FRLN Freeline Therapeutics Holdings plc

14.91
-0.22  -1%
Previous Close 15.13
Open 15.09
52 Week Low 14.6194
52 Week High 21.69
Market Cap $518,043,283
Shares 34,744,687
Float 34,744,687
Enterprise Value $416,858,052
Volume 3,284
Av. Daily Volume 11,554
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Upcoming Catalysts

Drug Stage Catalyst Date
FLT180a
Hemophilia B
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
FLT201
Gaucher disease
Phase 1
Phase 1
Phase 1 trial to be initiated late-2021.
FLT190 (MARVEL-1)
Fabry disease
Phase 1/2
Phase 1/2
Phase 1/2 first patient dosed August 2019.

Latest News

  1. Michael J. Parini joins from Vertex as President and Chief Operating Officer complementing recent General Counsel and Investor Relations appointments

    Chief Financial Officer Brian M. Silver leaving Freeline to pursue a new opportunity

    LONDON, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced the expansion of its executive leadership team with the appointment of Michael J. Parini to the newly-created role of President and Chief Operating Officer. This appointment complements…

    Michael J. Parini joins from Vertex as President and Chief Operating Officer complementing recent General Counsel and Investor Relations appointments

    Chief Financial Officer Brian M. Silver leaving Freeline to pursue a new opportunity

    LONDON, Feb. 16, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced the expansion of its executive leadership team with the appointment of Michael J. Parini to the newly-created role of President and Chief Operating Officer. This appointment complements the recent hires of Stephen P. Diamond, Jr. as Senior Vice President and General Counsel and David S. Arrington as Vice President of Investor Relations and Corporate Communications.

    The Company also announced today that Brian M. Silver, Chief Financial Officer and Head of Corporate Development, has decided to leave Freeline to pursue a new opportunity. Silver will continue in his current role until April 30, 2021, to facilitate the smooth transition of his responsibilities. The Company has begun an external search for a Chief Financial Officer.

    Michael Parini has been appointed to the role of President and Chief Operating Officer, effective March 15, 2021. Parini will be based in the US and will report to Chief Executive Officer Theresa Heggie, supporting her in executing the Company's global strategy and overseeing critical business and operational functions. These functions include Program Management, Corporate Strategy, Business Development, Legal, Compliance, Investor Relations, Corporate Communications and Information Technology.

    "Michael will play a critical role in continuing to develop our infrastructure, operational excellence and strategic focus to drive robust delivery of our mission to bring functional cures to patients," said Heggie. "Together with other recent executive hires including Stephen and David, Michael will assist us in building the business capabilities for the next stages of growth including the development and commercialization of the Company's proprietary pipeline."

    Parini brings more than 20 years of executive experience in the biopharmaceutical industry, including most recently at Vertex Pharmaceuticals, where he served as Executive Vice President and Chief Administrative, Legal and Business Development Officer and as a member of the company's Executive Committee. He also played a major operational role in establishing new structures and functions while recruiting world-class talent as Vertex built out its global infrastructure. Parini helped secure access deals in major European markets and successfully co-led the acquisition of leading gene editing and cell therapy companies and technologies.   

    "It's an exciting time to join Freeline as the Company progresses its mission to bring first-in-class or best-in-class gene therapies to transform the lives of patients," said Parini. "Freeline's promising pipeline, which includes full global rights to four disclosed programs, its fully-integrated gene therapy platform, and its experienced leadership team will be the foundation of the Company's transition into a commercial organization."

    Silver stated, "I am proud to have been part of the leadership team that helped transition Freeline from a privately-held company to a publicly-listed biotech with the potential to grow into a global leader in systemic gene therapy. I am excited that senior leaders from some of the most important companies in our sector are joining Freeline to help us deliver on our vision."

    Heggie added, "I would like to thank Brian for his leadership, sound financial judgment and significant contributions to Freeline over the past two and a half years, including helping to raise more than $300 million through both private financings and the Nasdaq IPO last year, which have built a strong financial foundation for Freeline."

    About Michael J. Parini

    Michael Parini joins Freeline from Vertex Pharmaceuticals where he started in 2016 after more than a decade at Pfizer Inc. serving in multiple leadership roles within the company's global legal team, including Senior Vice President and Associate General Counsel. He was responsible for the strategic management of Pfizer's global litigation activities, including civil, intellectual property, government and employment litigation. He also served as Chief Counsel for multiple key business units within Pfizer, advising Pfizer's leadership team on business issues related to investment decisions, patent disputes, global pricing strategies and commercial operations activities, among other responsibilities. Prior to joining Pfizer, Parini served as a healthcare attorney at Akin, Gump, Strauss, Hauer & Feld, L.L.P. in Washington, D.C., where he provided legal counsel on federal and state regulatory and policy issues. He is a member of the New York and District of Columbia bars. He received a Bachelor of Sciences degree in American government from Georgetown University and a law degree from Georgetown University Law Center.

