This section highlights FDA-related milestones and regulatory updates for drugs developed by Precision BioSciences (DTIL).
Over the past two years, Precision BioSciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ARCUS, PBGENE-HBV, PBGENE-DMD, and azer-cel. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
ARCUS FDA Regulatory Timeline and Events
ARCUS is a drug developed by Precision BioSciences for the following indication: Eliminate DNA of living cells and organisms.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ARCUS
- Announced Date:
- April 29, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced the activation of the first clinical trial site and the opening of patient screening and enrollment for PBGENE-DMD. PBGENE-DMD, the first-in-class in vivo gene editing treatment for Duchenne muscular dystrophy (DMD), is being evaluated in the Phase 1/2 FUNCTION-DMD study.
AI Summary
Precision BioSciences announced that Arkansas Children’s Hospital has been activated as the first clinical trial site and is now screening and enrolling patients for the Phase 1/2 FUNCTION-DMD study of PBGENE-DMD. The site activation opens patient recruitment for this first-in-human study, marking the start of clinical testing and allowing qualified patients to be evaluated for participation.
PBGENE-DMD is a first-in-class, in vivo gene editing therapy aimed at Duchenne muscular dystrophy (DMD). The program uses a muscle-targeted excision approach to remove or correct disease-causing genetic sequences in muscle tissue. The FUNCTION-DMD trial will assess safety, tolerability and early signs of clinical benefit as researchers determine whether this gene editing strategy can address the underlying cause of DMD. Additional sites and enrollments are expected as the study progresses.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- April 28, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that new preclinical data from its PBGENE-DMD program have been accepted for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts.
AI Summary
Precision BioSciences announced that new preclinical data from its PBGENE-DMD program were accepted for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting, taking place May 11–15, 2026, in Boston, Massachusetts. The abstract is titled "PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model." The submission reports that the gene‑editing approach produced meaningful, lasting improvements in muscle function and favorable safety signals in a humanized DMD mouse model, based on the company’s preclinical testing.
PBGENE-DMD is described as a muscle‑targeted excision program designed to correct DMD mutations through targeted gene editing. The oral presentation will provide detailed preclinical efficacy, safety, and functional outcome data, giving researchers and clinicians an early look at the program’s translational potential for treating Duchenne muscular dystrophy.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- March 10, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc. announced presentation of new preclinical study data supporting the potential long-term efficacy of PBGENE-DMD.
AI Summary
Precision BioSciences presented new preclinical data supporting the potential long-term efficacy of PBGENE-DMD. The results reinforce a differentiated in vivo gene‑editing approach aimed at editing muscle satellite cells to achieve durable functional muscle improvement. In a humanized Duchenne muscular dystrophy (DMD) mouse model, PBGENE‑DMD treatment improved muscle pathology and lowered biomarkers of muscle damage.
The studies also showed durable restoration of dystrophin protein across key muscle groups, including cardiac, diaphragm, and skeletal muscles, suggesting sustained benefit in tissues most affected by DMD. These findings support the idea that satellite cell editing can maintain dystrophin expression and functional gains over time, strengthening the rationale to advance PBGENE‑DMD toward clinical testing as a potential one‑time gene‑editing therapy for DMD.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- March 9, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Precision BioSciences announced that the U.S. Food and Drug Administration has granted Fast Track designation to PBGENE-DMD, its investigational gene‑editing therapy for Duchenne muscular dystrophy (DMD). Precision, which uses its ARCUS® genome‑editing platform to develop in vivo treatments, said the designation recognizes the serious nature of DMD and the urgent need for new therapies.
Fast Track status is intended to help speed development and review of treatments for serious conditions with unmet medical needs. For PBGENE‑DMD, the designation may enable more frequent and efficient interactions with the FDA and could accelerate the path to clinical testing and potential approval if early results are promising.
The company also plans a virtual expert webinar on March 17, 2026, to discuss PBGENE‑DMD and the planned Phase 1/2 FUNCTION‑DMD clinical study, underscoring its move toward clinical evaluation in patients with DMD.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- February 18, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that new preclinical study data supporting the potential safety and long-term efficacy of PBGENE-DMD has been selected as a poster presentation at the upcoming 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 8-11 in Orlando, Florida.
