FRLN Freeline Therapeutics Holdings plc

15.86
-0.1  -1%
Previous Close 15.96
Open 15.9
52 Week Low 14.6194
52 Week High 21.69
Market Cap $551,050,736
Shares 34,744,687
Float 34,744,687
Enterprise Value $435,908,564
Volume 9,437
Av. Daily Volume 38,710
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Drug Pipeline

Drug Stage Notes
FLT190 (MARVEL-1)
Fabry disease
Phase 1/2
Phase 1/2
Phase 1/2 first patient dosed August 2019.
FLT180a
Hemophilia B
Phase 1/2
Phase 1/2
Phase 1/2 trial ongoing. Pivotal trial to be initiated in 2021.

Latest News

  1. LONDON, Nov. 19, 2020 (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 management will participate in a fireside chat at the 3rd Annual Evercore ISI Health CONx Conference on 3 December, 2020 from 08.00 – 8.20 am ET.

    A live audio webcast of the fireside chat will be available on the events section of Freeline's website.  An archived replay will be available on the Company's website for a period of 90 days after the conference.

    About Freeline
    Freeline is a clinical-stage…

    LONDON, Nov. 19, 2020 (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 management will participate in a fireside chat at the 3rd Annual Evercore ISI Health CONx Conference on 3 December, 2020 from 08.00 – 8.20 am ET.

    A live audio webcast of the fireside chat will be available on the events section of Freeline's website.  An archived replay will be available on the Company's website for a period of 90 days after the conference.

    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, 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:

    United States

    LifeSci Advisors

    Dan Ferry

    +1 (617) 430 7576

    Europe

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877

     



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  2. LONDON, Nov. 02, 2020 (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, announced that Mark Baldry, MBA, has today been appointed as the Company's new Chief Commercial Officer, effective from today's date.

    Mark is an experienced commercial leader with a 30-year track record in positions of increasing responsibility at leading biopharmaceutical companies in marketing, sales and public affairs roles across Europe, Canada and the US. He has predominately worked across rare and specialty disease…

    LONDON, Nov. 02, 2020 (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, announced that Mark Baldry, MBA, has today been appointed as the Company's new Chief Commercial Officer, effective from today's date.

    Mark is an experienced commercial leader with a 30-year track record in positions of increasing responsibility at leading biopharmaceutical companies in marketing, sales and public affairs roles across Europe, Canada and the US. He has predominately worked across rare and specialty disease areas, where he has championed multiple commercial launches.

    "Mark's appointment as Chief Commercial Officer reflects his strong background in the commercialisation of products for rare diseases including in Fabry and Gaucher disease," said Theresa Heggie, Chief Executive Officer of Freeline. "This has been a transformative year for Freeline with encouraging clinical progress across our programmes and a strengthened management team to which Mark is a great addition. The potential of gene therapy to change patients' lives has never been greater, and I look forward to working with Mark as we continue to develop our pipeline of innovative product candidates through to commercialisation to meet the needs of patients."

    Mark joins Freeline from Wave Life Sciences where he served as Chief Commercial Officer and prior to that as Senior Vice President, Global Marketing & Commercial Operations at Amicus Therapeutics. In these roles, he was responsible for building global commercial strategies to support launches of innovative medicines in orphan diseases, including the first oral chaperone therapy for the treatment of Fabry disease.

    Prior to that, Mark held multiple leadership positions at Biogen, including Vice President, Public Affairs and Vice President, New Product Commercialisation. He also held leadership roles at the Human Genetic Therapies division of Shire including Head of Global Strategic Marketing and Head of Marketing, Market Access and Public Affairs, Europe, Middle East and Africa.

    Mark earned an MBA from Concordia University (Canada) and holds a BSc in Genetics from York University (UK).

    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, 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 an 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 programmes 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

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877

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  3. - Updated data on analytical technologies for determining the proportion of AAV capsids containing full-length vector genomes

    - Potentially increasing safety and efficacy in Freeline's AAV delivery model

    LONDON, Oct. 30, 2020 (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 new data on analytical technologies for determining the proportion of Adeno-associated viruses (AAV) capsids containing full-length vector genomes will be presented at the 12th Annual Protein and Antibody…

    - Updated data on analytical technologies for determining the proportion of AAV capsids containing full-length vector genomes

    - Potentially increasing safety and efficacy in Freeline's AAV delivery model

    LONDON, Oct. 30, 2020 (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 new data on analytical technologies for determining the proportion of Adeno-associated viruses (AAV) capsids containing full-length vector genomes will be presented at the 12th Annual Protein and Antibody Engineering Summit (PEGS), 9 – 13 November 2020.