    About Recent Executive Hires

    Stephen Diamond recently joined Freeline as Senior Vice President and General Counsel and will report to Michael Parini. Diamond was most recently at Pfizer Inc., where he spent more than 10 years in legal roles of increasing responsibility, most recently as Assistant General Counsel, Business Transactions, in which he led legal support for a number of gene therapy projects. A corporate attorney, Diamond has more than 20 years of experience across a range of industries including life sciences and financial institutions. He is a member of the New York bar, and holds a Bachelor of Arts degree in International Relations from New York University and a Juris Doctor from William & Mary Law School.

    David Arrington recently joined Freeline as Vice President of Investor Relations and Corporate Communications and will report to Michael Parini. Prior to Freeline, Arrington was Vice President of Investor Relations and Corporate Affairs at Coherus BioSciences where he supported commercialization of the company's first product. He has more than 20 years of experience in life sciences including leadership roles at small- to mid- cap biotechnology companies, Stanford Medicine and UC California San Francisco, and 10 years at Genentech and Bristol Myers Squibb. Arrington holds a Bachelor of Arts degree from Washington State University.

    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. We use our 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. Freeline'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.

    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 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 discussion of the Company's manufacturing, research, pipeline, and clinical trial plans, as well as its management succession 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 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 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; the Company's ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates; as well as the ability of the Company to successfully recruit, hire and retain officers and other employees with required or desired skills, training and education. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. 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.

    Contacts

    David S. Arrington

    Vice President Investor Relations & Corporate Communications

    Freeline Therapeutics



    +1 (646) 668 6947

    Julia Wilson

    JW Communications



    +44 (0) 7818 430877



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  2. WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom…

    WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom are independent. Dr. Krop will serve as a member of the Board's Nominating and Corporate Governance Committee and Dr. Taglietti will serve as a member of the Board's Audit Committee.

    "We are delighted to welcome Julie and Marco as new independent directors to the Aquestive Board of Directors," said Santo Costa, Aquestive's Chairman of the Board. "Their significant expertise in the pharmaceutical and biotechnology industry and impressive backgrounds complement our Board of Directors' skills and experiences. We are confident they will provide valuable perspectives as we continue to execute our strategy and enhance value for the Company's shareholders."

    Dr. Krop stated, "I am thrilled to join the Board of Directors of Aquestive and look forward to working with the Board and the leadership team to advance the Company's mission of providing novel alternatives to invasively administered standard of care therapies. I am particularly excited about helping management guide Aquestive's proprietary orally administered epinephrine through clinical development. This novel formulation of epinephrine has the potential to eliminate the burden of intramuscular or subcutaneous injections for patients at risk of anaphylaxis due to severe allergies."

    Dr. Taglietti commented, "Aquestive is at an important stage of its evolution and I am delighted to join the Board of Directors during this exciting time. I look forward to contributing to Aquestive's continued growth and success as the Company advances its Libervant™ (diazepam) Buccal Film application through approval and executes on its innovative development activities with the potential to change patients' lives."

    "Julie and Marco join Aquestive at an exciting time as we continue to focus on developing and bringing to market valuable products in the CNS and allergy spaces. Julie and Marco's extensive collective experience in leading clinical development, regulatory strategies and commercialization of key value assets is a great addition and complement to the skills already present on our board," remarked Keith Kendall, Director, President and Chief Executive Officer of Aquestive. "We've experienced significant growth and strengthened our capabilities as a commercial pharmaceutical company under Doug's leadership as the original Chairman of the Board and then director. I would like to thank him for his contributions and expert guidance that have well positioned Aquestive for continued momentum and success."

    "On behalf of the Board of Directors and the Company, I would like to thank Doug for his more than 17 years of service to our Board, including as former Chairman, in guiding the Company from its start-up phase to a public commercial pharmaceutical company and leader in the film-based therapeutics industry," said Mr. Costa.

    "It has been a great privilege to serve as a director and former Chairman of Aquestive's Board of Directors, the members of which I hold in very high regard. I'd like to thank Keith, his team, and the Board for many exciting and satisfying years, and look forward to watching them accomplish even more great things for Aquestive in the months and years to come," stated Mr. Bratton.

    About Douglas K. Bratton

    Mr. Bratton has served as a member of the Company's Board of Directors since January 2004 and was the Chairman of the Board from January 2004 until August 2018. Mr. Bratton is the Founder, President and Chief Investment Officer of Crestline Investors, an institutional alternative investment management firm. He has been an investment professional specializing in alternative asset strategies since 1983 and has managed assets on behalf of the Bass family of Fort Worth, Texas since 1988.

