Precision BioSciences

Pioneers in genome editing using our proprietary ARCUS® technology to develop potentially curative therapeutics to overcome cancer and cure genetic disease.

[Precision BioSciences @PrecisionBioSci]

In case you missed the live event, here are a few of our favorite moments from Precision BioSciences’ recent virtual investor event on Duchenne Muscular Dystrophy (DMD)!

Watch the full conversation to hear the Precision team and external experts discuss PBGENE‑DMD and the upcoming Phase 1/2 FUNCTION‑DMD clinical trial.

Click here for the full replay, https://t.co/UDiLrhP4yH

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences announced its first quarter 2026 earnings and provided a business update!

CFO, Alex Kelly, tells you what you need to know.

Read the full press release, https://t.co/nHvw5a8PpJ

#GeneEditing #Biotech $DTIL

[Precision BioSciences @PrecisionBioSci]

Nothing beats learning side‑by‑side with families.

The Precision BioSciences team recently joined Jett Foundation’s Family Workshop in Salt Lake City, Utah, to listen, learn, and speak with families impacted by Duchenne muscular dystrophy.

We appreciated the opportunity to share more about PBGENE‑DMD and to hear directly from caregivers and clinicians about what matters most as new treatment options move forward.

Learn more about PBGENE-DMD and the Phase 1/2 FUNCTION-DMD study here, https://t.co/AZBj6Y3gXa

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences is expanding its global Phase 1 ELIMINATE‑B trial evaluating PBGENE‑HBV following clinical trial application approval in two European countries.

“Expanding ELIMINATE-B into hepatitis sites in France and Romania is an important step in the continued development of PBGENE-HBV, the only gene editing therapy uniquely designed to eliminate cccDNA,” said Cindy Atwell, Chief Development and Business Officer of Precision BioSciences. “Given the strong investigator interest in PBGENE-HBV, especially following the late breaker oral presentation at The Liver Conference 2025, these new trial sites will build on our existing global clinical trial footprint as we advance PBGENE-HBV through the ELIMINATE-B trial.”

To learn more, visit https://t.co/Ry9jmROaJv

$DTIL

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences was proud to join the 2026 MDA Clinical & Scientific Conference last month, where the team presented new preclinical data on PBGENE‑DMD, the company’s in vivo gene editing candidate designed to restore near full‑length dystrophin and potentially benefit boys with mutations between exons 45–55.

As Debra Miller of CureDuchenne noted, “IND clearance for PBGENE‑DMD is an exciting step forward, and we look forward to collaborating to help connect innovation with the patient families we serve.”

Read the full release, https://t.co/AZBj6Y2J7C

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences announced its Q4 and fiscal year 2025 earnings and provided a business update!

CFO, Alex Kelly, tells you what you need to know.

Read the full press release, https://t.co/FBnLNnl2ZB

#GeneEditing #Biotech $DTIL

[Precision BioSciences @PrecisionBioSci]

Join Precision BioSciences for a virtual investor event on March 17 at 9 am ET!

The event features key opinion leaders (KOL), Dr. Aravindhan Veerapandiyan, MD, Pediatric Neurologist and Associate Professor of Pediatrics, Arkansas Children’s Hospital, and Pat Furlong, Founding President of Parent Project Muscular Dystrophy, who will join Precision leaders to discuss:

- The unmet need for DMD patients
- Current treatment landscape
- PBGENE-DMD for Duchenne muscular dystrophy (DMD) and
- An overview of the clinical trial design for the FUNCTION-DMD trial

Register here: https://t.co/mnWr9VXT7Y
Read the full PR: https://t.co/ZM3IZru0Xx

$DTIL

[Precision BioSciences @PrecisionBioSci]

From the podium to the photo wall, the Precision BioSciences team is making the most of TD Cowen’s 46th Annual Health Care Conference!

Cassie Gorsuch, PhD, Chief Scientific Officer, Alex Kelly, Chief Financial Officer, and Naresh Tanna, Head of Investor Relations, highlighted the company's focused in vivo gene editing strategy and upcoming milestones for our wholly owned programs in chronic hepatitis B and Duchenne muscular dystrophy.

Want to watch the presentation replay? Visit https://t.co/0aCy6VQoex

$DTIL

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences announced that it has earned a clinical development milestone payment of $7.5 million from TG Therapeutics for the ongoing development of azer-cel in multiple sclerosis.

The payment was triggered as a result of progress in TG Therapeutics' Phase 1 study evaluating azer-cel in adults with relapsing forms of multiple sclerosis, which is being conducted under its license agreement with Precision.

The milestone payment is expected to support Precision’s cash runway through 2028 and further advancement of the company’s wholly owned in vivo gene editing programs.