AI Summary
Precision BioSciences announced that new preclinical study data for PBGENE-DMD, which support the therapy’s potential safety and long-term efficacy, have been selected for a poster presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference. The meeting takes place March 8–11 in Orlando, Florida. The poster will allow researchers to review preclinical findings, including safety signals and durability of effect observed in animal models, and will highlight data points that inform next steps for development.
PBGENE-DMD is a genome-editing therapeutic candidate being developed to treat Duchenne muscular dystrophy (DMD). Having a poster at the MDA conference lets Precision share early data with clinicians and scientists, gather feedback, and explore collaboration opportunities. The company says the results support continued development and further studies to confirm safety and long-term benefit in patients, and it will discuss the findings with the scientific community at the conference.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- February 11, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA).
AI Summary
Precision BioSciences announced it received a Study May Proceed notification from the U.S. Food and Drug Administration, allowing the company to begin clinical trial site activation for the FUNCTION-DMD Phase 1/2 study in patients with Duchenne muscular dystrophy (DMD). This milestone clears an important regulatory step so investigators can start preparing sites to enroll patients.
The company said Institutional Review Board (IRB) reviews are already underway at multiple DMD clinical trial centers. The investigational product, PBGENE-DMD, is an in vivo gene editing therapy designed as a one-time treatment intended to correct the underlying genetic cause of DMD.
Precision plans a virtual event in March, following the Muscular Dystrophy Association meeting, to discuss PBGENE-DMD and the FUNCTION-DMD study. The event will include key opinion leaders from the medical and patient advocacy communities, with more details to be announced later.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- January 12, 2026
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc today provided a business update and announced strategic priorities for 2026, highlighting recent progress for its two lead programs, upcoming clinical milestones, and a strong financial position supporting execution through key value-inflection points.
AI Summary
Precision BioSciences provided a 2026 business update highlighting progress for its two lead programs and upcoming clinical milestones. PBGENE‑HBV (ELIMINATE‑B) is in a Phase 1/2a dose‑finding study with multiple dosing cohorts. Early results showed dose‑dependent antiviral activity and molecular evidence of viral DNA editing from paired biopsies, with a manageable safety profile up to 0.8 mg/kg. Cohorts 3–5 are being dosed, including shorter interval schedules, and 12 participants have received at least one dose. Additional biopsy data and data updates are expected in the first half of 2026 as the company seeks an optimal regimen and plans Part 2 expansion.
PBGENE‑DMD (FUNCTION‑DMD) expects IND clearance in Q1 2026, plans first patient dosing in late Q1/early Q2, and aims to report initial multi‑patient data by year‑end 2026 measuring near full‑length dystrophin in muscle biopsies. Precision reported approximately $137 million in unaudited cash and equivalents as of Dec 31, 2025, which it expects will fund key PBGENE‑HBV and PBGENE‑DMD milestones through 2028.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- November 10, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced a late-breaking oral presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2025.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- October 14, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that it has been selected to deliver a late-breaking oral presentation at the Liver Meeting® 2025.
AI Summary
Precision BioSciences, Inc., a clinical-stage gene editing company, has been chosen for a late-breaking oral presentation at the Liver Meeting® 2025. This annual conference, hosted by the American Association for the Study of Liver Diseases, will take place in Washington, D.C., from November 7–11, 2025.
The presentation will feature new data on PBGENE-HBV from the first two cohorts of the Phase 1 ELIMINATE-B trial. These initial results highlight the therapy’s safety profile and antiviral activity against chronic hepatitis B.
PBGENE-HBV is Precision’s in vivo gene editing program designed to eliminate the viral reservoir and inactivate integrated hepatitis B virus DNA. It is the first therapy of its kind to enter human clinical trials with the goal of achieving a functional cure.