    About Julie Krop, M.D.

    Dr. Krop is a seasoned biotech executive with more than two decades of experience successfully designing and executing clinical development programs from early stage development all the way through FDA approval. She has held senior leadership roles across clinical development, regulatory affairs, clinical operations, pharmacovigilance, medical affairs and program management during her career in the pharmaceutical and biotechnology industry. She currently serves as the Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN). Prior to assuming her position with Freeline Therapeutics, Dr. Krop was the Chief Medical Officer and Executive Vice President, Development, at AMAG Pharmaceuticals (NASDAQ:AMAG) from 2015 to 2020. From 2012 to 2015, Dr. Krop served as the Vice President, Clinical Development for Vertex Pharmaceuticals (NASDAQ:VRTX). In addition, Dr. Krop was Vice President, Clinical Development and Regulatory Affairs for Stryker Biotech (NYSE:SYK) from 2006 to 2012. Dr. Krop received a B.A. from Brown University and her M.D. from Brown University School of Medicine. She completed her fellowship in the Department of Endocrinology at the Johns Hopkins University School of Medicine where she was also a Robert Wood Johnson Foundation Clinical Scholar.

    About Marco Taglietti, M.D.

    Dr. Taglietti has more than three decades of experience in the pharmaceutical and biotechnology industry. He currently serves as a Director and President and Chief Executive Officer of SCYNEXIS Inc. (NASDAQ:SCYX). Prior to joining SCYNEXIS, Dr. Taglietti held various executive positions with Forest Laboratories (now AbbVie (NYSE: ABBV)) from 2007 until 2014, including President, Forest Research Institute, Chief Medical Officer and Executive Corporate Vice President, Research & Development. Dr. Taglietti was also the Senior Vice President, Head of Global Research and Development for Stiefel Laboratories, Inc. (now a GlaxoSmithKline company) from 2004 until 2007 and served in a number of executive positions from 1992 to 2004 with Schering-Plough Research Institute, including Vice President, Clinical Research Anti-Infectives, CNS, Dermatology and Endocrinology. From 1987 until 1992, Dr. Taglietti served in a number of executive positions with Marion Merrell Dow Research Institute, including as the European Product Team Leader – Anti-Infectives. Dr. Taglietti previously served on the boards of directors of Delcath (NASDAQ:DCTH) from 2014 to 2020 and NephroGenex (NASDAQ:NRX) from 2014 to 2017. Dr. Taglietti also served as a director of Stiefel International, Ltd., a private company, from 2004 to 2007 and a director of TransCelerate BioPharma, a non-profit pharma coalition dedicated to streamlining and accelerating the research and development of innovative new therapies, from 2013 to 2014. Since 2011, Dr. Taglietti has served on the board of directors of BioNJ, a life sciences trade association in New Jersey. In addition, Dr. Taglietti served on the board of directors of HINJ, Health Institute of New Jersey, a trade association for the leading research-based biopharmaceutical and medical technology companies in New Jersey, from 2011 to 2014, and is currently on the boards of directors of Orchestra of St. Luke, a New York City based orchestra, and American Foundation for Suicide Prevention, the largest non-profit organization dedicated to saving lives and bringing hope to those affected by suicide. Dr. Taglietti received his Degree in Medicine from the University of Pavia, Italy.

    About Aquestive Therapeutics

    Aquestive Therapeutics is a pharmaceutical company that applies innovative technology to solve therapeutic problems and improve medicines for patients. The Company has commercialized one internally-developed proprietary product to date, Sympazan® (clobazam) oral film, has a commercial proprietary product pipeline focused on the treatment of diseases of the central nervous system, or CNS, and other unmet needs, and is developing orally administered complex molecules to provide alternatives to invasively administered standard of care therapies. The Company also collaborates with other pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven capabilities for drug development and commercialization.

    Forward-Looking Statements

    Certain statements in this press release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believe," "anticipate," "plan," "expect," "estimate," "intend," "may," "will," or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement of Libervant and other product candidates through the regulatory and development pipeline; and business strategies, market opportunities, and other statements that are not historical facts. These forward-looking statements are subject to the uncertain impact of the COVID-19 global pandemic on our business including with respect to our clinical trials including site initiation, patient enrollment and timing and adequacy of clinical trials; on regulatory submissions and regulatory reviews and approvals of our product candidates; pharmaceutical ingredient and other raw materials supply chain, manufacture, and distribution; sale of and demand for our products; our liquidity and availability of capital resources; customer demand for our products and services; customers' ability to pay for goods and services; and ongoing availability of an appropriate labor force and skilled professionals. Given these uncertainties, the Company is unable to provide assurance that operations can be maintained as planned prior to the COVID-19 pandemic.

