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

Sanfilippo Syndrome

Ultragenyx Pharmaceutical Incgene_therapyPhase 2/3Active

59/ 100

Provisional rating · Methodology v0.2

Rating breakdown

2 Active assets in the Sanfilippo Syndrome cohort.

Asset / Sponsor	Phase	Rating
AMIDASE, SULFOGLUCOSAMINE SULF- Shire	Phase 2	B
Clofarabine Masonic Cancer Center, University of Minnesota	Phase 2	B
Evomela Masonic Cancer Center, University of Minnesota	Phase 2	B
Ambroxol Ozlem Goker-Alpan	Phase 2/3	B
IMMUNOGLOBULIN G1 (HUMAN-RAT MONOCLONAL CAMPATH-1H .GAMMA.1-CHAIN ANTI-HUMAN ANTIGEN CD52), DISULFIDE WITH HUMAN-RAT MONOCLONAL CAMPATH-1H LIGHT CHAIN, DIMER Masonic Cancer Center, University of Minnesota	Phase 2	B
Hydroxyurea Masonic Cancer Center, University of Minnesota	Phase 2	B
Hgt-1410 Shire	Phase 2	CCC
Duoc-01 Joanne Kurtzberg, MD	Phase 1	CC

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

mucopolysaccharidosis type 3ADormant

Indicative ·

59 / 100

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