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, azer-cel, PBGENE-DMD, and PBGENE-HBV. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
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:
- 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
azer-cel - FDA Regulatory Timeline and 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
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:
- 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
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:
- 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