    These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans for AQST-108 and our other drug candidates; risk of delays in FDA approval of our drug candidate Libervant and AQST-108 and our other drug candidates or failure to receive approval; ability to address the concerns identified in the FDA's Complete Response Letter dated September 25, 2020 regarding the New Drug Application for Libervant; risk of our ability to demonstrate to the FDA "clinical superiority" within the meaning of the FDA regulations of Libervant relative to FDA-approved diazepam rectal gel and nasal spray products including by establishing a major contribution to patient care within the meaning of FDA regulations relative to the approved products as well as risks related to other potential pathways or positions which are or may in the future be advanced to the FDA to overcome the seven year orphan drug exclusivity granted by the FDA for the approved nasal spray product of a competitor in the U.S. and there can be no assurance that we will be successful; risk that a competitor obtains FDA orphan drug exclusivity for a product with the same active moiety as any of our other drug products for which we are seeking FDA approval and that such earlier approved competitor orphan drug blocks such other product candidates in the U.S. for seven years for the same indication; risk inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risks and uncertainties concerning the royalty and other revenue stream of the KYNMOBI™ monetization transaction, achievement of royalty targets worldwide or in any jurisdiction and certain other commercial targets required for contingent payments under the monetization transaction, and of sufficiency of net proceeds of the monetization transaction after satisfaction of and compliance with 12.5% Senior Notes obligations, as applicable, and for funding the Company's operations; risk of development of our sales and marketing capabilities; risk of legal costs associated with and the outcome of our patent litigation challenging third party at risk generic sale of our proprietary products; risk of sufficient capital and cash resources, including access to available debt and equity financing and revenues from operations, to satisfy all of our short-term and longer term cash requirements and other cash needs, at the times and in the amounts needed; risk of failure to satisfy all financial and other debt covenants and of any default; our and our competitors' orphan drug approval and resulting drug exclusivity for our products or products of our competitors; short-term and long-term liquidity and cash requirements, cash funding and cash burn; risk related to government claims against Indivior for which we license, manufacture and sell Suboxone® and which accounts for the substantial part of our current operating revenues; risk associated with Indivior's cessation of production of its authorized generic buprenorphine naloxone film product, including the impact from loss of orders for the authorized generic product and risk of eroding market share for Suboxone and risk of sunsetting product; risks related to the outsourcing of certain marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance of our product and product candidates; the success of any competing products, including generics; risk of the size and growth of our product markets; risks of compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to the Company's products; risk of unexpected patent developments; the impact of existing and future legislation and regulatory provisions on product exclusivity; legislation or regulatory actions affecting pharmaceutical product pricing, reimbursement or access; claims and risks that may arise regarding the safety or efficacy of the Company's products and product candidates; risk of loss of significant customers; risks related to legal proceedings, including patent infringement, investigative and antitrust litigation matters; changes in government laws and regulations; risk of product recalls and withdrawals; uncertainties related to general economic, political, business, industry, regulatory and market conditions and other unusual items; and other uncertainties affecting the Company described in the "Risk Factors" section and in other sections included in our Annual Report on Form 10 K, in our Quarterly Reports on Form 10-Q, and in our Current Reports on Form 8-K filed with the Securities Exchange Commission (SEC). Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. All subsequent forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements or outlook or guidance after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law.

    PharmFilm®, Sympazan® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.

    Investor inquiries:

    Westwicke, an ICR Company

    Stephanie Carrington



    646-277-1282

     



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  3. Data demonstrate potential of FLT201 to deliver sustained levels of β-glucocerebrosidase (GCase) variant 85, a proprietary engineered GCase that penetrates target tissues in Gaucher disease

    First-in-human dose finding studies of FLT201 on-track for initiation in late 2021

    LONDON, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that it will deliver one oral and three e-poster presentations at the 17TH Annual WORLDSymposium™ taking place virtually from February 8…

    Data demonstrate potential of FLT201 to deliver sustained levels of β-glucocerebrosidase (GCase) variant 85, a proprietary engineered GCase that penetrates target tissues in Gaucher disease

    First-in-human dose finding studies of FLT201 on-track for initiation in late 2021

    LONDON, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that it will deliver one oral and three e-poster presentations at the 17TH Annual WORLDSymposium™ taking place virtually from February 8 – 12, 2021, highlighting data from its gene therapy programs in Gaucher and Fabry diseases.

    "FLT201 data suggest that our gene therapy candidate for Gaucher disease is capable of delivering β-glucocerebrosidase variant 85 (GCasevar85) to tissues not sufficiently addressed by standard-of-care enzyme replacement therapy ("ERT")," said Theresa Heggie, Chief Executive Officer of Freeline. "In addition, we believe steady delivery of enzyme into target tissues to enable sustained clearance of pathologic substrate has the potential to offer significant improvements in clinical outcomes over existing standard of care."

