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
Pfizergene_therapyPhase 3Active
CCC
51/ 100
Provisional rating · Methodology v0.2
Rating breakdown
- Clinical Evidence
- 36/ 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 |
Golodirsen Rare Disease Research, LLC | Phase 4 | BB |
Aoc 1044 Avidity Biosciences, Inc. | Phase 3 | BB |
Delandistrogene Moxeparvovec Sarepta Therapeutics, Inc. | Phase 3 | BB |
CHLOROPTIC-P S.O.P. COMPONENT PREDNISOLONE Anne M. Connolly | Phase 4 | BB |
View all 50 →
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →