5 assets in Mucopolysaccharidosis Type 3A

Therapeutic assets addressing Mucopolysaccharidosis Type 3A, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Dna (Synthetic Adeno-Associated Virus 9 Vector Scaav9.U1A.Hsgsh Human N-Sulfoglucosamine Sulfohydrolase-Specifying) Mucopolysaccharidosis Type 3A	Ultragenyx Pharmaceutical Inc	P2/3	B (59)
Autologous Cd34+ Cells Transduced With A Lentiviral Vector Containing The Human Sgsh Gene Mucopolysaccharidosis Type 3A	University of Manchester	P1/2	CCC (48)
Autologous Cd34+ Cells Transduced With Tns9.3.55 Mucopolysaccharidosis Type 3A	University of Manchester	P1/2	CCC (48)
dnl126 Mucopolysaccharidosis Type 3A	Denali Therapeutics Inc.	P1/2	CCC (48)
gc1130a Mucopolysaccharidosis Type 3A	GC Biopharma Corp	P1	NR ()