Freeline Raises $120 Million to Advance Gene Therapy Programs


Freeline, based in London, 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 round was led by Novo Holdings, Eventide Asset Management and Wellington Management Company. Cowen Healthcare Investments, Acorn Bioventures and Ample Plus Fund also participated.

In a separate announcement, the biotechnology company announced that it is considering additional capital fundraising options this year, with the possibility of an initial public offering (IPO) in the U.S. They have no timeline or any guarantees that they will actually raise more money, but discussions are underway.

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. It also plans to use the proceeds to continue developing its proprietary gene therapy platform using next-generation adeno-associated virus (AAV) technology and scale up its manufacturing capabilities.

“The potential of gene therapy to change patients’ lives has never been greater and we are delighted to have leading US and European biotechnology investors join us in this extended Series C financing,” said Theresa Heggie, chief executive officer of Freeline. “It comes at an exciting time for Freeline with our lead program in Hemophilia B progressing through clinical development, and with promising programs behind that, including a gene therapy treatment for Fabry Disease in the clinic and for Gaucher Disease and Hemophilia A in late preclinical development.”

In February, Freeline presented additional data from its Phase I/II B-AMAZE clinical trial of its FLT180a for Hemophilia B at the 13th Annual Congress of the European Association for Hemophilia and Allied Disorder (EAHAD), The Hague, Netherlands. The study had dosed two patients in four separate dose cohorts. The data suggested that the fourth dose cohort at 9.75e11 vg/kg had the potential to provide durable Factor IX (FIX) activity in the normal range.

FLT180a leverages the company’s proprietary AAVS3 capsid and a gain of function variant of human factor IX. Hemophilia B is an inherited, X-linked disease marked by spontaneous and prolonged bleeds in all tissues, including joints, muscle and the central nervous system. The disease is the result of mutations of the F9 gene that codes for FIX. FIX plays an essential role in normal blood coagulation.

Hemophilia can have lasting effects like chronic joint pain and disorders (arthropathy), life-threatening soft tissue bleeds, and hemorrhages of the CNS that can be fatal. Current treatments involve frequent intravenous infusions of FIX, although the risk of arthropathy continue and patients are at continued risk of serious, possibly fatal bleeds from trauma.

The gene therapy attempts to replace the mutated gene with a functional one.

In May, the U.S. Food and Drug Administration (FDA) granted the company’s FLT190 for Fabry Disease Orphan Drug Designation. The therapy has also received the Orphan Drug Designation from the European Commission.

Fabry Disease is an inherited, X-linked disease marked by the progressive accumulation of glycosphingolipids in lysosomes throughout the body. It is caused by mutations in the gene for alpha-galactosidase A, an enzyme responsible for the breakdown of globotriaosylceramide (Gb3). Progressive accumulation of Gb3 can cause organ damage, major disability and early death. Symptoms include neuropathic pain, impaired sweating gastrointestinal symptoms, renal failure, heart disease and increased risk of stroke.

FLT190 is a liver-directed AAV gene therapy.

“Freeline’s product candidates have significant potential to achieve functional cures for patients across a broad array of systemic diseases and we are very pleased with the progress the company has made to date,” said Chris Hollowood, chief investment officer of Syncona and chairman of the board of directors of Freeline. “We are focused on maximizing its ambition to develop gene therapy product candidates, and are pleased with the outcome of this funding round which brings in like-minded partners to support the company as it continues to scale and drive multiple programs through the clinic.”


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