    "We are excited by the transformational science that underlies the Freeline gene therapy platform," continued Ms. Heggie. "We look forward to progressing both the Gaucher and Fabry programs through the clinic with the planned initiation of our first-in-human study in Gaucher disease and dose escalation in Part 1 of the ongoing MARVEL-1 Phase 1/2 study in Fabry disease later this year."

    "FLT201 represents our innovative solution for the treatment of Type 1 Gaucher disease. This candidate leverages the Company's proprietary high-potency adeno-associated virus capsid, known as AAVS3. In addition, our protein engineering group developed GCasevar85, a proprietary GCase variant which, compared to wild-type GCase, has a greater than 20-fold increase in half-life in lysosomal pH and a 6-10 fold increase in half-life in serum, resulting in a 20-fold increase in potency of the vector. FLT201 is a combination of the clinically validated, potent AAVS3 capsid combined with a liver-specific promoter that drives the expression of GCasevar85" said Romuald Corbau, Ph.D., Chief Scientific Officer of Freeline. "These data demonstrate preclinical proof-of-concept for the potential of the program to provide functional cures in patients with the most common form of Gaucher disease, Type 1. Included in these data are demonstration of GCase expression, cellular uptake, tissue penetration, enzymatic activity, and clearance of disease causing substrate, glucosylsphingosine ("lyso-Gb1"). Considered in totality, these data suggest FLT201 may be able to deliver sustained GCase expression in difficult-to-reach tissues, such as bone marrow and lung, as evidenced by the substrate clearance."

    Presentation highlights from the platform presentation and e-poster titled, FLT201: An AAV-Mediated Gene Therapy for Type 1 Gaucher Disease Designed to Target Difficult to Reach Tissues, presented by Dr. Corbau, include:

    • In wild-type mice, FLT201 resulted in robust GCase expression in the liver and sustained GCase secretion into the plasma, with enhanced and sustained GCase uptake observed in key tissues involved in Gaucher disease including spleen, bone marrow and lung, as compared with velaglucerase alfa, a standard-of-care ERT for Gaucher disease. The data demonstrate that GCasevar85 secreted from the liver was taken up by macrophages in the spleen and trafficked to lysosomes.
    • In Gba-deficient mice, restoration of GCase activity after FLT201 injection was observed in difficult-to-reach tissues as shown by decreased levels of disease causing substrate, lyso-Gb1, increased concentrations of plasma GCase and reduced activated macrophages and inflammation in the lung. In addition, dose-dependent reductions of lyso-Gb1 were observed in all tissues analyzed including bone marrow and lung.
    • In vitro studies demonstrated that GCasevar 85 was taken up by human peripheral blood mononuclear cells ("PBMCs") and macrophages at levels comparable to those seen with ERT.
    • FLT201 treatment in rhesus macaques was well-tolerated with rapid and robust increases in plasma GCase levels.
    • Based on these results, Freeline believes that administration of FLT201 to patients with Gaucher disease can lead to continuous production of GCasevar85 in hepatocytes, in turn resulting in the steady presence of GCase in plasma, and improved penetration in Gaucher target tissues as compared with ERT, with potential improved outcomes in hard-to-reach tissues such as bone marrow and lung.
    • Freeline currently expects to initiate first-in-human dose finding studies of FLT201 in late 2021.

    Presentation highlights from an e-poster titled Generation of β-Glucocerebrosidase Variants with Increased Half-Life in Human Plasma for Liver Directed AAV Gene Therapy Aimed at the Treatment of Gaucher Disease Type 1, presented by Fabrizio Comper, Scientific Director, Freeline, include preclinical data on the generation and characterization of GCasevar85, which is part of the Company's development candidate for Gaucher disease, FLT201:

    • Human GCase enzyme is a protein known for its short half-life in human plasma. To provide an improved solution to Type 1 Gaucher patients, we addressed the short half-life of the GCase protein by designing, through our protein engineering efforts, 86 GCase variants that were engineered and assessed for stability and sustained activity at neutral and acidic pH.
    • Variant 85 showed the highest level of GCase activity when transduced using AAVS3 in Huh7 cells, with more than an 80-fold increase in activity over wild-type GCase, and was chosen as the lead development construct.
    • GCasevar85 showed increased stability in different physiological media compared with ERT, without differing in its fundamental enzymatic parameter KM.
    • The higher stability translated into a substantially higher level of GCase activity in plasma and target organs of mice transduced with a very low dose of AAV vector (6x1010 vg/kg), including in difficult to treat organs such as lung and bone marrow.

