecting hundreds of millions. While existing therapies manage the disease, a curative treatment remains elusive. The company's ARCUS platform, and specifically PBGENE-HBV's focus on cccDNA elimination, positions it uniquely within the gene editing space, but success hinges on demonstrating robust efficacy and navigating the complexities of regulatory approval for a gene editing therapy.

What we're watching

Clinical Efficacy
The presentation's data will be scrutinized for the degree and durability of cccDNA elimination, as this is the core value proposition of PBGENE-HBV and a key indicator of potential curative impact.
Regulatory Pathway
The FDA’s stated endpoint of sustained HBV DNA loss will likely influence the trial design and regulatory strategy, and future data releases will be assessed against this benchmark.
Competitive Landscape
Given PBGENE-HBV's claim as the only gene editing program targeting cccDNA elimination, the emergence of competing approaches will be a key factor in its long-term commercial viability.

Precision BioSciences Expands HBV Trial, Bolstering cccDNA Elimination Strategy

Event summary

  • Precision BioSciences received Clinical Trial Application (CTA) approval to expand the ELIMINATE-B trial for PBGENE-HBV in France and Romania.
  • The expansion adds to existing trial sites in the UK, Moldova, New Zealand, Hong Kong, and the US.
  • Patient screening in France and Romania is expected to begin in Q2 2026.
  • The trial investigates PBGENE-HBV, a gene editing therapy targeting cccDNA, the root cause of chronic hepatitis B.
  • PBGENE-HBV has received Fast Track designation from the FDA.

The big picture

Precision BioSciences’ expansion of the ELIMINATE-B trial represents a significant step in the race to develop a curative therapy for chronic hepatitis B, a disease affecting hundreds of millions globally. The company’s focus on eliminating cccDNA, rather than merely suppressing viral load, differentiates it from existing treatments and addresses a key unmet medical need. However, the success of this strategy remains contingent on demonstrating clinical efficacy and securing regulatory approval in a competitive landscape.

What we're watching

Execution Risk
The success of the trial expansion hinges on rapid site initiation and patient enrollment in France and Romania, which could be impacted by logistical challenges or investigator capacity.
Regulatory Dynamics
The FDA's continued acceptance of sustained loss of HBV DNA as an approvable endpoint will be crucial for PBGENE-HBV's potential market approval, and any shift in guidance could significantly alter the development pathway.
Competitive Landscape
The emergence of alternative therapies targeting hepatitis B, or advancements in existing treatments, could erode PBGENE-HBV’s competitive advantage and impact its commercial viability.

Precision BioSciences to Present at Needham Healthcare Conference

Event summary

  • Precision BioSciences (DTIL) will participate in the 25th Annual Needham Virtual Healthcare Conference.
  • The conference runs April 13-16, 2026, with Precision BioSciences’ presentation scheduled for Tuesday, April 14, 2026, at 9:30 AM ET.
  • A replay of the presentation will be available on the company’s website following the event.
  • Precision BioSciences is utilizing its ARCUS® platform for in vivo gene editing therapies.

The big picture

Precision BioSciences, a clinical-stage gene editing company, is seeking to gain visibility and investor confidence amidst a crowded and rapidly evolving gene editing sector. The Needham Healthcare Conference provides a platform to showcase the ARCUS platform and its in vivo gene editing therapies, which target high unmet needs. Success will depend on demonstrating clinical efficacy and differentiation from competitors like CRISPR Therapeutics and Intellia Therapeutics.

What we're watching

Investor Relations
The conference participation signals an ongoing effort to engage with investors and analysts, potentially driven by the need to maintain or increase valuation in a competitive gene editing landscape.
Clinical Progress
The content of the presentation will be scrutinized for updates on the PBGENE-HBV and PBGENE-DMD programs, as progress in these clinical trials is critical to the company’s near-term outlook.
Platform Adoption
The success of Precision BioSciences hinges on broader adoption of the ARCUS platform; the conference provides an opportunity to articulate the platform’s advantages and address any lingering technical or commercial concerns.

