PBGENE-DMD / Precision BioSci - LARVOL DELTA

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