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)

Charcot-Marie-Tooth Disease Type 1A(Metagenomi)

Lysosomal storage disease therapeutics (bioOasis)

Recombinant Ebola Virus Disease Vaccine(Adenovirus Type 5 Vector)

Rx

Beclin activator peripheral (Charcot Marie Tooth disease type 1A)

Lysosomal storage disease 2(Belief Biomed)