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
Standing together in red for #WorldDuchenneAwarenessDay, the Precision BioSciences team proudly supports the global fight against Duchenne muscular dystrophy and reaffirms our commitment to advancing hope through groundbreaking gene editing solutions.
Learn more about PBGENE-DMD, Precision’s development program for the treatment of DMD, at our website!
https://t.co/Vn1Pqw405u
#CureDuchenne #SupportDuchenne #DMD #WDAD
Precision BioSciences Reports Fourth Quarter and Fiscal Year 2024 Financial Results and Provides Business Update!
✅ Announced first in vivo clinical validation for ARCUS gene editing across two distinct programs including wholly owned PBGENE-HBV and partnered program ECUR-506
✅ Commenced Phase 1 ELIMINATE-B clinical trial for PBGENE-HBV, the first gene editing technology studied for Hepatitis B, in the fourth quarter of 2024
✅ Demonstrated PGENE-HBV was safe and well tolerated with substantial antiviral activity measured by reduction of Hepatitis B surface antigen after only one administration at lowest dose level; Subsequent doses of PBGENE-HBV to follow with data expected throughout 2025 -
✅ Announced complete clinical response in first infant dosed by partner iECURE in ongoing Phase 1/2 clinical trial in Ornithine Transcarbamylase (OTC) Deficiency -
✅ Anticipated cash runway into the second half of 2026 is expected to enable achievement of key Phase 1 clinical inflection points across both wholly owned in vivo gene editing programs -
Let Chief Financial Officer, Alex Kelly, tell you what you need to know.
For more info, visit https://t.co/znq3MzqwQU.
#GeneEditing #Biotech #DTIL
Precision BioSciences Reports Fourth Quarter and Fiscal Year 2024 Financial Results and Provides Business Update!
✅ Announced first in vivo clinical validation for ARCUS gene editing across two distinct programs including wholly owned PBGENE-HBV and partnered program ECUR-506
✅ Commenced Phase 1 ELIMINATE-B clinical trial for PBGENE-HBV, the first gene editing technology studied for Hepatitis B, in the fourth quarter of 2024
✅ Demonstrated PGENE-HBV was safe and well tolerated with substantial antiviral activity measured by reduction of Hepatitis B surface antigen after only one administration at lowest dose level; Subsequent doses of PBGENE-HBV to follow with data expected throughout 2025 -
✅ Announced complete clinical response in first infant dosed by partner iECURE in ongoing Phase 1/2 clinical trial in Ornithine Transcarbamylase (OTC) Deficiency -
✅ Anticipated cash runway into the second half of 2026 is expected to enable achievement of key Phase 1 clinical inflection points across both wholly owned in vivo gene editing programs -
Let Chief Financial Officer, Alex Kelly, tell you what you need to know.
For more info, visit https://t.co/znq3MzqwQU.
#GeneEditing #Biotech #DTIL
Nice to speak with Chari Cohen from the Hepatitis B Foundation and Mark Sulkowski, MD from Johns Hopkins about Hepatitis B during the Global Hepatitis Summit.
🇺🇸 What does the recent U.S. FDA Investigational New Drug (IND) clearance for PBGENE-HBV mean for clinicians?
🧬 What stands out about the early clinical data generated at the first dose level of PBGENE-HBV?
💡 What are the biggest challenges that Hepatitis B patients face today in terms of treatment and disease management?
As the sun set on Day 1 of the Global Hepatitis Summit, Alex Kelly, Chief Financial Officer, sat down with Chari Cohen, DrPH, MPH, President of the Hepatitis B Foundation, and Mark Sulkowski, M.D., Professor at Johns Hopkins University School of Medicine and Precision Scientific Advisory Board Member, to answer these questions and more.
Listen to their full conversation on Precision's YouTube channel, https://t.co/kUwLRM6BgJ.
Read our latest press release about the IND approval, https://t.co/hrXrGTSYEr
#GHS2025 #GeneEditing #Biotech
Precision receives $13 Million in proceeds from Imugene Limited convertible note maturity.