Precision BioSciences Posts Strong 2025, Advances Gene Editing Pipeline

Event summary

  • Precision BioSciences reported $34.2 million in revenue for Q4 2025, up from $0.6 million in the same period of 2024, driven by revenue recognition from Novartis and Imugene.
  • The company extended its cash runway through 2028, supported by a $75 million financing round in November 2025.
  • Clinical data for PBGENE-HBV, the company’s Hepatitis B program, showed dose-dependent antiviral activity and molecular evidence of viral DNA gene editing.
  • Precision BioSciences received IND clearance for PBGENE-DMD (Duchenne muscular dystrophy) in February 2026, paving the way for clinical trial initiation.

The big picture

Precision BioSciences' 2025 results highlight the increasing momentum behind in vivo gene editing, a field attracting significant investment and showing early clinical promise. While the company's revenue is still relatively small, the progress on PBGENE-HBV and PBGENE-DMD positions it as a key player in the race to develop curative therapies for genetic diseases. The extended cash runway provides breathing room, but continued clinical success and strategic partnerships will be essential for long-term value creation.

What we're watching

Clinical Efficacy
The ongoing ELIMINATE-B trial will be critical to determine if PBGENE-HBV can achieve a functional cure for Hepatitis B, and whether the observed molecular evidence translates to sustained viral suppression.
Regulatory Pathway
The FDA's feedback on the PBGENE-DMD program and the potential for a Priority Review Voucher will significantly impact the program's timeline and commercial prospects.
Partner Synergies
The continued collaboration with Novartis, beyond hemoglobinopathies, will reveal the breadth of Precision’s ARCUS platform and potential for future revenue streams.

Precision BioSciences Bolsters HBV Program IP with Key Patent Allowances

Event summary

  • Precision BioSciences received two Notices of Allowance from the USPTO for patent applications related to its PBGENE-HBV program.
  • Application No. 19/347,136 covers the ARCUS nuclease used in PBGENE-HBV, providing composition-of-matter protection.
  • Application No. 19/273,982 covers a novel polypeptide linker, potentially applicable to future ARCUS nucleases.
  • The granted patents are expected to expire in November 2044.
  • PBGENE-HBV is currently in a Phase 1/2 clinical trial targeting Hepatitis B cccDNA elimination.

The big picture

The patent allowances strengthen Precision BioSciences’ intellectual property position in a competitive gene editing landscape, particularly as in vivo gene editing for chronic viral infections gains traction. While the PBGENE-HBV program represents a significant opportunity, the company's success hinges on demonstrating clinical efficacy and defending its IP. The broader gene editing market is attracting substantial investment, and robust IP protection is essential for securing long-term value.

What we're watching

Clinical Efficacy
The clinical trial data for PBGENE-HBV will be critical in determining the therapeutic potential of the ARCUS platform for in vivo gene editing and the value of the newly protected IP.
Patent Defense
The strength and enforceability of these patents against potential challenges from competitors will influence Precision BioSciences’ long-term market position.
Platform Expansion
How quickly Precision BioSciences can leverage the novel linker technology to develop and patent new ARCUS nucleases for other therapeutic targets will indicate the platform’s versatility.

Precision BioSciences DMD Data Bolsters Gene Editing Approach Ahead of Trial

Event summary

  • Precision BioSciences presented preclinical data for PBGENE-DMD at the Muscular Dystrophy Association Clinical & Scientific Conference 2026.
  • The data, from a humanized DMD mouse model, showed improvements in muscle damage markers, including a ~50-65% reduction in CK levels at 90 days post-treatment.
  • PBGENE-DMD aims to treat up to 60% of DMD patients with mutations in exons 45-55 by restoring dystrophin production.
  • The Phase 1/2 FUNCTION-DMD clinical trial is expected to enroll patients aged 2-7 and begin in the first half of 2026.

The big picture

Precision BioSciences' PBGENE-DMD represents a differentiated approach to treating Duchenne muscular dystrophy, moving beyond microdystrophin and exon skipping therapies. The company’s reliance on its ARCUS platform positions it within a rapidly evolving gene editing landscape, where durability and targeted delivery remain critical challenges. Success with PBGENE-DMD could validate Precision BioSciences’ in vivo gene editing strategy and potentially open doors for similar therapies targeting other genetic diseases.

What we're watching

Clinical Translation
The success of the FUNCTION-DMD trial will hinge on whether preclinical improvements translate to meaningful patient outcomes, particularly given the complexity of DMD.
Satellite Cell Editing
The long-term durability of PBGENE-DMD’s effect will depend on the continued activity of edited muscle satellite cells, which requires ongoing monitoring.
Regulatory Pathway
Given the novelty of the gene editing approach, the FDA’s ongoing assessment of safety and efficacy will be a key determinant of future development and commercialization.

