Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease

LONDON, May 22, 2023 – Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that FLT201 has been awarded the Innovation Passport for the treatment of Gaucher disease type 1 under the Innovative Licensing and Access Pathway (ILAP) process by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). The Innovation Passport is the first step in the ILAP process, which in collaboration with MHRA and partner agencies, aims to accelerate time to market and facilitate patient access to medicines in the UK for life-threatening or seriously debilitating conditions, or conditions for which there is a significant patient or public health need.

“We believe FLT201 has life-changing potential for people with Gaucher disease type 1, the most common type of the disease,” said Pamela Foulds, MD, Chief Medical Officer of Freeline. “There remains a significant unmet need in Gaucher disease. Existing therapies come with a heavy life-long treatment burden, and even with treatment, many patients continue to experience serious symptoms. We believe that FLT201 may improve outcomes for patients with a one-time gene therapy. We look forward to working with MHRA to bring FLT201 to patients in the UK as efficiently as possible.”

The Innovation Passport is granted by the UK's ILAP Steering Group, which consists of representatives from MHRA, the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC), the All Wales Therapeutics and Toxicology Centre (AWTTC), and the National Health Service (NHS) England. Benefits of ILAP include opportunities for enhanced regulatory interactions through the development and approval process, as well as access to a range of development tools, such as the potential for a 150-day accelerated Marketing Authorization Application (MAA) assessment, rolling review and a continuous benefit-risk assessment.      

About Gaucher Disease

Gaucher disease is caused by a mutation in the GBA gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells that then accumulate in various organs, causing inflammation and dysfunction. Gaucher disease is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease type 1, the most common form of the disease, which affects the health of the spleen, liver, bone and lungs. Despite treatment with existing treatments, many people with Gaucher disease continue to experience symptoms and disease progression. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, European Union and Israel.

About FLT201

FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1. FLT201 is designed to generate durable increases in glucocerebrosidase (GCase) and reduce the accumulation of harmful substrates, with the aim of providing a one-time treatment that can stop disease progression, improve outcomes and free people from lifelong treatment. FLT201 uses Freeline’s proprietary AAVS3 capsid to introduce a novel transgene into liver cells to produce a rationally designed GCase variant. In preclinical studies, the GCase variant has demonstrated a 20-fold increase in half-life at lysosomal pH conditions compared to wild-type human GCase. Preclinically, FLT201 has shown robust GCase expression, as well as significant GCase uptake and substrate reduction in key tissues. FLT201 has been granted Orphan Drug Designation for the treatment of Gaucher disease by US and EU regulatory authorities.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company focused on developing transformative gene therapies for chronic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into a patient’s bloodstream. The company is currently advancing FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, in a Phase 1/2 clinical trial in people with Gaucher disease type 1. Freeline is headquartered in the UK and has operations in the United States. For more information, visit or connect with Freeline on LinkedIn and Twitter.

Forward-Looking Statements
This press release contains statements that constitute “forward looking statements” as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express opinions, expectations, beliefs, plans, objectives, assumptions or projections of Freeline Therapeutics Holdings plc (the “Company”) regarding future events or future results, in contrast with statements that reflect historical facts. These statements relate to future events, including the potential of FLT201 to be a first-in-class or life-changing gene therapy and improve outcomes for patients with a one-time gene therapy, and involve known and unknown risks, uncertainties and other factors that may cause the actual results, levels of activity, performance or achievements of the Company or its industry to be materially different from those expressed or implied by any forward-looking statements. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will,” “would,” “could” or “should,” the negative of these terms or similar expressions. Forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. The Company cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated, or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties, and other matters can be found in the Company’s Annual Report on Form 20-F for the fiscal year ended December 31, 2022, and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned “Cautionary Statement Regarding Forward-Looking Statements” and “Item 3.D. Risk factors.” Many of these risks are outside of the Company’s control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Company’s reports and documents filed with the U.S. Securities and Exchange Commission (the “SEC”). You may review these documents by visiting EDGAR on the SEC website at

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