FLT201: Initial Clinical Data
Lead clinical program with first- and best-in-class potential
with research extending innovation into larger disease area
Indication
Gaucher
Disease Type 1
FLT201
GBA1-linked
Parkinson's
Disease
Approximate
patient #
~18K
~190K
Research
Preclinical
Phase 1/2
Phase 3
Worldwide
rights
Wholly owned
$1B-plus peak
sales potential
Wholly owned
Estimated patient numbers for Gaucher disease Type 1 are for US, UK, EU4 and Israel (Hematology. 2017 Mar;22(2):65-73. doi: 10.1080/10245332.2016.1240391; this figure represents the total theoretical genetic
prevalence of the indication. The seroprevalence of antibodies against the AAVS3 capsid renders some patients ineligible for AAV gene therapy. We estimate approximately 60% would be eligible for AAVS3 gene therapy.
Company estimate of patient numbers for GBA1-linked PD are for US, UK and EU4.
FREELNE 4View entire presentation