Precision BioSciences Gains FDA Fast Track for DMD Gene Edit

Event summary

  • Precision BioSciences received FDA Fast Track designation for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy (DMD).
  • The company plans to initiate a Phase 1/2 FUNCTION-DMD clinical study, enrolling patients aged 2-7 with specific DMD mutations.
  • A virtual KOL event is scheduled for March 17, 2026, featuring Dr. Aravindhan Veerapandiyan and Pat Furlong.
  • PBGENE-DMD targets exons 45-55, covering approximately 60% of DMD patients, using a gene excision approach.
  • Preclinical data indicates PBGENE-DMD restores dystrophin production across multiple muscle tissues, including cardiac tissue.

The big picture

The Fast Track designation for PBGENE-DMD underscores the growing interest in gene editing as a potential treatment for rare genetic diseases like DMD, a market with significant unmet need. While the DMD therapeutic landscape is crowded, Precision BioSciences' differentiated approach – a gene excision strategy – positions them to potentially capture a portion of this market, but success hinges on demonstrating superior efficacy and safety compared to existing and emerging therapies. The KOL event and subsequent clinical trial data will be key indicators of the program’s viability.

What we're watching

Clinical Efficacy
The FUNCTION-DMD trial's results will be critical in determining whether PBGENE-DMD can deliver on its promise of durable functional muscle improvement, and whether the observed preclinical benefits translate to meaningful patient outcomes.
Regulatory Pathway
The Fast Track designation expedites the process, but the FDA’s ultimate approval will hinge on the safety and efficacy data generated in the FUNCTION-DMD trial, and any potential novel regulatory hurdles for in vivo gene editing therapies.
Competitive Landscape
Given the unmet need in DMD, Precision BioSciences faces competition from other gene therapy and microdystrophin approaches; the company's differentiation through gene excision will need to be clearly demonstrated to secure market share.

Precision BioSciences Collects $7.5M Milestone Payment for MS Gene Therapy

Event summary

  • Precision BioSciences received $7.5 million from TG Therapeutics related to the clinical progress of azer-cel, a gene therapy for multiple sclerosis.
  • The payment includes $5.25 million in cash and $2.25 million in Precision BioSciences common stock.
  • The milestone was triggered by progress in a Phase 1 clinical trial of azer-cel for progressive forms of multiple sclerosis.
  • Precision BioSciences anticipates the proceeds, combined with existing resources, will support operations through 2028.
  • The license agreement grants TG Therapeutics exclusive worldwide rights to develop and commercialize azer-cel for autoimmune diseases.

The big picture

This milestone payment provides Precision BioSciences with a near-term financial boost, but the company's long-term success hinges on the clinical validation of azer-cel and the broader adoption of its ARCUS platform. The deal highlights the growing trend of gene editing companies leveraging partnerships to accelerate development and share risk, particularly in areas like autoimmune disease where traditional therapies often fall short.

What we're watching

Clinical Efficacy
The Phase 1 trial data for azer-cel will be critical in determining the therapy’s potential and influencing future development decisions, and will likely dictate the timing and size of subsequent milestone payments.
Financial Stability
Precision BioSciences’ ability to extend its cash runway through 2028 will depend on continued milestone payments from TG Therapeutics and potentially other financing activities.
Partnering Strategy
The success of the TG Therapeutics partnership will shape Precision BioSciences’ approach to licensing its ARCUS platform and seeking external funding for its in vivo gene editing pipeline.

Precision BioSciences Presents Data on DMD Gene Editing Therapy

Event summary

  • Precision BioSciences' PBGENE-DMD preclinical data, focused on safety and long-term efficacy, will be presented as a poster at the 2026 MDA Clinical & Scientific Conference.
  • The study, abstract number 142M, demonstrates functional improvement in a humanized DMD mouse model using Precision BioSciences’ ARCUS® platform.
  • PBGENE-DMD targets exons 45-55, impacting approximately 60% of boys with Duchenne muscular dystrophy (DMD).
  • The therapy has received Investigational New Drug (IND) clearance from the FDA and holds Rare Pediatric Disease (RPD) and Orphan Drug (ODD) designations.

