Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh Gene

Mucopolysaccharidosis Type IIIA

University of ManchesterunclassifiedPhase 1/2Active

CCC

48/ 100

Provisional rating · Methodology v0.2

Rating breakdown

4 Active assets in the Mucopolysaccharidosis Type IIIA cohort.

Asset / Sponsor	Phase	Rating
DNA (synthetic adeno-associated virus 9 vector scAAV9.U1a.hSGSH human N-Sulfoglucosamine sulfohydrolase-specifying) Ultragenyx Pharmaceutical Inc	Phase 2/3	B
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 2 VECTOR LYS-SAF302 HUMAN N-SULFOGLUCOSAMINE SULFOHYDROLASE CDNA-SPECIFYING) LYSOGENE	Phase 2/3	B
Saf-301 LYSOGENE	Phase 1/2	CCC
Autologous Cd34+ Cells Transduced With Tns9.3.55 University of Manchester	Phase 1/2	CCC
dnl126 Denali Therapeutics Inc.	Phase 1/2	CCC
Sobi003 Swedish Orphan Biovitrum	Phase 1/2	NR
gc1130a GC Biopharma Corp	Phase 1	NR

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