PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN.
Duchenne Muscular Dystrophy
Explore 56 assets in Duchenne Muscular Dystrophy →Floatz Rating
CCC51/100
Confidence
Indicative
Sponsor
Pfizer
Modality
gene_therapy
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
36Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05689164CT.gov A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. | — | Terminated | — | — | — |
NCT05429372CT.gov Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy | — | Terminated | — | — | — |
NCT04281485CT.gov Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | — | Active Not Recruiting | — | — | — |
NCT03362502CT.gov A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy | — | Terminated | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Duchenne Muscular Dystrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN. (this asset) | Pfizer | P3 | CCC · 51 |
| Viltolarsen | NS Pharma, Inc. | P4 | BBB |
| Golodirsen | Rare Disease Research, LLC | P4 | BB |
| Aoc 1044 | Avidity Biosciences, Inc. | P3 | BB |
| Delandistrogene Moxeparvovec | Sarepta Therapeutics, Inc. | P3 | BB |
| Glutamine | Assistance Publique - Hôpitaux de Paris | P3 | BB |
| Eteplirsen | Sarepta Therapeutics, Inc. | P3 | BB |
| Prednisolone | Anne M. Connolly | P4 | BB |
| Casimersen | Kevin Flanigan | P3 | BB |
+41 more in the Duchenne Muscular Dystrophy cohort
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. PF-06939926 IS A RECOMBINANT AAV VECTOR CONTAINING THE AAV SEROTYPE 9 (AAV9) CAPSID PACKAGING A MINIATURIZED CODON-OPTIMIZED VERSION OF THE DMD GENE(CALLED OPTI-DYS.DELTA.3978) WHICH ENCODES THE DOMAINS MINIMALLY REQUIRED FOR FUNCTIONALITY OF THE DYSTROPHIN PROTEIN (MINI-DYSTROPHIN). A SYNTHETIC POLYADENYLATION (SPOLYA) SEQUENCE FOLLOWS THE OPTI-DYS.DELTA.3978 GENE. CARDIAC AND SKELETAL MUSCLE-SPECIFIC TRANSGENE EXPRESSION IS MEDIATED BY A SYNTHETIC ENHANCER AND PROMOTER. THE EXPRESSION CONSTRUCT IS FLANKED BY THE AAV2 INVERTED TERMINAL REPEATS (ITRS). THE 3978 BASEPAIR MINI-DYSTROPHIN OPEN READING FRAME ENCODES THE N-TERMINUSHINGE REGIONS H1, H3 AND H4SPECTRIN-LIKE REPEATS R1, R2 AND R22-24AND THE C-TERMINAL CYSTEINE-RICH REGION OF THE DYSTROPHIN PROTEIN. in Duchenne Muscular Dystrophy. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/pf-06939926-is-a-recombinant-aav-vector-containing-the-aav-serotype-9-aav9-capsid-packaging-a-miniaturized-codon-optimized-version-of-the-dmd-genecalled-opti-dysdelta3978-which-encodes-the-domains-minimally-required-for-functionality-of-the-dystrophin-protein-mini-dystrophin-a-synthetic-polyadenylation-spolya-sequence-follows-the-opti-dysdelta3978-gene-cardiac-and-skeletal-muscle-specific-transgene-expression-is-mediated-by-a-synthetic-enhancer-and-promoter-the-expression-construct-is-flanked-by-the-aav2-inverted-terminal-repeats-itrs-the-3978-basepair-mini-dystrophin-open-reading-frame-encodes-the-n-terminushinge-regions-h1-h3-and-h4spectrin-like-repeats-r1-r2-and-r22-24and-the-c-terminal-cysteine-rich-region-of-the-dystrophin-protein-duchenne-muscular-dystrophy
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