glycogen synthase inhibitors (glycogen storage diseases)

Glycogen Storage Disease 1b (Prime Medicine)

mini-GDE gene therapy (AAV9, glycogen storage disease type III)

G6PT mRNA replacement therapy (lipid nanoparticles, glycogen storage disease type 1b)

SLC37A4 gene therapy (lipid nanoparticle vector, glycogen storage disease 1b)

Lysosomal storage disease therapeutics (bioOasis)

Bovine type I collagen(Gintuit)

Recombinant Ebola Virus Disease Vaccine(Adenovirus Type 5 Vector)

Rx

Lysosomal storage disease 2(Belief Biomed)

Lysosomal Storage Disease Therapeutics(Orphazyme Aps)