    Presentation highlights from an e-poster titled Development of a GLA NAb Assay with a Fully-Human, Neutralizing IgG4 Positive Control to Characterize Antibody Response in Fabry Disease Patients, presented by Sujata Ravi, Scientist, Freeline, include data on the development of a NAb assay for the characterization and monitoring of NAbs in patients with Fabry disease who are receiving ERT or gene therapy:

    • A custom in vitro phage display library against the enzyme α-galactosidase A (GLA) was used to screen and develop a unique GLA neutralizing IgG4 antibody, which is not described in the literature or commercially available.
    • A semi-quantitative GLA NAb assay was developed to determine the titer of GLA NAbs in Fabry patient serum, enabling further characterization and monitoring of Fabry patients receiving ERT or gene therapy.
    • Early development and qualification data suggest that the assay performance is acceptable and accurate in detecting NAbs in Fabry disease samples. These results support further development and validation as a robust, standardized assay for use in gene therapy trials, which may overcome the limitations associated with available assays that lack positive controls and show intra-lab variability.

    The poster presentations will be available on the events section of the Freeline website beginning at 4:01 pm EST on Monday, February 8, 2021. Registered WORLDSymposium™ attendees can access a recording of the oral presentation on Thursday, February 11, 2021.

    About Gaucher Disease

    Gaucher Disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages in certain organs due to the lack of functional GCase. The disorder is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease Type 1, the most common type, which impacts the health of the spleen, liver, blood system and bones. The current standard of care is intravenous infusion of ERT every two weeks, which is a significant treatment burden on the patient. The aim of Freeline's investigational gene therapy program is to deliver a one-time treatment of a long-lasting gene therapy that will provide a sustained, therapeutically relevant level of endogenous GCase, thus eliminating the need for ERT.

    About FLT201 for Gaucher Disease

    FLT201 is an investigational liver-directed AAV gene therapy in preclinical development for the treatment of Type 1 Gaucher disease. FLT201 contains a liver-specific promoter and a GBA1 sequence that expresses our novel, proprietary GCasevar85 variant, which has a 20-fold longer half-life at lysosomal pH conditions than wild-type GCase protein. Freeline's high-transducing AAVS3 capsid advances our goal to address unmet needs for those affected by Gaucher disease by enabling sustained, endogenous production of GCase following one-time intravenous infusion. To our knowledge, Freeline is the only company to date that has announced a program for the development of an AAV gene therapy for the treatment of Type 1 Gaucher disease and plans to file an IND for this program in 2021.

    About Fabry Disease

    Fabry disease is an inherited, X-linked disease characterized by the progressive accumulation of glycosphingolipids in lysosomes throughout the body. It is caused by mutations in the gene encoding of the α-galactosidase A (αGLA) enzyme responsible for the breakdown of globotriaosylceramide (Gb3), a type of glycosphingolipid.

    The condition ranges from mild to severe and may appear anytime from childhood to adulthood. The progressive accumulation of Gb3 leads to organ damage, major disability, and often early mortality. Symptoms and signs include neuropathic pain, impaired sweating, gastrointestinal symptoms, renal failure, heart disease and increased risk of stroke. Current treatment consists of ERT and chaperone therapy to temporarily clear Gb3 accumulation and alleviate symptoms.

    About FLT190 for Fabry Disease

    FLT190 is an investigational liver-directed AAV gene therapy for the treatment of Fabry disease. We believe the program is the first clinical-stage AAV gene therapy international study in Fabry disease. FLT190 is an in vivo gene therapy administered as a one-time intravenous infusion.

    The study, named MARVEL-1, is a multi-center, international, dose-finding Phase 1/2 study in adult males with classic Fabry disease. The study is focused on assessing the safety of FLT190 and its ability to transduce liver cells to produce continuous high levels of αGLA. In addition to safety, endpoints in the study include clearance of Gb3 and LysoGb3 from the plasma and urine, baseline renal and skin biopsies (repeated in long term follow up), renal and cardiac function, αGLA immune response, viral shedding and quality of life.

    About Freeline Therapeutics

    Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies. The Company is dedicated to the mission of transforming patient lives with the ambition of developing innovative products to mediate functional cure through a one-time treatment paradigm for inherited systemic debilitating diseases. Freeline leverages a proprietary, rationally-designed capsid and AAV vector technology cassette that delivers a functional copy of a therapeutic gene into the human liver, delivering a durable level of missing proteins into the patient's bloodstream. Freeline's integrated expression platform includes capabilities in research, manufacturing, clinical development, 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.

    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 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 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 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 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. 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.

    Contacts

    David S. Arrington

    Vice President Investor Relations & Corporate Communications

    Freeline Therapeutics



    +1 (646) 668 6947

    Julia Wilson

    JW Communications



    +44 (0) 7818 430877



    Primary Logo

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  4. Targeting initiation of dose confirmation trial by end of 2021 with data readout by end of 2022

    Initiation of Phase 3 pivotal trial targeted by mid-2023, with data readout and BLA filing targeted by end of 2024

    LONDON, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline") a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced a modification to the clinical development plan for its FLT180a program for Hemophilia B.

