PBGENE-DMD / Precision BioSci - LARVOL DELTA

PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model (ASGCT 2026) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Precision BioSciences Inc…announced that the U.S. Food and Drug Administration has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy. (Investing.com) - "The FUNCTION-DMD study is expected to enroll ambulatory DMD patients between ages 2-7 with mutations between exons 45 and 55."

Fast track • Duchenne Muscular Dystrophy

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) (clinicaltrials.gov) - P1/2 | N=18 | Not yet recruiting | Sponsor: Precision BioSciences, Inc.

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Precision BioSciences…announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA) (Businesswire) - "This allows Precision BioSciences to initiate IRB activities and clinical trial site activation for the FUNCTION-DMD Phase 1/2 clinical trial for PBGENE-DMD for the treatment of ambulatory Duchenne muscular dystrophy (DMD) patients at highly specialized clinical trial sites. The objective of the study is to evaluate safety, tolerability and efficacy, including dystrophin protein expression and functional outcomes in patients afflicted with DMD....'We’re excited to bring this novel gene excision approach for DMD to the clinic with the goal of activating the first clinical site in the U.S. in the first half of 2026'."

IND • New P1/2 trial • Duchenne Muscular Dystrophy

FDA grants orphan drug status to Precision BioSciences’ DMD treatment (Investing.com) - "Precision BioSciences, Inc...announced Wednesday that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its PBGENE-DMD treatment for Duchenne muscular dystrophy (DMD)."

Orphan drug • Duchenne Muscular Dystrophy

Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD Further Supporting Advancement of Novel Gene Editing Approach for the Treatment of Duchenne Muscular Dystrophy Towards Clinic (Businesswire) - "Precision BioSciences, Inc...announced updated additional preclinical data further validating the scientific rationale supporting the rapid development of PBGENE-DMD...'In a long-term durability study in a DMD diseased mouse model, we have observed up to a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius, up to 85% of cells were dystrophin-positive.'....The final IND-enabling toxicology studies are currently underway with IND and/or CTA filing targeted in 2025 and initial clinical data expected in 2026."

IND • Preclinical • Duchenne Muscular Dystrophy

Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (Businesswire) - "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).....'We look forward to completing the final IND-enabling toxicology studies currently underway and generating initial clinical data which is expected in 2026'."

Clinical data • FDA event • Duchenne Muscular Dystrophy

ARCUS-Mediated Gene Editing Excision of Exons 45-55 of the Human Dystrophin Gene using PBGENE-DMD Leads to Functional Dystrophin Protein and Durable Restoration of Skeletal Muscle-Function In Vivo for the Treatment of Duchenne Muscular Dystrophy (ASGCT 2025) - "This in vivo durability and efficacy study further supports the therapeutic potential of PBGENE-DMD for the treatment of DMD and ongoing development toward clinical trials. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Becker Muscular Dystrophy • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy • Pediatrics • Respiratory Diseases • PAX7

Precision BioSciences Presents Preclinical Efficacy and Durability Data on PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (Businesswire) - "Key findings from the study include: (i) Functional dystrophin protein production: PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein across multiple muscles, including heart, diaphragm, and skeletal muscles at levels expected to provide therapeutic benefit; (ii) Enhanced Muscle Resilience: Treated mice exhibited a 66% improvement in resistance to eccentric injury, an indicator of enhanced muscle resilience, compared to untreated diseased counterparts; (iii) Long-Term Functional Improvement: In mice treated with PBGENE-DMD the maximum force output (MFO), a critical functional metric, reached up to 93% of the MFO in healthy control mice with improvement observed in PBGENE-DMD-treated mice between 3 and 6 months."

Preclinical • Duchenne Muscular Dystrophy

Precision BioSciences Announces Oral Presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (Businesswire) - "Precision BioSciences, Inc...today announced that the company will present preclinical data from the PBGENE-DMD 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."

Preclinical • Duchenne Muscular Dystrophy