This further reinforces Precision's expected cash runway into 2H 2026.
https://t.co/EKx5BxtwZZ
#GeneEditing $DTIL
Precision will present at Sidoti Micro-Cap Investor Conference on Aug 15 @ 10am ET!
A live webcast of the presentation will be accessible on Precision’s website at https://t.co/MvVj4AlDWl.
#GeneEditing#Biotech#DTIL
World-Class Clinical Investigators added to Precision’s SAB as PBGENE-HBV Program Nears Clinical Readiness!
Together, they bring immense expertise as we expect to submit the IND &/or CTA applications for PBGENE-HBV later this year.
Learn More 👉 https://t.co/ltI1oKX62w
$DTIL
We're honored to have Wendy Shoop, PhD, represent the Precision team at the United Mitochondrial Disease Foundation’s (UMDF) Mitochondrial Medicine 2024 Conference!
https://t.co/KGsZGqlRyY
#GeneEditing#MitoMed24#DTIL
Our team had a great time attending the @HepBFoundation's Annual Gala supporting #HBV awareness and honoring continued progress towards potential cures! We’re honored to advocate for this community and are hopeful for a momentous year ahead in #hepatitis B treatments. #DTIL
Tomorrow, Wendy Shoop, PhD, will be presenting data for our PBGENE-PMM program targeting m.3243-associated primary mitochondrial myopathy at the Mitochondrial Medicine – Therapeutic Development Annual Conference. #MitoMed24#DTIL
Exciting news from iECURE! Their gene editing therapy ECUR-506 received UK approval for a clinical trial to treat OTC deficiency in infants. This CTA approval provides further regulatory support for ARCUS, our in vivo gene editing platform. #genediting
https://t.co/VSGNP0M9Mj
Following pre-IND regulatory feedback from the FDA and ex-US regulators, we are advancing PBGENE-HBV toward an IND/CTA filing in 2024. Chari Cohen, president of the @HepBFoundation, shares why this is a positive prospect for #HBV patients.
https://t.co/jVSZMJeLUv
#JPM News: We’ve completed a deal with @TGTherapeutics to develop azer-cel for autoimmune diseases. The deal extends our cash runway into 1H 2026 & funds our wholly owned PBGENE-HBV & PBGENE-PMM in vivo gene editing programs through Phase I Clinical Data. https://t.co/PqbFtNGtiZ
Precision’s Wendy Shoop, Ph.D., & @univmiami’s @Carlos_T_Moraes recently spoke with @BioWorld about the latest @NatMetabolim publication on mitoARCUS and our #investigational approach to primary mitochondrial myopathy. Read more from their conversation: https://t.co/liMMgnTC6b
We’re excited to share that our late-breaking abstract detailing a potentially curative strategy for chronic hepatitis B was accepted for presentation at the @AASLDtweets Liver Meeting on Nov. 13. We look forward to seeing you in Boston! #TLM23
This #MitoAwarenessWeek, we’re proud of advances made with our ARCUS gene editing platform, which can specifically edit mutant mitochondrial genomes, targeting primary mitochondrial myopathies. The simplicity of our technology is one way ARCUS is unique from other platforms.
It's all about the cut, size and simplicity. These three key features differentiate ARCUS from other gene editing tools. Learn more about how our ARCUS #geneediting platform drives defined outcomes here: https://t.co/ypdsoFon5x
In a scene in the movie, Sound of Freedom, Jim Caviezel, who plays the role of Tim Ballard, a former U.S. Special Agent for the Department of Homeland Security, who quit his job to rescue children from sex traffickers, is asked why he is involved in the rescue of children.
Caviezel replies : Because God’s children are not for sale 🔥
Exciting news! Our late-breaking abstract featuring new preclinical data for our PBGENE-DMD program for the potential treatment of #Duchenne muscular dystrophy has been accepted for oral presentation on May 19 at #ASGCT23: https://t.co/JPZGBPVWOR @ASGCTherapy