Drugs / PBGENE-DMD
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07429240 | Apr 2026 → Nov 2029 expected | DMD-related muscular dystrophy | Precision BioSciences, Inc. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2026-05-14 | Precision BioSciences, Inc. | Results Precision BioSciences Presents New Preclinical Data Supporting the Advancement of PBGENE-DMD into Clinic at the American Society of Gene & Cell Therapy 2026 Annual Meeting precisionbiosciences.com ↗
The new data show that treatment with PBGENE-DMD in early-juvenile mice resulted in significantly higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. |
| 2026-04-28 | Precision BioSciences, Inc. | Results Precision BioSciences Announces Oral Presentation at the American Society of Gene and Cell Therapy (ASGCT) 2026 Annual Meeting precisionbiosciences.com ↗
The accepted abstract highlights new data demonstrating compelling efficacy observed in early-juvenile mice supporting the potential benefit of earlier intervention with PBGENE-DMD in younger patient populations. |
| 2026-03-10 | Precision BioSciences, Inc. | Results Precision BioSciences Presents Preclinical PBGENE-DMD Data Highlighting Durable Dystrophin Expression and Functional Benefit at the Muscular Dystrophy Association Clinical & Scientific Conference 2026 precisionbiosciences.com ↗
today announced presentation of new preclinical study data supporting the potential long-term efficacy of PBGENE-DMD. |
| 2026-03-09 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Receives FDA Fast Track Designation for PBGENE-DMD and Announces Duchenne Muscular Dystrophy Investor Event precisionbiosciences.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). |
| 2026-02-11 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application for First-in-Class PBGENE-DMD for Treatment of Duchenne Muscular Dystrophy precisionbiosciences.com ↗
today announced that the Company received a Study May Proceed notification from the U.S. Food and Drug Administration (FDA). |
| 2025-09-30 | Precision BioSciences, Inc. | Results Precision BioSciences Announces Late-Breaking Poster Presentation at the 30th Annual International Congress of the World Muscle Society precisionbiosciences.com ↗
The preclinical data to be presented demonstrates PBGENE-DMD’s ability to drive significant and sustained improvements in muscle function over time through increased frequency of dystrophin positive cells, increased dystrophin protein expression, and satellite stem cell editing. |
| 2025-08-07 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Reports Second Quarter 2025 Financial Results and Provides Business Update precisionbiosciences.com ↗
Granted FDA Rare Pediatric Disease Designation and Orphan Drug Designation for PBGENE-DMD for the treatment of DMD; |
| 2025-07-23 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Receives FDA Orphan Drug Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy precisionbiosciences.com ↗ |
| 2025-07-16 | Precision BioSciences, Inc. | Results 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 precisionbiosciences.com ↗
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. |
| 2025-06-25 | Precision BioSciences, Inc. | Regulatory Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy precisionbiosciences.com ↗
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today 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). |
| 2025-05-14 | Precision BioSciences, Inc. | Results Precision BioSciences Accelerates Development of PBGENE-DMD Within its Wholly Owned Organic Pipeline and Highlights Pre-clinical Evidence at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting precisionbiosciences.com ↗ |
| 2025-03-19 | Precision BioSciences, Inc. | Results 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 precisionbiosciences.com ↗
PBGENE-DMD restored dystrophin protein expression and significantly improved muscle function over time while demonstrating long-term durability in an in vivo DMD disease model |
| 2023-10-26 | Precision BioSciences, Inc. | Results Precision BioSciences Presents Preclinical Data Highlighting Capabilities of ARCUS for Gene Insertion and Excision at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress precisionbiosciences.com ↗
today announced that the company will present two posters at the ESGCT 30 th Annual Congress that highlight ARCUS’ differentiated ability to make efficient, durable, and targeted insertion edits in non-human primates (NHPs), as well as preclinical data demonstrating the potential of ARCUS to achieve large excision of a commonly mutated region of the dystrophin gene for the potential treatment of Duchenne muscular dystrophy (DMD). |
| 2023-09-12 | Precision BioSciences, Inc. | Results Precision BioSciences Hosts Virtual R&D Day Highlighting its Proprietary ARCUS Technology and Recent In Vivo Gene Editing Program Developments precisionbiosciences.com ↗
The Company will highlight preclinical data demonstrating the potential of ARCUS in vivo gene editing for large gene excisions and that the edited dystrophin variant was observed in multiple tissue types frequently involved in progression of DMD |
All press releases naming this drug 15 releases
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PBGENE-DMD | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07429240 ↗ |
| Route | Intravenous | “single IV dose of PBGENE-DMD” NCT07429240 ↗ |