Floatz logo

Saf-301

Mucopolysaccharidosis Type III A

LYSOGENEgene_therapyPhase 1/2Dormant
CCC
55/ 100
Provisional rating · Methodology v0.2

Rating breakdown

Clinical Evidence
42/ 100
Competitive Position
95/ 100

Competitive context

5 Active assets in the Mucopolysaccharidosis Type III A cohort.

Asset / SponsorPhaseRating
DNA (synthetic adeno-associated virus 9 vector scAAV9.U1a.hSGSH human N-Sulfoglucosamine sulfohydrolase-specifying)
Ultragenyx Pharmaceutical Inc
Phase 2/3B
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 2 VECTOR LYS-SAF302 HUMAN N-SULFOGLUCOSAMINE SULFOHYDROLASE CDNA-SPECIFYING)
LYSOGENE
Phase 2/3B
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

Need depth?

Order a full diligence report on this asset.

Order report →

Are you the sponsor?

Submit your data room for a Verified Rating. The public-data rating remains visible alongside.

Contact →