PF-07209326 | New project
Sickle Cell Disease (Biologic) (ORPHAN - U.S.)
Anti-E-selectin inhibitor
PF-06755347 | New project
Chronic Inflammatory Demyelinating Polyneuropathy (ORPHAN-US); Primary Immune Thrombocytopenia (Biologic)
Immunomodulation
recifercept | New project
Achondroplasia (Biologic) (ORPHAN - U.S., EU)
Soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy
PF-06730512 | New project
Focal Segmental Glomerulosclerosis (FSGS) (Biologic)
Fusion protein containing SLIT ligand portion of ROBO2 receptor
GBT021601 | Project advanced
Sickle Cell Disease (ORPHAN - U.S.)
HbS polymerization inhibitor
PF-07265803 | New project
Dilated Cardiomyopathy due To Lamin A/C Gene Mutation (ORPHAN - U.S.)
p38 Mitogen-Activated Protein Kinase Antagonist
inclacumab | Project advanced
Sickle Cell Disease (Biologic) (ORPHAN - U.S.)
Anti-P-selectin inhibitor
marstacimab (PF-06741086) | New project
Hemophilia (Biologic) (FAST TRACK - U.S.; ORPHAN - U.S., E.U.)
Anti-tissue factor pathway inhibitor
fordadistrogene movaparvovec (PF-06939926) | New project
Duchenne Muscular Dystrophy Ambulatory (Biologic) (FAST TRACK - U.S.; ORPHAN - U.S., E.U.)
Gene therapy, minidystrophin
somatrogon (PF-06836922) | New project
Adult Growth Hormone Deficiency (Biologic) (ORPHAN - U.S., E.U.)
Human growth hormone agonist
giroctocogene fitelparvovec (PF-07055480) | New project
Hemophilia (Biologic) (RMAT, FAST TRACK, ORPHAN - U.S., E.U.)
Gene therapy, coagulation factor VIII (F8)
fidanacogene elaparvovec (PF-06838435) | New project
Hemophilia (Biologic) (BREAKTHROUGH, ORPHAN - U.S., E.U., PRIME - E.U.)
Gene therapy, coagulation factor IX (F9)
somatrogon (PF-06836922) | New project
Pediatric Growth Hormone Deficiency (Biologic) (ORPHAN - U.S.)
Human growth hormone agonist