The ELIMINATE-B study is enrolling HBeAg-negative patients across multiple countries to determine the optimal dosing strategy for clearing viral DNA and driving long-term responses.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- September 30, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc. announced a late-breaking poster presentation at the 30th Annual International Congress of the World Muscle Society (WMS) being held October 7-12, 2025, in Vienna, Austria.
AI Summary
Precision BioSciences announced a late-breaking poster presentation at the 30th Annual International Congress of the World Muscle Society in Vienna, Austria, October 7–12, 2025, to share new data on PBGENE-DMD. This gene editing therapy for Duchenne Muscular Dystrophy excises exons 45–55 to restore near full-length dystrophin protein.
PBGENE-DMD uses two ARCUS nucleases in one AAV to edit the dystrophin gene. In mice, it restored dystrophin in key muscles – quadriceps, gastrocnemius, heart, and diaphragm – for up to nine months. Treated mice had up to 85% dystrophin-positive cells and stronger maximum force output at 3, 6, and 9 months. Edited satellite stem cells suggested lasting benefit.
Targeting mutations in up to 60% of DMD patients, Precision plans to file an IND or CTA by the end of 2025. If approved, clinical trials could start in 2026. PBGENE-DMD may become a first-in-class, one-time gene editing treatment offering lasting gains for patients with Duchenne Muscular Dystrophy.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- September 30, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that its On track to file an IND and/or CTA by the end of 2025; clinical data anticipated in 2026-
AI Summary
Precision BioSciences shared long-term preclinical results for PBGENE-DMD, its one-time gene editing therapy for Duchenne muscular dystrophy. Using two ARCUS® nucleases in a single AAV delivery, the program drove durable increases in dystrophin protein, boosted the number of dystrophin-positive muscle cells, and produced sustained muscle strength improvements in a mouse model up to nine months after treatment. PBGENE-DMD also edited satellite stem cells, suggesting the potential for long-lasting benefit.
The company is advancing final toxicology studies and remains on track to file an IND or CTA by the end of 2025. Initial clinical data are anticipated in 2026, moving PBGENE-DMD closer to possible first-in-class status for patients with dystrophin mutations between exons 45–55.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- September 12, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that the Company presented clinical data from the Phase 1 ELIMINATE-B trial of PBGENE-HBV in patients with chronic Hepatitis B.
AI Summary
Precision BioSciences, a clinical stage gene editing company, presented early results from its Phase 1 ELIMINATE-B trial of PBGENE-HBV at the ICE-HBV Symposium in Berlin on September 12. PBGENE-HBV uses the ARCUS platform to directly target and remove cccDNA and inactivate integrated HBV DNA in chronic hepatitis B patients. After two dose levels across Cohort 1 and Cohort 2, the therapy has been well tolerated and showed the first evidence of substantial HBsAg reductions. The trial is enrolling HBeAg-negative patients at sites in Moldova, Hong Kong, and New Zealand, with upcoming expansion to the U.S. and U.K. Lipid nanoparticle delivery was provided by Acuitas Therapeutics. Cohort 3 dosing has begun, and further data updates are expected in 2025. These results support PBGENE-HBV’s potential as a first-in-human, curative gene editing treatment for chronic hepatitis B.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- July 16, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the Company's first-in-class in vivo gene editing approach for Duchenne muscular dystrophy (DMD), towards first-in-human clinical investigation.
