FLT-210 / Spur Therapeutics - LARVOL DELTA

Freeline Reports New Data from Fabry Disease Program, Pipeline and Company Updates, Including Appointment of Pamela Foulds, MD as Chief Medical Officer, and Third Quarter 2021 Financial Results (GlobeNewswire) - "The Company is engaging with regulatory authorities to update the study protocol for the Phase 1/2 B-LIEVE dose-confirmation trial of FLT180a. The protocol changes and subsequent review shifts guidance by approximately one quarter for the FLT180a program, with study start expected in the first quarter of 2022 instead of by year-end 2021. We are currently evaluating the timing of our Phase 3 pivotal trial and filing of a Biologics License Application and will provide more concrete guidance next year. The Company continues to anticipate interim data readouts in 2022....The Company expects to complete ongoing preclinical studies of FLT210 for the treatment of hemophilia A by year end...Based on data generated in these studies to date, the Company is evaluating whether additional studies will enhance an IND filing and the necessity of conducting these additional studies."

Clinical protocol • P1/2 data • Preclinical • Trial initiation date • Genetic Disorders • Hemophilia

Freeline Announces 2022 Corporate Priorities and Guidance (GlobeNewswire) - "Freeline Therapeutics Holdings plc...today announced its 2022 corporate priorities and provided updated guidance....Following this review, the Company has decided to halt further development of our preclinical work for FLT210 in hemophilia A, given the additional work needed. Freeline will seek to explore potential third-party partnership opportunities to progress this important work....Initiated the B-LIEVE study, a Phase 1/2 dose-confirmation study of FLT180a manufactured at commercial scale and using a starting dose of 7.7e11 vg/kg and a short course of prophylactic immune management, in Q4 2021....The Company expects to complete dosing of the first dose cohort in the B-LIEVE study and report data from the first dose cohort in the first half of 2022."

Discontinued • Trial status • Genetic Disorders • Hemophilia

[VIRTUAL] A secondgeneration twoplasmid packaging system for manufacturing of AAV vectors further improves quality and yield (ESGCT 2021) - "Advantages regarding quality and yield are particularly pronounced for larger AAV vectors such as FLT210, used in a haemophilia A gene therapy program with a vector genome size of 4.7kb. Applicability of the secondgeneration plasmid system was demonstrated in small and largescale formats (from dish to iCELLis bioreactor). In summary, the secondgeneration plasmid system enables the production of AAV vectors with a further improved quality and yield compared with the firstgeneration."

Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

[VIRTUAL] Protein Engineering in FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate (PEGS 2020) - No abstract available

Form 424B4 Freeline Therapeutics (Streetinsider.com) - "We currently have two product candidates in the clinic and expect to complete the Phase 1/2 B-AMAZE clinical trial of FLT180a for the treatment of hemophilia B by the end of 2020....Our other preclinical program, FLT210 for the treatment of hemophilia A, has achieved preliminary lead candidate selection....If the results of our planned pivotal clinical trial confirm the activity and safety profile observed to date, we intend to submit a biologics license application, or BLA, and a Marketing Authorization Application, or MAA, for approval of FLT180a for the treatment of hemophilia B in the second half of 2023....We expect...the commencement of our planned pivotal clinical trial in 2021, subject to agreement with regulatory authorities."

BLA • New trial • Preclinical • Trial completion date • Genetic Disorders • Hemophilia

Freeline Therapeutics and Checkmate Pharma Make Opening IPO Moves (Xconomy) - "The biotech IPO queue has added two more companies. Freeline Therapeutics and Checkmate Pharmaceuticals have each filed the regulatory paperwork to offer shares to the public....Freeline says in its prospectus that it expects to report additional data from the FLT180a study in the second half of this year. The company plans to advance the gene therapy to a pivotal study in 2021. The other programs in its pipeline include FLT190, a gene therapy for the rare enzyme deficiency Fabry disease that is in Phase 1/2 testing. Two other programs, FLT201 for Gaucher disease, another rare disorder caused by an enzyme deficiency, and FLT210 for hemophilia A, are in preclinical development."

Financing • Fabry Disease • Gaucher Disease • Genetic Disorders • Hemophilia • Type 1 Gaucher Disease

Freeline Raises $120 Million to Advance Gene Therapy Programs (Pharmalive) - "Freeline...announced the closing of a $120 million extended Series C financing. This was on top of Syncona’s previously announced Series C investment in Freeline of $40 million, with another $80 million in new capital....The company plans to use the monies raised to advance its lead program in Hemophilia B into a pivotal trial. It also will continue its Phase I/II clinical program for Fabry Disease and advance its pipeline for Gaucher Disease and Hemophilia A."

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