Pursuing Slow but Steady Gene Therapy Development


Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.

"It’s important to calibrate the benefit risk and make sure that we're being smart about developing these medicines for patients. That's something that I'm proud of Freeline for doing, where maybe we're not pushing the boundaries quite the same way that others are in terms of dose, but we're doing it very responsibly. This is going to be a long-term investment in moving the needle forward for patients and we're committed to making that investment and doing it the right way.”

Freeline Therapeutics is an adenovirus-associated vector (AAV) gene therapy company targeting hemophilia and rare diseases. The company has 3 programs in phase 1/2 trials: FLT180a in hemophilia B (B-LIEVE; NCT05164471), FLT190 in Fabry disease (MARVEL-1; NCT04040049), and FLT201 in Gaucher disease (GALILEO-1; NCT05324943).

The company announced in June 2022 that the second cohort had initiated dosing in the B-LIEVE trial for FLT180a based on positive data seen in cohort 1 and the advice of an Independent Data Monitoring Committee. Updated data from the trial is expected in the second half of 2022 and the first half of 2023. Freeline also plans to initiate phase 3 start-up activities in the first half of 2023.

CGTLive spoke with Michael Parini, JD, chief executive officer and director, Freeline Therapeutics, to learn more about the company, its pipeline, and proprietary platform. He discussed the progress of the different programs as well as the company’s focus on safety and slow but steady progress.

Freeline Initiates dosing of second cohort in B-LIEVE dose confirmation trial of FLT180a in hemophilia B. News release. Freeline Therapeutics. June 14, 2022. https://www.freeline.life/investors/newsroom/freeline-initiates-dosing-of-second-cohort-in-b-lieve-dose-confirmation-trial-of-flt180a-in-hemophilia-b/
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