FLT-200 / Spur Therapeutics - LARVOL DELTA

[VIRTUAL] FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1 (ASGCT 2021) - "GCasevar85 catalytic parameters (KM) are comparable to wild-type GCase and ERTs used to treat GD1.The potency and functionality of GCasevar85 when secreted by the liver following treatment with AAV8 pseudotyped FLT201 [AAV8-FLT201] were evaluated in GCase-deficient mice (Gba9v/null) and compared to liver-secreted wild-type GCase following treatment with AAV8 pseudotyped FLT200 [AAV8-FLT200] and velaglucerase alfa (an ERT used to treat GD). Based on the data collected in Gba9v/null mice and clinical data for our AAVS3 platform, the use of FLT201 compared to FLT200 should allow reduction of the predicted efficacious clinical dose by at least 10-fold.Pre-clinical data show that AAV8 pseudotyped FLT201 genome is well tolerated and results in a significant increase in potency in GCase deficient mice, including hard-to-reach tissues such as bone marrow and lung. Therefore, FLT201 has the potential to address areas of current unmet medical need in GD1 patients."

Gaucher Disease • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Type 1 Gaucher Disease

IPO Launch: Freeline Therapeutics Proposes $125 Million IPO (The Street) - "According to an amended registration statement, Freeline Therapeutics (FRLN) intends to raise $125 million from the selling of ADSs that represent underlying common stock at an IPO. The company is developing a pipeline of candidates for the treatment of hemophilia, Gaucher disease, and Fabry disease....FRLN seeks an above-average size of IPO transaction to advance its hemophilia, Gaucher disease and Fabry disease care pipeline."

Financing • Fabry Disease • Gaucher Disease • Genetic Disorders • Hemophilia