2 assets in Lysosomal Storage Disease

Therapeutic assets addressing Lysosomal Storage Disease, rated under Floatz methodology v0.2 and sorted by current rating.

Asset	Sponsor	Phase	Rating
Dna (Recombinant Adeno-Associated Virus Vector Aav-Spark100-Gaa Human Liver-Specific Expression Cassette Including Human Lysosomal ?-Glucosidase Codon-Optimized Gene Gaa Plus Adeno-Associated Virus 2 Inverted Terminal Repeat Flanks) Lysosomal Storage Disease	Spark Therapeutics, Inc.	P1/2	CCC (48)
Lsd Lysosomal Storage Disease	University of Chicago	Early P1	C (39)