AI Summary
Precision BioSciences announced new preclinical data that further supports the rapid development of PBGENE-DMD, its first-in-class in vivo gene editing therapy for Duchenne muscular dystrophy (DMD). In studies using a DMD mouse model, the therapy showed a strong increase in dystrophin-positive muscle cells across key muscle groups, including the quadriceps, calf, heart, and diaphragm. Notably, up to 85% of cells in the calf were positive for dystrophin. The results suggest that the sustained improvement in muscle function may be due, in part, to the editing of muscle satellite cells. PBGENE-DMD is designed to permanently correct dystrophin mutations by excising specific exons, potentially benefiting up to 60% of DMD patients with mutations in the targeted “hot spot” region. These compelling findings bolster the approach and support a move toward first-in-human clinical investigations.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- June 25, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Precision BioSciences has received a Rare Pediatric Disease Designation from the FDA for its PBGENE-DMD therapy, a significant milestone in the fight against Duchenne muscular dystrophy (DMD). This designation highlights the urgent need for new treatments, as DMD affects a small population of children and is both serious and life-threatening. PBGENE-DMD leverages the company’s ARCUS® gene editing platform to excise exons 45-55 in the dystrophin gene. This process aims to restore a near full-length dystrophin protein, potentially offering lasting functional benefits for up to 60% of DMD patients. Additionally, if the therapy is approved, Precision may be eligible for a Priority Review Voucher, which can either expedite the review of another product or be sold to support further research. The company is currently finalizing toxicology studies and aims to start clinical trials in 2026.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- April 15, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PBGENE-HBV, the Company's lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes.
AI Summary
Precision BioSciences has received Fast Track designation from the FDA for its lead in vivo gene editing program, PBGENE-HBV, which is designed to cure chronic hepatitis B. This treatment targets the underlying cause of the infection by eliminating cccDNA—the main source of replicating hepatitis B virus—and by inactivating integrated HBV DNA in liver cells. The Fast Track status aims to speed up PBGENE-HBV’s development by allowing more frequent interactions with the FDA and a rolling review process. Using the innovative ARCUS platform, the program focuses on a direct approach to potentially cure chronic hepatitis B, offering a new option compared to current lifelong therapies that only suppress the virus. This milestone highlights the promising potential of PBGENE-HBV to address the urgent need for improved treatment alternatives for the millions affected by chronic hepatitis B worldwide.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- March 19, 2025
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of Duchenne muscular dystrophy (DMD) during an oral presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19, 2025 in Dallas, TX.
AI Summary
Precision BioSciences, Inc. announced that it will present preclinical data for its PBGENE-DMD development program at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, TX from March 16-19, 2025. The presented data highlights a pioneering gene editing approach that precisely removes exons 45-55 from the dystrophin gene. This correction enables the body to produce a near full-length, functional dystrophin protein, potentially benefiting up to 60% of Duchenne muscular dystrophy (DMD) patients. In preclinical studies using a humanized DMD mouse model, PBGENE-DMD restored dystrophin protein expression, improved muscle function significantly, and demonstrated long-term durability. The treatment also showed gene correction in muscle satellite stem cells, suggesting a permanent benefit. These promising results support the further clinical development of this widely applicable gene editing therapy for DMD.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- December 18, 2024
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced that it has received Clinical Trial Application (CTA) approval in Hong Kong to study PBGENE-HBV in the ongoing ELIMINATE-B Phase I trial. PBGENE-HBV is Precision's lead wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), and inactivating integrated HBV DNA in hepatocytes.
AI Summary
Precision BioSciences has received Clinical Trial Application approval in Hong Kong for its Phase I ELIMINATE-B trial studying PBGENE-HBV. This trial marks a key milestone as the first approval for an in vivo gene editing approach targeting chronic hepatitis B in Hong Kong. PBGENE-HBV is Precision’s lead program designed to cure chronic hepatitis B by eliminating the covalently closed circular DNA (cccDNA), which is the main source of viral replication, and by inactivating integrated HBV DNA in liver cells.
The trial is part of a global, multi-site study that is actively recruiting patients, including at a top infectious disease site in Hong Kong and in Moldova. The company expects to report emerging clinical data throughout 2025, with U.S. investigational new drug plans also anticipated later this year.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- November 15, 2024
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc today will present preclinical data supporting the advancement of PBGENE-HBV into clinical development.
AI Summary
Precision BioSciences, Inc. announced today that its preclinical data supports advancing its PBGENE-HBV therapy into clinical development. The data demonstrates a strong safety profile, high specificity, and effective distribution to liver cells, which is critical for targeting the root causes of chronic hepatitis B. The results showed robust viral DNA editing and dual action in eliminating both covalently closed circular DNA (cccDNA) and integrated viral DNA. These promising findings back the transition to a first-in-human study.
