Freeline pauses development of Fabry disease gene therapy FLT190

Move will free up funds to focus on developing Gaucher disease gene therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Freeline Therapeutics is pausing development of FLT190, an experimental gene therapy for Fabry disease.

The move will let the company free up funds to focus on advancing FLT201, a gene therapy being developed to treat another genetic disorder called Gaucher disease.

“While we remain encouraged by the data on FLT190 in Fabry disease, we have paused its development and are further streamlining the organization to extend our cash runway and focus on FLT201 in Gaucher disease,” Michael Parini, CEO of Freeline, said in a company press release. “I want to extend my sincere gratitude to all of our colleagues for their dedication and their contributions to Freeline, as well as to the investigators and patients who have participated in and supported the development of FLT190.”

Fabry disease is caused by mutations in the GLA gene, which provides instructions for making the enzyme alpha-galactosidase A (alpha-gal A) for breaking down certain fatty molecules, particularly globotriaosylceramide (Gb3) and lyso-Gb3.

In Fabry disease, the enzyme doesn’t function properly, so these fatty molecules build up to toxic levels in tissues, ultimately leading to disease symptoms.

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FLT190 is designed to deliver a healthy version of the gene encoding the alpha-gal A enzyme to cells in the liver, allowing a working version of the enzyme to be produced. The therapy is administered by a onetime infusion into the bloodstream and delivers its genetic payload using an engineered viral vector.

Experiments in preclinical models of Fabry disease showed FLT190 could increase alpha-gal A activity and reduce levels of Gb3 and lyso-Gb3.

Freeline has been conducting a Phase 1/2 clinical trial called MARVEL-1 (NCT04040049), with the aim of assessing the gene therapy’s safety in up to 15 adults with Fabry disease. Data from the first participants suggested a good safety and efficacy profile.

Along with pausing development of FLT190, Freeline announced it will be reducing its workforce by about 30%. This will facilitate a cash runway to continue developing FLT201, which is currently in clinical testing.

“FLT201 is our greatest potential value driver and our greatest opportunity to benefit patients,” Parini said.

Freeline also announced changes to its leadership. Paul Schneider, the company’s chief financial officer (CFO), will be joining the board of directors, while Amit Nathwani, MD, a professor at University College London who co-founded the company, will retire from the board, but continue to serve as a clinical and scientific adviser.