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
REGENXBIO Inc.gene_therapyPhase 2/3Active
CCC
55/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 42/ 100
- Competitive Position
- 81/ 100
Competitive context
55 Active assets in the Duchenne Muscular Dystrophy cohort.
| Asset / Sponsor | Phase | Rating |
|---|---|---|
Viltolarsen NS Pharma, Inc. | Phase 4 | BBB |
Delandistrogene Moxeparvovec Sarepta Therapeutics, Inc. | Phase 3 | BB |
Aoc 1044 Avidity Biosciences, Inc. | Phase 3 | BB |
Golodirsen Rare Disease Research, LLC | Phase 4 | BB |
CHLOROPTIC-P S.O.P. COMPONENT PREDNISOLONE Anne M. Connolly | Phase 4 | BB |
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