The upcoming global Phase 1 trial, called ELIMINATE-B, will initially enroll up to 45 patients who are controlled, yet not cured, by existing treatments. Precision BioSciences aims to assess both safety and potential antiviral efficacy during this trial, with patient dosing expected to begin in 2025. This milestone highlights a significant step forward in developing a novel gene editing approach for chronic hepatitis B treatment.
Read Announcement- Drug:
- ARCUS
- Announced Date:
- September 12, 2024
- Indication:
- Eliminate DNA of living cells and organisms.
Announcement
Precision BioSciences, Inc announced changes within the Company's clinical leadership team strengthening its infectious disease and hepatitis capabilities as it prepares to initiate development of its first in vivo gene editing candidate for hepatitis.
AI Summary
Precision BioSciences is strengthening its clinical leadership team to enhance its infectious disease and hepatitis capabilities ahead of launching its first in vivo gene editing candidate for hepatitis. The company has appointed Murray Abramson, MD, MPH, as Senior Vice President and Head of Clinical Development. Dr. Abramson will lead clinical, safety, and medical affairs for both the lead hepatitis program, PBGENE-HBV, and another clinical candidate nearing its regulatory filings. John Fry has been named Strategic Clinical Advisor, focusing on hepatitis, and will support the clinical development strategy for PBGENE-HBV with his extensive experience in early phase clinical trials. Meanwhile, Chief Medical Officer Alan List will retire from his executive role, transitioning to a clinical consultant role on the scientific advisory board to provide ongoing strategic guidance. These appointments are designed to solidify the company’s expertise as it prepares for pivotal clinical milestones in hepatitis treatment.
Read Announcement
PBGENE-HBV FDA Regulatory Timeline and Events
PBGENE-HBV is a drug developed by Precision BioSciences for the following indication: For chronic hepatitis B virus (HBV).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PBGENE-HBV
- Announced Date:
- April 15, 2026
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc announced that it has received Clinical Trial Application (CTA) approval to expand the ongoing global ELIMINATE-B clinical trial of PBGENE-HBV.
AI Summary
Precision BioSciences announced it received Clinical Trial Application (CTA) approval to expand the ongoing global ELIMINATE-B clinical trial of PBGENE-HBV. The expansion adds clinical sites in France and Romania to support broader patient enrollment and continued trial execution. Site initiation activities are underway, and the company expects initial patient screening in Q2 2026. Precision said the move aims to enable treatment of as many eligible clinical trial participants as possible with PBGENE-HBV across multiple countries.
PBGENE-HBV is a clinical-stage program designed to eliminate covalently closed circular DNA (cccDNA), the viral reservoir that allows chronic hepatitis B to persist, with the goal of achieving sustained loss of HBV DNA. The ELIMINATE-B trial will continue to evaluate the safety and antiviral effects of this approach across the expanded global sites.
Read Announcement- Drug:
- PBGENE-HBV
- Announced Date:
- November 19, 2025
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc announced that it will deliver an oral presentation at the Hep-DART 2025 meeting, to take place December 7–11, 2025, at the Hilton Hawaiian Village in Honolulu, Hawaii. The presentation will feature data from the Phase 1 ELIMINATE-B trial of PBGENE-HBV.
AI Summary
Precision BioSciences announced it will deliver an oral presentation at Hep‑DART 2025 in Honolulu (Dec 7–11). The talk, scheduled for Tuesday, December 9 at 4:45 p.m. HST, will be given by Man‑Fung Yuen and is titled “PBGENE‑HBV, a first‑in‑class gene editing therapy for chronic hepatitis B, demonstrates safety and antiviral activity across three cohorts.” The presentation will share clinical data from the Phase 1 ELIMINATE‑B trial of PBGENE‑HBV.