Read the full release, https://t.co/xyFucV7Ekp

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences has received a U.S. FDA “Study May Proceed” notification for its investigational new drug (IND) application for PBGENE‑DMD, enabling initiation of clinical trial site activation for the Phase 1/2 FUNCTION‑DMD clinical study in patients with Duchenne muscular dystrophy (DMD).

PBGENE‑DMD is Precision’s wholly owned, first‑in‑class in vivo gene editing investigational product designed to correct the underlying genetic cause of DMD through a novel one‑time gene editing therapy targeting mutations between exons 45 and 55, the “hot‑spot” region accounting for approximately 60% of boys with DMD.

PBGENE‑DMD has been granted U.S. FDA Rare Pediatric Disease and Orphan Drug designations for the treatment of DMD.

Read the full release, https://t.co/v1z8tECvYi

[Precision BioSciences @PrecisionBioSci]

Celebrating 20 years of Precision!

Last week, our team of “Precisioneers” came together to reflect on two decades of advancing gene-editing science and the progress we’re making toward new in vivo therapies for conditions such as chronic hepatitis B and Duchenne muscular dystrophy.

As a clinical-stage gene editing company, we’re using our proprietary ARCUS platform to develop one-time, potentially durable treatments aimed at overcoming hard-to-treat genetic and infectious diseases.

Here’s to our future of hope, and to the patients, partners, and colleagues who inspire us to keep pushing the science forward.

$DTIL

[Precision BioSciences @PrecisionBioSci]

Precision BioScience is proud to share new data at #AASLD2025 for PBGENE-HBV, our first-in-class gene editing therapy for chronic hepatitis B, presented by Man-Fung Yuen, MBBS, M.D., PhD, DSc, ELIMINATE-B Investigator, Chair Professor of The University of Hong Kong, and the Chief of the Division of Gastroenterology and Hepatology at Queen Mary Hospital.

These new late-breaking data represent a milestone for Precision BioSciences and for the entire field of chronic Hepatitis B because it is the first time clinicians have been able to target the root viral source of the disease.

The safety data and tolerability profile, along with dose-dependent durable reductions in hepatitis B surface antigen and the first-ever liver biopsy data provide evidence that antiviral activity is being achieved through directly editing the viral genome in patients with chronic Hepatitis B.

This further validates ARCUS as a differentiated platform with true curative potential. With no observed dose-limiting toxicities, we look forward to finishing dosing the third cohort to generate additional data for PBGENE-HBV in our pursuit of a cure that has been so elusive in the field of Hepatitis B drug development.

Thank you to all of our investigators, collaborators, and patients who help drive this important progress. More updates to come as we push toward new standards of care for HBV.

For more information, visit https://t.co/h6sjv2SZyb

#GeneEditing #HBV #AASLD #Biotech #PrecisionBioSciences #DTIL

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences announced its Q3 2025 earnings and provided a business update!

CFO, Alex Kelly, tells you what you need to know.

Read the full press release, https://t.co/xj3pLR7Vfv

#GeneEditing #Biotech $DTIL

[Precision BioSciences @PrecisionBioSci]

Precision BioSciences was proud to join leaders from across the genetic medicines field at Chardan’s 9th Annual Genetic Medicines Conference in New York City.

Connecting with experts, investors, and innovators in this space is vital as we advance our mission to deliver transformative gene editing therapies.

Thank you to Chardan for creating a forum for meaningful discussion on the future of genetic medicines and to everyone who engaged with our team during the event.

Listen to the full webcast, https://t.co/pLqheySKps

#GeneEditing #CGMC2025 $DTIL

[Precision BioSciences @PrecisionBioSci]

Join us on Monday, November 3, at 11 am ET for a Precision BioSciences Virtual Symposium!

The company's lead muscle program, PBGENE-DMD, represents the first in vivo gene editing therapy designed to restore functional dystrophin protein and improve long-term muscle function for patients with Duchenne Muscular Dystrophy (DMD).

DMD is a rare, life-limiting disease that affects more than 15,000 individuals in the United States and many more globally. Current therapies offer only limited benefit. PBGENE-DMD has demonstrated significant and sustained improvement in muscle strength and performance in preclinical models by restoring the body’s ability to produce a near full-length dystrophin protein. This program has the potential to deliver a one-time, durable treatment that could redefine the DMD treatment landscape.

