LONDON, May 11, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”), a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases, today presents six posters detailing research data from its proprietary gene therapy platform at the American Society of Gene and Cell Therapy (“ASGCT”) Annual Meeting 2021, taking place May 11-14, 2021.
“These data presented today at ASGCT highlight the strong scientific foundation that underlies our gene therapy programs and broader technology platform,” said Theresa Heggie, Chief Executive Officer of Freeline. “Collectively, these posters demonstrate our potential to innovate across capsid, protein engineering, transduction efficiency, analytics and manufacturing, with the goal of delivering better gene therapy products for patients. These presentations also are a harbinger of things to come as our platform capabilities have the potential to unlock new diseases for which gene therapy could provide transformative benefits for patients.”
- Established a 96-well high-throughput suspension platform to facilitate both rAAV manufacturing platform optimization and candidate screening and selection to enable the continued growth of the Company’s gene therapy pipeline and manufacturing processes.
- Developed and completed scale up of suspension cell-based AAV manufacturing which reduces process variability, eliminates animal-derived components and reduces overall cost of goods, thereby maintaining the high quality and potency of vectors manufactured with the Freeline iCELLis® commercial scale process.
- Developed a dosing assay strategy for AAV-based gene therapies that enables the accurate and reproducible quantification of vector genome titer, which is required across the product lifecycle of AAV-based gene therapies. Utilization of the dosing assay is critical to ensure consistent potency of vector across in vitro and in vivo disease model studies and patient dosing.
- Developed a universally applicable transducing titer assay for AAV-based gene therapies that quantifies the percentage of liver cells functionally expressing a gene of interest at a given dose. This assay is complementary to traditional in vitro potency assays and provides an orthogonal tool to improve the understanding and quantification of functional AAV transduction for liver-directed AAV gene therapies.
- FLT190, the Company’s AAV gene therapy candidate for Fabry disease, demonstrated GLA uptake and metabolic cross correction in Fabry disease relevant cell lines, with a significant proportion of GLA enzyme effectively delivered to lysosomal compartments, where it exerted its degradative function as measured by Gb3 clearance in Fabry cells. These results are supportive of the Company’s ongoing development of FLT190, which is being evaluated in an ongoing Phase 1/2 clinical trial.
- FLT201, the Company’s AAV gene therapy candidate for Gaucher disease Type 1, administered as a single infusion in animal models, produced steady-state expression of a novel GCase variant (GCasevar85) in plasma to drive uptake of GCase in target cells and tissues. Importantly, restoration of tissue GCase activity achieved with FLT201 resulted in clearance of lipid storage cells and normalization of lyso-Gb1 biomarker levels in all tissues examined.
Development of a 96-Well Plate-Based High-Throughput System for rAAV Manufacturing Platform Optimization and Candidate Selection
Development and Scale Up of a Suspension Cell-Based AAV Manufacturing Process
Defining a Reliable Quantification Assay Strategy for Adeno-Associated Virus (AAV)-Based Gene Therapies
Development of an Assay to Measure Transduction Efficiency of Adeno-Associated Virus (AAV)-Based Gene Therapies
GLA Uptake and Metabolic Cross Correction in Fabry Disease Relevant Cell Lines: A Rationale for Liver-Directed AAV Gene Therapy
FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1
These poster presentations are available on the Investors section of the Freeline website.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (“AAV”) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, 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 protein into the patient’s bloodstream. The Company’s integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in Hemophilia B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.
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 the Company’s opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include, among other topics, discussion of the Company’s manufacturing, research, pipeline and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project” or “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, including the Company’s recurring losses from operations; the uncertainties inherent in research and development of the Company’s product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review; the Company’s ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt and delay the Company’s clinical trial pipeline; the Company’s failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Company’s ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Company’s product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Company’s ability to obtain and maintain regulatory approval of its product candidates; the Company’s limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Company’s ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We 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 our Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in our 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. You may get these documents by visiting EDGAR on the SEC website at www.sec.gov.
David S. Arrington
Vice President Investor Relations & Corporate Communications
+1 (646) 668 6947