PBGENE‑HBV is an in vivo gene editing program designed to remove HBV cccDNA and inactivate integrated HBV DNA, with the goal of achieving a cure for chronic hepatitis B. The Phase 1 ELIMINATE‑B study is enrolling HBeAg‑negative patients at sites in Hong Kong, New Zealand, the United States and Moldova to define safe dosing and number of administrations. Precision plans to expand the study to the U.K. and recruit a more diverse patient population. Lipid nanoparticle delivery is provided by Acuitas Therapeutics.
Read Announcement- Drug:
- PBGENE-HBV
- Announced Date:
- August 6, 2025
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc announced ELIMINATE-B results as of the data cutoff of July 28, 2025.
AI Summary
Precision BioSciences announced results from its Phase 1 ELIMINATE-B trial of PBGENE-HBV as of the July 28, 2025 data cutoff. PBGENE-HBV is the first clinical gene editing therapy designed to eliminate covalently closed circular DNA (cccDNA) and inactivate integrated hepatitis B viral DNA, aiming for a complete cure. In Cohort 1 (0.2 mg/kg), three patients tolerated three doses without serious side effects, and all showed antiviral activity. The greatest reduction in hepatitis B surface antigen (HBsAg) ranged from 47 percent to 69 percent, with one patient maintaining a 50 percent HBsAg drop seven months after dosing.
Early safety data from Cohort 2 (0.4 mg/kg) confirm no severe adverse events or dose-limiting toxicities in the first three patients. These findings support dose escalation and shorter dosing intervals in upcoming cohorts.
Precision also expanded its cash runway to the second half of 2027 and named Dr. Mark Sulkowski as Head Clinical Development Advisor to guide the ongoing study and future trials.
Read Announcement- Drug:
- PBGENE-HBV
- Announced Date:
- May 7, 2025
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc announced that it will present initial safety data from the Phase 1 ELIMINATE-B trial evaluating PBGENE-HBV program for the treatment of chronic hepatitis B during a late breaking poster presentation at the European Association for the Study of the Liver (EASL) Congress being held May 7-10, 2025, in Amsterdam, Netherlands.
AI Summary
Precision BioSciences recently announced that it will present initial safety data from its Phase 1 ELIMINATE-B trial at the European Association for the Study of the Liver (EASL) Congress in Amsterdam from May 7-10, 2025. The trial evaluates PBGENE-HBV, a gene editing therapy designed to treat chronic hepatitis B. In a late-breaking poster presentation, the company will share early clinical results from three patients who received repeat dosing. These initial findings indicate that PBGENE-HBV is well-tolerated with no dose limiting toxicities or serious adverse events, offering encouraging signals that support planned repeat administrations and ongoing dose escalation. This presentation marks a significant step in assessing the safety and potential of the ARCUS-based therapy as it moves forward in clinical development.
Read Announcement - Drug:
- PBGENE-HBV
- Announced Date:
- March 20, 2025
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc presents preclinical data supporting repeat dosing of PBGENE-HBV in the ELIMINATE-B trial to treat patients with chronic HBV.
AI Summary
Precision BioSciences, Inc. has presented preclinical data that support the use of repeat dosing of its gene editing therapy, PBGENE-HBV, in the ELIMINATE-B Phase 1 trial for chronic hepatitis B. The company’s studies in non-human primates showed that multiple administrations of PBGENE-HBV, delivered via lipid nanoparticles, are safe and well tolerated, with no dangerous drug accumulation or effect on germ cells. These results suggest that additional doses could increase the cumulative gene editing needed to eliminate both viral DNA and cccDNA, which are crucial for a potential cure. The trial is designed to explore up to three dose administrations per dose level, aiming to achieve a durable functional cure. Further details on these promising findings will be shared at the Global Hepatitis Summit 2025.
Read Announcement- Drug:
- PBGENE-HBV
- Announced Date:
- March 17, 2025
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PBGENE-HBV.