    The modification to the FLT180a clinical development plan is intended to address feedback Freeline received from…

    Targeting initiation of dose confirmation trial by end of 2021 with data readout by end of 2022

    Initiation of Phase 3 pivotal trial targeted by mid-2023, with data readout and BLA filing targeted by end of 2024

    LONDON, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline") a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced a modification to the clinical development plan for its FLT180a program for Hemophilia B.

    The modification to the FLT180a clinical development plan is intended to address feedback Freeline received from the U.S. Food and Drug Administration ("FDA") relating to the characterization and comparability of the investigational drug product used in its Phase 1/2 B-AMAZE trial that was produced at smaller scale, as compared to its current investigational drug product that is produced at commercial scale.

    Under the modified clinical development plan, Freeline now plans to conduct dose confirmation in a FLT180a Phase 1/2 trial instead of in the Phase 2b part of the previously planned Phase 2b/3 pivotal trial. Freeline believes this modified clinical development plan should enable it to meet its objectives of initiating the clinical trial sites for the dose confirmation trial by the end of 2021, while in parallel, working to meet FDA's Chemistry, Manufacturing and Control ("CMC") requirements in advance of commencing the planned Phase 3 pivotal trial. The Company is targeting a data readout for the Phase 1/2 dose confirmation trial by the end of 2022, initiation of the Phase 3 pivotal trial by the middle of 2023 and filing of a Biologic License Application ("BLA") with the FDA by the end of 2024.

    The Company expects that the Phase 1/2 dose confirmation trial will be a six-month single dose safety and efficacy trial designed to confirm the dose and immune management regimen for the planned Phase 3 pivotal trial, with costs similar to those of the dose confirmation part of the previously planned Phase 2b/3 pivotal trial. Informed by the results of the dose-ranging B-AMAZE trial, which demonstrated a dose response across four cohorts, the Company believes that a dose of FLT180a between 7.5e11 and 9.75e11 vg/kg1 is likely to bring patients into the normal range of Factor IX ("FIX") activity level of 50-150%.

    The Company anticipates that the design of the Phase 3 pivotal trial will be nearly identical to the Phase 3 part of the previously planned Phase 2b/3 pivotal trial. Assuming the Phase 3 pivotal trial demonstrates robust data at 26 weeks, the Company believes it would be able to file a BLA with the FDA by the end of 2024 utilizing the Accelerated Approval pathway. The Company anticipates that the BLA filing would be based on the surrogate endpoint of FIX activity in approximately 20 patients, combined with demonstration of a positive correlation between 26-week FIX activity levels and 52-week annualized bleeding rates ("ABR") in a subset of these 20 patients. The Phase 3 pivotal trial would continue to enroll up to an additional 30 patients to evaluate 52-week ABR to support full regulatory approval.

    "We believe conducting dose confirmation in a Phase 1/2 trial should allow us to achieve similar objectives as our previously planned trial, whilst also enabling us to work with FDA on CMC prior to initiating the Phase 3 pivotal trial. We are still targeting the filing of our FLT180a BLA in 2024 and are committed to bringing a functional cure to Hemophilia B patients as soon as possible," said Theresa Heggie, Chief Executive Officer of Freeline. "To streamline the development path for our subsequent programs, we have developed our entire pipeline from Phase 1 onward using the same commercial-scale manufacturing platform that we are now using for FLT180a."

    ________________

    1 The Company's B-AMAZE trial dose escalated across four dose levels. For future data updates, Freeline expects results will be reported using a new dose level nomenclature in the assay for the commercial-scale process in which the former doses of 4.5e11, 7.5e11, 9.75e11, and 1.5e12 vg/kg correspond to 3.8e11, 6.4e11, 8.32e11, and 1.28e12 vg/kg, respectively.

    About Freeline Therapeutics

    Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus ("AAV") vector-mediated gene therapies. The Company is dedicated to the mission of transforming patient lives with the ambition of developing innovative products to mediate functional cure through a one-time treatment paradigm for inherited systemic debilitating diseases. Freeline leverages a proprietary, rationally-designed capsid and AAV vector technology cassette that delivers a functional copy of a therapeutic gene into the human liver, delivering a durable level of missing proteins into the patient's bloodstream. Freeline's integrated expression platform includes capabilities in research, manufacturing, clinical development, 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.