Virtual Event Details—
Date: Monday, November 3, 2025
Time: 11:00 AM Eastern Time
Moderator: Cassie Gorsuch, PhD, Chief Scientific Officer, Precision BioSciences
Featuring: Pat Furlong, President, Parent Project Muscular Dystrophy, and
Aravindhan Veerapandiyan, M.D. (Dr. Panda), Associate Professor of pediatrics in the Division of Pediatric Neurology and DMD clinical investigator at the University of Arkansas for Medical Sciences at Arkansas Children’s Hospital

Discussion Topics
• The unmet medical need in Duchenne Muscular Dystrophy
• The science behind ARCUS gene editing technology
• Precision BioSciences’ roadmap toward IND and CTA filings in 2025
• The path to anticipated clinical data in 2026

This event will offer a detailed look at the science, mission, and future vision driving PBGENE-DMD and how Precision BioSciences is advancing gene editing to deliver meaningful, long-term impact for patients and families affected by DMD.

Register to join at the link below!

https://t.co/d6x6DwPZla

#PrecisionBioSciences #GeneEditing #DuchenneMuscularDystrophy #PBGENEDMD $DTIL

[Precision BioSciences @PrecisionBioSci]

We’re proud to share that our ARCUS® platform is featured in a new peer-reviewed publication in Nucleic Acids Research, further demonstrating the broad and versatile power of our genome editing technology. The manuscript highlights how ARCUS nucleases can achieve high-frequency, precise gene insertions in both dividing and non-dividing cells—a significant milestone for therapeutic development.

This work illustrates ARCUS’ unique ability to enable complex gene edits with high efficiency, accuracy, and versatility. As we continue advancing our in vivo gene editing pipeline, these results support our vision to provide lasting solutions for diseases where patients need them most.

Congratulations to our research team and collaborators for this important achievement!

For more information, visit https://t.co/lARFnjfUjl

#GeneEditing #ARCUS #PrecisionBioSciences

[Precision BioSciences @PrecisionBioSci]

Cassie Gorsuch, PhD, Chief Scientific Officer at Precision BioSciences, represented our team and shared our latest progress at this year’s 30th Annual International Congress of the World Muscle Society.

Cassie presented new long-term preclinical data for PBGENE-DMD, showcasing lasting improvements in muscle functionality and increased dystrophin levels that highlight the potential of our one-time, in vivo gene editing approach for Duchenne muscular dystrophy.

We are proud to be on track for IND/CTA submission by late 2025, with clinical results anticipated in 2026.

Thank you to all who connected to learn more about our mission to advance durable gene editing solutions for patients.

Read the full press release, https://t.co/KKRVMoNdCp

#GeneEditing #WMS2025 #Duchenne $DTIL

[Precision BioSciences @PrecisionBioSci]

We’re excited to announce the opening of Precision BioSciences’ first U.S. clinical trial site for the ELIMINATE-B Phase 1 study, now actively recruiting patients at Massachusetts General Hospital in Boston. This marks a major milestone in Precision’s mission to develop a potential cure for Chronic Hepatitis B using our proprietary ARCUS gene editing platform.

Chronic hepatitis B affects millions worldwide and can lead to serious liver diseases. Our groundbreaking gene editing therapy, PBGENE-HBV, targets the root cause of infection, aiming to eliminate the virus for good.

We thank Massachusetts General Hospital for its partnership and leadership in this important trial. Together, we’re advancing new hope for patients with high unmet medical needs.

For more info or to see if you or someone you know qualifies, visit https://t.co/Al08pqIHQW using identifier NCT06680232.

Read the full release at https://t.co/WlLdjVvkSG

$DTIL #GeneEditing #HepatitisB #ELIMINATEB #ClinicalTrial

[Precision BioSciences @PrecisionBioSci]

This week, Precision BioSciences announced a late-breaking poster presentation at the 30th Annual International Congress of the World Muscle Society on Friday, October 10th in Vienna, Austria!

Cassie Gorsuch, Ph.D., Chief Scientific Officer, will present these encouraging results, which underscore PBGENE-DMD’s potential to transform the treatment paradigm for patients with DMD.

Precision remains on track to file an IND and/or CTA filing by the end of 2025 with clinical data anticipated in 2026.

Want to read the full release? Visit https://t.co/KKRVMoNdCp

#GeneEditing #Biotech $DTIL

[Precision BioSciences @PrecisionBioSci]

Emily Harrison, Ph.D., Head of Translational Science at Precision, presents data from the Phase 1 ELIMINATE-B trial of PBGENE-HBV with the community at this year’s 6th International Coalition to Eliminate HBV Cure Symposium.

We believe the clinical profile we have observed so far demonstrates the first evidence of substantial HBsAg reductions by a novel therapeutic approach designed to directly target cccDNA for elimination and inactivate integrated HBV DNA in patients with chronic Hepatitis B.

In the ELIMINATE-B trial, following multiple administrations across two dose levels (Cohort 1 and Cohort 2), PBGENE-HBV has been well-tolerated and active.

With the recent initiation of dosing patients in Cohort 3, we remain on track to provide further data updates in 2025.

Learn more and find the full presentation at https://t.co/HbsiU2pG7O.

#GeneEditing #Biotech $DTIL