Floatz Rating
CC47/100
Confidence
Indicative
Sponsor
Nationwide Children's Hospital
Modality
gene_therapy
Development Phase
Phase 1
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
32Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT01519349CT.gov Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Becker Muscular Dystrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Raav1.Cmv.Hufollistatin344 (this asset) | Nationwide Children's Hospital | P1 | CC · 47 |
| (-)-Epicatechin | Craig McDonald, MD | P2 | BB |
| Tadalafil | Cedars-Sinai Medical Center | P4 | BB |
| Carvedilol | Catholic University, Italy | P4 | BB |
| Ataluren | PTC Therapeutics | P3 | BB |
| Lisinopril | Cooperative International Neuromuscular Research Group | P2/3 | B |
| Corpril | Catholic University, Italy | P4 | B |
| coq10 | Cooperative International Neuromuscular Research Group | P2/3 | B |
| Sevasemten | Edgewise Therapeutics, Inc. | P2 | B |
+10 more in the Becker Muscular Dystrophy cohort
Other indications for Raav1.Cmv.Hufollistatin344
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Duchenne Muscular Dystrophy | — | P1/2 | CCC · 48 |
| Inclusion Body Myositis | — | P1 | CC · 47 |
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. Raav1.Cmv.Hufollistatin344 in Becker 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/raav1cmvhufollistatin344-becker-muscular-dystrophy
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