    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 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 statements that the Phase 1/2 dose confirmation trial for FLT180a is expected to be initiated by the end of 2021 and provide a data readout in 2022, that the Phase 3 pivotal trial is expected to be initiated by the middle of 2023, that a BLA filing for FLT180a is expected to be made by the end of 2024, and that the Company's revised clinical development plan for FLT180a will allow it to address CMC feedback from the FDA in parallel with the Phase 1/2 dose confirmation trial, that the costs of the Phase 1/2 dose confirmation trial will be similar to the dose confirmation part of the prior Phase 2b/3 pivotal trial, as well as any other discussion of the Company's strategies, financing plans, 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 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 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. 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. In particular, no assurance can be provided as to whether the Company will be able to meet the milestones in its revised clinical development plan for FLT180a, whether the Company will be able to adequately address the FDA's feedback relating to the CMC that is required to initiate a pivotal trial, whether the FDA will provide any feedback on the revised clinical development plan that impacts the timing or design of its clinical trial plans for FLT180a or any of its other product candidates, whether the Company's revised clinical development plan will impact trial costs or the Company's cash burn rate or whether there will be any impact to the Company's regulatory submissions timeline for FLT180a or any of its other product candidates as a result of the CMC or any other feedback from the FDA or otherwise. 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.

    Contacts

    David S. Arrington

    Vice President Investor Relations & Corporate Communications

    Freeline Therapeutics



    +1 (646) 668 6947

    Julia Wilson

    JW Communications



    +44 (0) 7818 430877



    Primary Logo

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  5. LONDON, Jan. 26, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases, today announced that it will present two e-posters at the 2021 Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) taking place 3-5 February 2021. In addition, the Company will present data on its gene therapy programmes for Gaucher and Fabry Disease, in one oral platform presentation and three e-posters at the 17TH Annual WORLDSymposium™ taking place 8-12 February 2021.

    EAHAD Presentation

    LONDON, Jan. 26, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases, today announced that it will present two e-posters at the 2021 Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) taking place 3-5 February 2021. In addition, the Company will present data on its gene therapy programmes for Gaucher and Fabry Disease, in one oral platform presentation and three e-posters at the 17TH Annual WORLDSymposium™ taking place 8-12 February 2021.

    EAHAD Presentation Details

    Title:  Follow-up on a Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)
    Presenter: Pratima Chowdary, Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, University College London
    Presentation #:  ABS-114
    Date and Time: Tuesday, 2 February 2021, 6:00 CET



    Title: Insight into the Persistent Clinical Burden Underlying Problem Joints, Pain, and Bleeding in Adults from Across Europe with Haemophilia A and B: The CHESS II Study
    Presenter:  Sharmila Kar, Head of Real-World Evidence, Freeline
    Presentation #:  ABS-134
    Date:   Tuesday, 2 February 2021, 6:00 CET
       
    The e-poster presentations will be available from Tuesday, 2 February 2021, as recordings on the events section of the Freeline website.



    WORLDSymposium™ Presentation Details

    Platform presentation:

    Title: FLT201: An AAV-Mediated Gene Therapy for Type 1 Gaucher Disease Designed to Target Difficult to Reach Tissues
    Author:  Romuald Corbau, Chief Scientific Officer, Freeline
    Date and Time:   Thursday, 11 February 2021, 11:36am EST

    Poster presentations:

    Title: FLT201: An AAV-Mediated Gene Therapy for Type 1 Gaucher Disease Designed to Target Difficult to Reach Tissues
    Author:  Romuald Corbau, Chief Scientific Officer, Freeline
    Presentation #:  44
    Date:   Thursday, 11 February 2021



    Title: Generation of β-Glucocerebrosidase Variants with Increased Half-Life in Human Plasma for Liver Directed AAV Gene Therapy Aimed at the Treatment of Type 1 Gaucher Disease
    Author: Fabrizio Comper, Scientific Director, Freeline
    Presentation #:       41
    Date: Thursday, 11 February 2021



    Title:  Development of a GLA nAb Assay with a Fully-Human, Neutralizing IgG4 Positive Control to Characterize Antibody Response in Fabry Disease Patients
    Author:  Sujata Ravi, Scientist, Freeline
    Presentation #:    211
    Date: Thursday, 11 February 2021

    The poster presentations will be available on the events section of the Freeline website from 2:30 pm EST on Monday, 8 February 2021. A recording of the platform presentation with a live Q&A will be available after the presentation concludes on Thursday, 11 February 2021, also on the events section of the Freeline website.

    About Freeline

    Freeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the UK and has operations in Germany and the US.

    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 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 discussion of the Company's strategies, financing plans, 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 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; 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 U.S., U.K. or EU, including the COVID-19 pandemic, to disrupt 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 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. 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.

    Further Information

    David S. Arrington

    VP Investor Relations & Corporate Communications

    +1 (646) 668 6947

    United States

    LifeSci Advisors

    Dan Ferry

    +1 (617) 430 7576

    Europe

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877



    Primary Logo

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