The big picture

Precision BioSciences' PBGENE-DMD represents a novel gene editing approach for Duchenne muscular dystrophy, a devastating genetic disorder with limited treatment options. The company's ARCUS® platform aims to provide a potentially curative therapy, but the success hinges on translating preclinical findings into robust clinical outcomes and differentiating itself from existing, albeit less effective, therapies. The FDA’s IND clearance and RPD/ODD designations are positive indicators, but the clinical trial data will be the ultimate arbiter of its value.

What we're watching

Clinical Translation
The efficacy observed in the mouse model may not translate directly to human clinical trials, and the observed improvements must be replicated in human subjects to validate the therapy's potential.
Durability
The long-term durability of the gene editing effect, particularly the editing of muscle satellite cells, will be critical for sustained functional improvement in DMD patients and requires careful monitoring in clinical trials.
Competitive Landscape
Given existing microdystrophin and exon skipping treatments, PBGENE-DMD’s differentiated approach will need to demonstrate a clear clinical advantage to gain significant market share and justify its development costs.

Precision BioSciences Secures FDA Nod to Advance DMD Gene Editing Trial

Event summary

  • Precision BioSciences received a Study May Proceed notification from the FDA for its PBGENE-DMD gene editing therapy for Duchenne Muscular Dystrophy (DMD).
  • The Phase 1/2 FUNCTION-DMD clinical trial will target ambulatory DMD patients with mutations between exons 45 and 55, representing approximately 60% of DMD cases.
  • PBGENE-DMD aims to restore near full-length dystrophin protein production, a larger construct than currently approved therapies.
  • Initial data from the trial is expected by year-end 2026, assessing dystrophin protein expression from muscle biopsies.

The big picture

Precision BioSciences’ advancement of PBGENE-DMD represents a significant step in the gene editing space, particularly for rare diseases like DMD where treatment options are limited. The company’s ARCUS platform, utilizing a gene excision approach, differentiates it from competitors pursuing micro-dystrophin or exon skipping strategies. The success of this trial could validate the ARCUS platform and open doors for its application to other genetic disorders, but also faces the inherent risks associated with in vivo gene editing and the complexities of the DMD disease landscape.

What we're watching

Clinical Efficacy
The initial data release in late 2026 will be critical in determining whether PBGENE-DMD’s gene excision approach translates to meaningful dystrophin restoration and functional improvement in patients.
Regulatory Path
The FDA’s alignment on a regulatory path following the initial patient data will dictate the speed and likelihood of commercialization, potentially influencing Precision BioSciences’ valuation.
Competitive Landscape
The success of PBGENE-DMD will be weighed against the progress of other DMD therapies, including micro-dystrophin constructs and exon skipping treatments, impacting Precision BioSciences’ market share potential.

Precision BioSciences Prioritizes HBV, DMD Gene Editing Programs Amidst Cash Runway

Event summary

  • Precision BioSciences (DTIL) expects ~$137 million in cash, cash equivalents, and restricted cash as of December 31, 2025, providing a runway through 2028.
  • The Phase 1/2a ELIMINATE-B trial for chronic Hepatitis B (PBGENE-HBV) has shown dose-dependent antiviral activity and is expanding to include additional cohorts with shorter dosing intervals.
  • The Phase 1/2 FUNCTION-DMD trial for Duchenne Muscular Dystrophy (PBGENE-DMD) is expected to dose the first patient in late Q1/early Q2 2026.
  • Precision received an $8 million milestone payment from Imugene related to progress with the Azer-Cel CAR-T therapy.

The big picture

Precision BioSciences' focus on HBV and DMD highlights the growing interest in gene editing therapies for previously intractable diseases. While the ARCUS platform holds promise, the company's financial runway and reliance on partner progress create inherent risks. The success of these programs will be a key test of the viability of in vivo gene editing approaches in addressing significant unmet medical needs.

What we're watching

Clinical Efficacy
The durability of the antiviral response in the ELIMINATE-B trial will be critical to demonstrating a potential cure for Hepatitis B and justifying further development.
Regulatory Path
The FDA’s feedback on the FUNCTION-DMD trial design and initial data will dictate the speed and likelihood of a potential Biologics License Application.
Partner Performance
Imugene and iECURE’s progress with Azer-Cel and ECUR-506, respectively, will impact Precision’s future milestone payments and royalty revenue.
CID: 268