AI Summary
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for PBGENE-HBV. This clearance marks a significant regulatory milestone for the company’s lead in vivo gene editing program that aims to cure chronic hepatitis B by eliminating cccDNA and inactivating integrated HBV DNA in liver cells. PBGENE-HBV is the first gene editing therapy of its kind to enter clinical trials in the U.S. as part of the Phase 1 ELIMINATE-B study. The trial, already underway in Moldova, Hong Kong, and New Zealand, will now expand to include U.S. sites, with enrollment starting at Massachusetts General Hospital. This development highlights the potential of Precision BioSciences’ ARCUS® platform to offer new curative treatments for a disease affecting millions globally.
Read Announcement- Drug:
- PBGENE-HBV
- Announced Date:
- September 30, 2024
- Indication:
- For chronic hepatitis B virus (HBV)
Announcement
Precision BioSciences, Inc announced that the Company submitted Clinical Trial Applications (CTA) to initiate a Phase 1study evaluating PBGENE-HBV.
AI Summary
Precision BioSciences, Inc. has submitted Clinical Trial Applications (CTA) to begin a Phase 1 clinical study evaluating its novel gene editing therapy, PBGENE-HBV. This therapy is designed to treat chronic hepatitis B by directly targeting and eliminating the viral cccDNA and inactivating integrated HBV DNA, which are the root causes of disease progression. The approach offers a potential functional cure that goes beyond traditional treatments, which only provide limited chances of long-term suppression.
The company’s application is supported by robust non-human primate safety data and multiple preclinical efficacy studies, marking a significant milestone in advancing the first clinical-stage in vivo gene editing program for HBV. Precision BioSciences plans to share final clinical candidate safety results and detailed Phase 1 trial plans in November, ahead of the American Association for the Study of Liver Diseases (AASLD) meeting.
Read Announcement
PBGENE-DMD FDA Regulatory Timeline and Events
PBGENE-DMD is a drug developed by Precision BioSciences for the following indication: For the Treatment of Duchenne Muscular Dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- PBGENE-DMD
- Announced Date:
- March 10, 2026
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Precision BioSciences, Inc. announced presentation of new preclinical study data supporting the potential long-term efficacy of PBGENE-DMD.
AI Summary
Precision BioSciences announced new preclinical data supporting the potential long-term efficacy of PBGENE-DMD, an in vivo gene editing therapy for Duchenne muscular dystrophy. The studies, carried out in a humanized DMD mouse model, describe a differentiated approach designed to edit satellite cells — the muscle stem cells that help regenerate tissue — with the goal of achieving durable functional muscle improvement rather than only short-term benefits.
In treated mice, PBGENE-DMD improved muscle pathology and reduced biomarkers of muscle damage. The studies showed durable restoration of dystrophin protein across key muscle groups, including the heart, diaphragm, and skeletal muscles. These results suggest the therapy could provide long-lasting dystrophin expression and broad tissue benefit, and the company presented the data to highlight the therapy’s potential and inform next steps in development.
Read Announcement- Drug:
- PBGENE-DMD
- Announced Date:
- February 11, 2026
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Precision BioSciences, Inc announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA).
AI Summary
Precision BioSciences announced it received a Study May Proceed notification from the U.S. Food and Drug Administration, allowing the company to begin clinical trial site activation for the Phase 1/2 FUNCTION‑DMD study in patients with Duchenne muscular dystrophy (DMD). This notification permits site activation activities such as staff training and logistics to get the trial ready.
PBGENE‑DMD is an investigational in vivo gene editing therapy designed as a one‑time treatment to correct the underlying genetic cause of DMD. Institutional Review Board (IRB) review is already underway at multiple world‑class DMD clinical sites, a necessary step before those centers can start screening and enrolling patients.
Precision plans a virtual event in March, after the Muscular Dystrophy Association meeting, to discuss PBGENE‑DMD and the FUNCTION‑DMD study. The session will include key opinion leaders and patient advocates, and the company said further details will be announced later.
Read Announcement- Drug:
- PBGENE-DMD
- Announced Date:
- July 23, 2025
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). This milestone highlights the critical need for new therapies that can improve muscle function in boys living with DMD.
Cindy Atwell, Chief Development and Business Officer at Precision BioSciences, said the designation, together with their Rare Pediatric Disease status and strong preclinical data, gives the company confidence as it moves PBGENE-DMD toward clinical trials. Initial human data is expected in 2026 as they continue discussions with the FDA.
