ORGAN
PROGRAM & TARGET
STATUS
PREVALENCE
PYC-003
PKD1
Autosomal Dominant Polycystic Kidney Disease
Clinical trials (Phase 1b)
Discovery
Pre-clinical
IND-enabling
Clinical trials
Marketed
1 in 1,000
PYC-002
SHANK3
Phelan-McDermid Syndrome
IND-enabling
Discovery
Pre-clinical
IND-enabling
Clinical trials
Marketed
1 in 7,300
PYC-001
OPA1
Autosomal Dominant Optic Atrophy
Clinical trials (Phase 1/2)
Discovery
Pre-clinical
IND-enabling
Clinical trials
Marketed
1 in 35,000
VP-001*
PRPF31
Retinitis Pigmentosa type 11
Clinical trials (Phase 1/2)
Discovery
Pre-clinical
IND-enabling
Clinical trials
Marketed
1 in 100,000
Not disclosed
Haploinsufficiencies of major unmet need
Discovery programs
Discovery
Discovery
Pre-clinical
IND-enabling
Clinical trials
Marketed
*PYC owns 97.1% of VP-001, with the remaining 2.9% held by the Lions Eye Institute, Australia, and wholly owns all other pipeline programs