Precision BioSciences

This past weekend, Team Precision hit the courts in Raleigh, NC for the 5th Annual Dinking for Duchenne Pickleball Tournament, proudly sponsoring the event and fielding seven teams among 170+ players and supporters.

Dinking for Duchenne was started by the Foster family after their son Nathan was diagnosed with Duchenne muscular dystrophy (DMD) in 2019. It's grown into an annual event raising awareness and support for families living with DMD, benefiting Cure44 and the newly formed Never Break Foundation.

DMD is a rare, progressive genetic disorder affecting about 1 in every 5,000 boys worldwide, caused by mutations in the dystrophin gene that lead to muscle degeneration over time. There is currently no cure.

This urgent unmet need drives our work every day. Precision is developing PBGENE-DMD, an in vivo gene-editing therapy for boys ages 2-7 with Duchenne whose mutations are amenable to excision of exons 45-55, and we recently dosed the first patient in our Phase 1/2 FUNCTION-DMD trial.

Thank you to the Dinking for Duchenne community for another strong year. Grateful to be a part of it.

Read our latest DMD update, https://loom.ly/QIQX9EA

Cassie Gorsuch, Chief Scientific Officer, and Naresh Tanna, Chief Financial Officer, recently joined Patrick Trucchio at the H.C. Wainwright 28th Annual Global Investment Conference to discuss Precision BioSciences’ wholly owned clinical-stage programs.

The presentation covers PBGENE‑HBV and the ongoing ELIMINATE‑B trial, including Precision’s approach to advancing a program designed to eliminate cccDNA and support a potential path toward a viral cure for chronic hepatitis B.

Cassie and Naresh also discuss PBGENE‑DMD and the Phase 1/2 FUNCTION‑DMD study evaluating the program for Duchenne muscular dystrophy.

Watch the full presentation: https://loom.ly/gnZl9Jw

Read our latest press release: https://loom.ly/QIQX9EA

September 7 is World Duchenne Awareness Day. Today, we recognize the individuals living with Duchenne muscular dystrophy, as well as the families, caregivers, advocates, and researchers whose dedication strengthens this community every day.

Living with Duchenne brings ongoing challenges that affect not only those diagnosed, but also the people who support them. We are continually inspired by the resilience of individuals and families navigating this journey, the advocates advancing awareness and access, and the researchers working to expand what may be possible for the future.

This year marked an important milestone in Duchenne research: the first participant in the FUNCTION-DMD clinical trial received an investigational gene-editing treatment, PBGENE-DMD, designed to permanently address the underlying cause of his Duchenne muscular dystrophy. Progress like this is only possible because of the individuals and families who choose to participate in research and the clinical teams who partner with them every step of the way.

At Precision BioSciences, we are grateful for the opportunity to learn from and work alongside the Duchenne community with the hope of making a meaningful difference for individuals and families affected by Duchenne.

Precision BioSciences has commenced dosing in the Phase 1/2 FUNCTION‑DMD study, with the first patient receiving PBGENE‑DMD at Arkansas Children’s Hospital.

This milestone marks the first clinical gene‑editing program for Duchenne muscular dystrophy and advances our wholly owned in vivo gene editing approach into the clinic.

PBGENE‑DMD is designed to permanently edit a patient’s own dystrophin gene, with the aim of restoring production of a near full‑length, functional dystrophin protein. The study is enrolling ambulatory boys ages 2–7 with mutations between exons 45 and 55, a key hotspot region representing up to 60% of boys living with Duchenne.

The Precision team is grateful to the patient, their family, and the clinical team for their commitment to advancing this important work. Initial safety data are expected by year-end 2026.

Read the full release, https://loom.ly/QIQX9EA

How many of the 240 million people living with chronic hepatitis B worldwide will ever receive treatment or know they have it?

On World Hepatitis Day, we’re reminded that hepatitis B remains a leading cause of liver cancer and liver‑related death globally, yet fewer than 5% of people living with chronic HBV are receiving treatment. Most remain undiagnosed and untreated.

At Precision BioSciences, we are striving to advance PBGENE‑HBV, our wholly owned in vivo gene editing program being evaluated in the Phase 1 ELIMINATE‑B trial, and the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic hepatitis B, cccDNA. Elimination of cccDNA results in HBV viral cure because cccDNA is the only source of infectious replication (HBV DNA).

On a day dedicated to hepatitis awareness, we pause to recognize the global burden of hepatitis B and the responsibility of working on approaches that aim to change what is possible for people living with this infection.

For more information, visit https://loom.ly/sBJzafI

At PPMD’s 2026 Annual Conference in Orlando, PBGENE‑DMD was highlighted in the “Gene Therapy: Today and Tomorrow” session led by Dr. Aravindhan Veerapandiyan of Arkansas Children’s Hospital, followed by a clinician–industry panel featuring Gary Owens, Senior Director of Gene Therapy and Program Lead at Precision BioSciences.

Our team also hosted a resource booth, meeting with families and individuals with Duchenne to listen, answer questions, and share how we’re working to advance in vivo gene editing.

Interested in the latest news from PBGENE-DMD? Read, https://loom.ly/VwR_dj4

Advancing next‑generation neuromuscular therapies with ARCUS at the Oppenheimer CNS & Neuro‑Muscular Summit in Miami.

Precision BioSciences was pleased to participate, joining leaders across neurology and neuromuscular disease to discuss next‑generation therapeutic approaches.

Chief Financial Officer Alex Kelly represented Precision BioSciences and PBGENE-DMD, the Company's development program for the treatment of Duchenne Muscular Dystrophy (DMD). Alex spoke on a panel focused on emerging treatments for genetic muscle disorders and the potential of in vivo gene editing.