Orphan Drug Designation is reserved for treatments targeting rare diseases affecting fewer than 200,000 people in the U.S. It provides financial incentives for clinical development and up to seven years of market exclusivity if approved.
PBGENE-DMD uses two ARCUS nucleases delivered by a single AAV vector to remove exons 45–55 of the dystrophin gene. This one-time treatment aims to restore near full-length dystrophin protein and could help up to 60% of DMD patients.
In animal studies, PBGENE-DMD effectively targeted key muscles, including cardiac tissue, produced durable functional improvements, restored dystrophin production, and edited satellite muscle stem cells for long-term benefit.
Read Announcement- Drug:
- PBGENE-DMD
- Announced Date:
- July 16, 2025
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Precision BioSciences, Inc announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD, the Company's first-in-class in vivo gene editing approach for Duchenne muscular dystrophy (DMD), towards first-in-human clinical investigation.
AI Summary
Precision BioSciences, Inc. has released promising new preclinical data for its PBGENE-DMD candidate, a first-in-class in vivo gene editing therapy for Duchenne muscular dystrophy (DMD). The study in a DMD mouse model showed up to a three-fold increase in dystrophin-positive muscle cells in muscles such as the quadriceps, calf, heart, and diaphragm, with 85% positive cells observed in the calf muscle. These results suggest that PBGENE-DMD not only enhances muscle function but may also achieve sustained benefits by editing muscle satellite cells. This unique approach aims to restore near full-length dystrophin protein in patients and could potentially help about 60% of DMD patients with mutations in the critical exons 45-55 region. Precision BioSciences plans to file for an Investigational New Drug (IND) or Clinical Trial Application (CTA) in 2025, with clinical results expected in 2026.
Read Announcement- Drug:
- PBGENE-DMD
- Announced Date:
- June 25, 2025
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Precision BioSciences, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Precision BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for its gene editing candidate, PBGENE-DMD, aimed at treating Duchenne muscular dystrophy (DMD). This designation acknowledges the significant unmet need for new therapies for this severe and life-threatening pediatric condition, which affects fewer than 200,000 people in the U.S., including about 15,000 with DMD. PBGENE-DMD uses the company’s ARCUS gene editing platform to excise exons 45-55 of the dystrophin gene, aiming to restore near full-length dystrophin protein in patients. This approach could potentially benefit up to 60% of DMD patients by offering improved, durable functional outcomes. In addition, the designation may help Precision BioSciences secure a Priority Review Voucher upon FDA approval, adding a valuable financial incentive to accelerate further product development.
Read Announcement
Azer-cel FDA Regulatory Events
Azer-cel is a drug developed by Precision BioSciences for the following indication: for Multiple Sclerosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- azer-cel
- Announced Date:
- August 9, 2024
- Indication:
- for Multiple Sclerosis
Announcement
Precision BioSciences, Inc. announced that its partner TG Therapeutics has received U.S. Food and Drug Administration (FDA) clearance for its Investigational New Drug (IND) Application to investigate Azercabtagene Zapreleucel (azer-cel) in human clinical trials for the treatment of progressive forms of multiple sclerosis.
AI Summary
Precision BioSciences announced that its partner, TG Therapeutics, has received U.S. FDA clearance for its Investigational New Drug (IND) application to study Azercabtagene Zapreleucel (azer-cel) in human clinical trials. Azer-cel is an experimental allogeneic CAR T therapy designed for treating progressive forms of multiple sclerosis. The clearance allows TG Therapeutics to initiate a phase 1 clinical trial in 2024, which will evaluate how safe and effective azer-cel is for patients with chronic autoimmune conditions. Precision BioSciences first discovered azer-cel and has since licensed it to TG Therapeutics for development outside of cancer treatments. This milestone could mark an important step in extending CAR T therapies into the autoimmune field, potentially offering new treatment options for individuals suffering from progressive multiple sclerosis and other similar diseases.
Read Announcement
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