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DNA (synthetic adeno-associated virus 9 vector scAAV9.U1a.hSGSH human N-Sulfoglucosamine sulfohydrolase-specifying)

MPS IIIA

Ultragenyx Pharmaceutical Incgene_therapyPhase 2/3Active
B
59/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
44/ 100
Competitive Position
95/ 100

Competitive context

4 Active assets in the MPS IIIA cohort.

Asset / SponsorPhaseRating
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 2 VECTOR LYS-SAF302 HUMAN N-SULFOGLUCOSAMINE SULFOHYDROLASE CDNA-SPECIFYING)
LYSOGENE
Phase 2/3B
Saf-301
LYSOGENE
Phase 1/2CCC
Autologous Cd34+ Cells Transduced With Tns9.3.55
University of Manchester
Phase 1/2CCC
dnl126
Denali Therapeutics Inc.
Phase 1/2CCC
Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh Gene
University of Manchester
Phase 1/2CCC
Sobi003
Swedish Orphan Biovitrum
Phase 1/2NR
gc1130a
GC Biopharma Corp
Phase 1NR

Same molecule, other indications

DNA (synthetic adeno-associated virus 9 vector scAAV9.U1a.hSGSH human N-Sulfoglucosamine sulfohydrolase-specifying) is also rated in:

mucopolysaccharidosis type 3Dormant
B
Indicative ·
59 / 100
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