Recombinant, Non-Replicating Adeno-Associated Virus Serotype 8 (Raav8) Vector, Encoding A Codon-Optimised Miniaturised Version Of Human Dystrophin (Microdystrophin Variant Abd1-H1-R1-R2-R3-H3-R24-H4-Cr-Ct194), Under Control Of A Synthetic Muscle-Specific Promoter (Spc5-12) And A Small Synthetic Polyadenylation Signal; Flanked By Aav2 Inverted Terminal Repeats. The Expressed Microdystrophin Variant Contains The Actin-Binding Domain 1 (Abd1), Four Rod Domains (R1-3, R24), Three Hinge Domains (H1, H3, H4) And A Cysteine Rich (Cr) Domain; It Also Features The Proximal Portion Of The C-Terminal Domain, Amino Acids 3361-3554 (Ct194)
Duchenne Muscular Dystrophy
Explore 56 assets in Duchenne Muscular Dystrophy →Floatz Rating
CCC55/100
Confidence
Indicative
Sponsor
REGENXBIO Inc.
Modality
gene_therapy
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
42Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05693142CT.gov AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
81Moderate confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Duchenne Muscular Dystrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Recombinant, Non-Replicating Adeno-Associated Virus Serotype 8 (Raav8) Vector, Encoding A Codon-Optimised Miniaturised Version Of Human Dystrophin (Microdystrophin Variant Abd1-H1-R1-R2-R3-H3-R24-H4-Cr-Ct194), Under Control Of A Synthetic Muscle-Specific Promoter (Spc5-12) And A Small Synthetic Polyadenylation Signal; Flanked By Aav2 Inverted Terminal Repeats. The Expressed Microdystrophin Variant Contains The Actin-Binding Domain 1 (Abd1), Four Rod Domains (R1-3, R24), Three Hinge Domains (H1, H3, H4) And A Cysteine Rich (Cr) Domain; It Also Features The Proximal Portion Of The C-Terminal Domain, Amino Acids 3361-3554 (Ct194) (this asset) | REGENXBIO Inc. | P2/3 | CCC · 55 |
| Viltolarsen | NS Pharma, Inc. | P4 | BBB |
| Golodirsen | Rare Disease Research, LLC | P4 | BB |
| Delandistrogene Moxeparvovec | Sarepta Therapeutics, Inc. | P3 | BB |
| Aoc 1044 | Avidity Biosciences, Inc. | P3 | BB |
| Prednisolone | Anne M. Connolly | P4 | BB |
| Glutamine | Assistance Publique - Hôpitaux de Paris | P3 | BB |
| Eteplirsen | Sarepta Therapeutics, Inc. | P3 | 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. Recombinant, Non-Replicating Adeno-Associated Virus Serotype 8 (Raav8) Vector, Encoding A Codon-Optimised Miniaturised Version Of Human Dystrophin (Microdystrophin Variant Abd1-H1-R1-R2-R3-H3-R24-H4-Cr-Ct194), Under Control Of A Synthetic Muscle-Specific Promoter (Spc5-12) And A Small Synthetic Polyadenylation Signal; Flanked By Aav2 Inverted Terminal Repeats. The Expressed Microdystrophin Variant Contains The Actin-Binding Domain 1 (Abd1), Four Rod Domains (R1-3, R24), Three Hinge Domains (H1, H3, H4) And A Cysteine Rich (Cr) Domain; It Also Features The Proximal Portion Of The C-Terminal Domain, Amino Acids 3361-3554 (Ct194) 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/recombinant-non-replicating-adeno-associated-virus-serotype-8-raav8-vector-encoding-a-codon-optimised-miniaturised-version-of-human-dystrophin-microdystrophin-variant-abd1-h1-r1-r2-r3-h3-r24-h4-cr-ct194-under-control-of-a-synthetic-muscle-specific-promoter-spc5-12-and-a-small-synthetic-polyadenylation-signal-flanked-by-aav2-inverted-terminal-repeats-the-expressed-microdystrophin-variant-contains-the-actin-binding-domain-1-abd1-four-rod-domains-r1-3-r24-three-hinge-domains-h1-h3-h4-and-a-cysteine-rich-cr-domain-it-also-features-the-proximal-portion-of-the-c-terminal-domain-amino-acids-3361-3554-ct194-duchenne-muscular-dystrophy
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