We are currently advancing a diversified portfolio of investigational therapeutic programs organized around core areas of focus: hematology and immunology, liver, lung, ocular and neuromuscular. Across each core area, our initial focus is on genetic diseases with a fast, direct path to treating patients, and those with high unmet need not currently addressable using other gene editing approaches. Over time, we intend to maximize Prime Editing’s broad and versatile therapeutic potential to unlock opportunities beyond the genetic diseases in our initial pipeline, potentially including immunological diseases, cancers, infectious diseases, and targeting genetic risk factors in common diseases, which collectively impact millions of people. We have designed a modular platform within each core area which we believe can be leveraged to drive pipeline acceleration, efficiency and execution.
prime medicine pipeline
Modular
Platform
Indication
Delivery
Discovery
Lead
Optimization
IND-
Enabling
Phase 1/2
Hematology & Immunology
Chronic Granulomatous Disease
ex vivo
Other programs in discovery: Fanconi Anemia, Cell Shielding
Liver
Wilson’s Disease
LNP
Glycogen Storage Disease 1b
LNP
Undisclosed
LNP
Lung
Cystic Fibrosis*
LNP/AAV
Ocular
Retinitis Pigmentosa/ Rhodopsin
AAV
Other programs in discovery: Retinitis Pigmentosa/Usher Syndrome, Fuchs’ Endothelial Corneal Dystrophy
Neuro
Friedreich’s Ataxia
AAV
Other programs in discovery: Amyotrophic Lateral Sclerosis, Huntington's Disease, Fragile X Syndrome
Muscular
Myotonic Dystrophy Type 1
viral/
non-viral
Other programs in discovery: Oculopharyngeal Muscular Dystrophy, Duchenne Muscular Dystrophy
Additional Programs
Advancing as potential partnership opportunities
CAR-T (oncology/autoimmune)
ex vivo
Other programs in discovery: Usher Syndrome (Type 3) (ear); Non-Syndromic Hearing Loss – GJB2 (ear)
*Initial research funding provided by the Cystic Research Foundation
Pipeline reflects the current development stage and will be updated quarterly