We are currently progressing a diversified portfolio of 18 programs, initially focused on genetic diseases. The “immediate” category includes diseases with a fast, direct path to treating patients, and the “differentiation” category includes diseases with high unmet need not currently addressable using other gene-editing approaches. Over time, we intend to maximize Prime Editing’s broad therapeutic potential and go beyond the genetic diseases in our initial pipeline, potentially into cell therapies, oncology, immunology, infectious diseases, xenotransplantation, and targeting genetic risk factors in common diseases. Our initial 18 programs are outlined below.
prime medicine pipeline
target
tissue
indication
delivery
discovery
lead
optimization
ind-
enabling
phase 1/2
immediate
Blood
Chronic Granulomatous Disease
ex vivo
Fanconi Anemia
ex vivo
Liver
Wilson’s Disease
LNP
Glycogen Storage Disease 1b
LNP
EYE
Retinitis Pigmentosa/Rhodopsin
AAV
Retinitis Pigmentosa/Usher Syndrome
AAV
EAR
Usher Syndrome Type 3
AAV
Non-Syndromic Hearing Loss – GJB2
AAV
differentiation: repeat expansion diseases
NEURO- MUSCULAR
Friedreich’s Ataxia
AAV
Myotonic Dystrophy Type 1
viral/non-viral
Amyotrophic Lateral Sclerosis
viral/non-viral
Oculopharyngeal Muscular Dystrophy
LNP
Fragile X Syndrome
viral/non-viral
Huntington’s Disease
TBD
EYE
Fuchs’ Endothelial Corneal Dystrophy
viral/non-viral
differentiation: other
MUSCLE
Duchenne Muscular Dystrophy
AAV
LUNG
Cystic Fibrosis
LNP
partnered programs
BLOOD
Sickle Cell Disease 
ex vivo
*Pipeline reflects the current development stage and will be updated quarterly