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PBGENE-DMD (IV)

Phase 1/2Recruiting
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Interest: 29/100
29
Hype Score

Development Stage

✓
Pre-clinical
2
Phase 1
3
Phase 2
4
Phase 3
5
Approved
Indication / Disease

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Conditions

Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Trial Timeline

Mar 1, 2026 → Dec 1, 2029

NCT ID
NCT07429240

About PBGENE-DMD (IV)

PBGENE-DMD (IV) is a phase 1/2 stage product being developed by Precision BioSciences for Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD. The current trial status is recruiting. This product is registered under clinical trial identifier NCT07429240. Target conditions include Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD.

What happened to similar drugs?

2 of 20 similar drugs in Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD were approved

Approved (2) Terminated (8) Active (11)
✅Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]Sarepta TherapeuticsApproved
✅vamorolone 40 mg/mL oral suspensionSanthera PharmaceuticalsApproved
❌Tadalafil + PlaceboEli LillyPhase 3
🔄Viltolarsen + PlaceboNippon ShinyakuPhase 3
🔄ViltolarsenNippon ShinyakuPhase 3
❌fordadistrogene movaparvovecPfizerPhase 3
🔄Ataluren + PlaceboPTC TherapeuticsPhase 3

Hype Score Breakdown

Clinical
9
Activity
15
Company
5
Novelty
0
Community
0

Clinical Trials (1)

NCT IDPhaseStatus
NCT07429240Phase 1/2Recruiting

Competing Products

20 competing products in Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

See all competitors
ProductCompanyStageHype Score
Phase 1, SQY51 + Phase 2a, SQY51 (cohort 1) + Phase 2a, SQY51 (cohort 2) + Phase 2a, SQY51 (cohort 3)BiotrialPhase 1/2
29
ENTR-601-44 + ENTR-601-44 - matching placeboEntrada TherapeuticsPhase 1/2
29
ENTR-601-45 + ENTR-601-45 - matching placeboEntrada TherapeuticsPhase 1/2
29
Tadalafil + PlaceboEli LillyPhase 3
32
DS-5141bDaiichi SankyoPhase 2
39
DS-5141bDaiichi SankyoPhase 1/2
32
Bocidelpar + PlaceboAstellas PharmaPhase 1
21
scAAV9.U7.ACCAAstellas PharmaPhase 1/2
32
NS-065/NCNP-01Nippon ShinyakuPhase 2
35
Viltolarsen + PlaceboNippon ShinyakuPhase 3
40
ViltolarsenNippon ShinyakuPhase 2
35
NS-089/NCNP-02 + NS-089/NCNP-02Nippon ShinyakuPhase 2
39
NS-065/NCNP-01Nippon ShinyakuPhase 1
29
NS-050/NCNP-03 + PlaceboNippon ShinyakuPhase 1/2
39
NS-089/NCNP-02Nippon ShinyakuPhase 2
42
ViltolarsenNippon ShinyakuPhase 3
36
NS-089/NCNP-02Nippon ShinyakuPhase 1/2
32
NS-065/NCNP-01 + PlaceboNippon ShinyakuPhase 2
35
ACE-031 0.5 mg/kg q4wk + ACE-031 1.0 mg/kg q2wkMerckPhase 2
27
ACE-031 (Extension of cohort 1 from core study, A031-03) + ACE-031 (Extension of cohort 2 from core study, A031-03) + ACE-031 (Extension of cohort 3 from core study, A031-03)MerckPhase 2
27