    Freeline will present updated data on the use of analytical technologies for determining the proportion of AAV capsids containing full-length vector genomes, including the use of a panel of assays and combination of methodologies to fully characterise the AAV product. The ‘full-to-empty' ratio discussed is an important quality attribute for AAVs with potential impact on safety and efficacy.

    "We are pleased to report additional data on analytical technologies," said Theresa Heggie, CEO of Freeline Therapeutics. "This announcement comes in the final quarter of a pivotal year for Freeline, with a strengthened balance sheet following the successful launch of our IPO, continued progress in our clinical programmes in Haemophilia B and Fabry Disease, as well as advancements in our preclinical programmes in Gaucher Disease and Haemophilia A. It has always been our ambition to produce the best-in-class capsid, and these technologies will allow us to potentially increase safety and efficacy across our platform which will benefit all of our programmes."

    Title: Analytical Technologies for Determining the Proportion of AAV Capsids Containing Full-Length Vector Genomes

    Presenter: Sonya Schermann, Director of Analytical Development at Freeline

    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, 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 an 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 programmes 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

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877

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  4. Will report updated durability data from Phase 1/2 B-AMAZE trial in Haemophilia B in Q4

    Third wholly-owned programme expected to be in the clinic in 2021

    Strong cash position with over $250 million at 30 September

    LONDON, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage, fully integrated, next generation, systemic, liver directed, AAV-based gene therapy company today announced its financial results for the first half of 2020 and provided a business update.

    "2020 has been a transformative year for Freeline, marked by encouraging clinical progress in our Haemophilia B and Fabry programmes, a strengthened management team and the successful completion of our…

    Will report updated durability data from Phase 1/2 B-AMAZE trial in Haemophilia B in Q4

    Third wholly-owned programme expected to be in the clinic in 2021

    Strong cash position with over $250 million at 30 September

    LONDON, Oct. 29, 2020 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage, fully integrated, next generation, systemic, liver directed, AAV-based gene therapy company today announced its financial results for the first half of 2020 and provided a business update.

    "2020 has been a transformative year for Freeline, marked by encouraging clinical progress in our Haemophilia B and Fabry programmes, a strengthened management team and the successful completion of our Series C round and Nasdaq initial public offering," said Theresa Heggie, Chief Executive Officer of Freeline Therapeutics. "With a strengthened balance sheet, we are now well positioned to achieve upcoming clinical milestones, which include the presentation of updated data from our B-AMAZE trial for Haemophilia B by the end of this year evaluating FLT180a, the only gene therapy candidate showing durable Factor 9 (FIX) activity in the normal range. In 2021 we expect to continue our progress in the clinic with the initiation of a pivotal trial in Haemophilia B, continued progress in our MARVEL1 study in Fabry disease, and the initiation of our Phase 1/2 trial for Gaucher disease. On the preclinical front, we will continue to advance our potentially best-in-class Haemophilia A programme. Importantly, all these programmes are wholly owned and leverage Freeline's proprietary AAVS3 capsid which has been engineered for high potency to achieve durable, functional cures by achieving normal levels of protein expression at doses lower than those seen in wild-type AAV constructs."

    Business Highlights and Upcoming Milestones

    Haemophilia B

    • Completed dosing and reported initial data from the Phase 1/2 adaptive dose finding B-AMAZE trial to assess the safety and efficacy of systemic administration of FLT180a in adult patients with severe Haemophilia B
      • Dosed 10 patients across four dose cohorts, with all patients having completed 6-month follow-up and now enrolled in the ongoing long-term extension study
    • Reported data that suggest FLT180a is the only treatment that has demonstrated durable levels of FIX activity in the normal range, with no bleeds requiring supplemental FIX to date
    • Refined our prophylactic immune management regimen, enabling control of transaminitis and preservation of gene expression, and demonstrated an overall favourable safety profile
    • Began enrollment of patients in the 6-month baseline run in study for the planned Phase 2b/3 pivotal trial, with initiation expected mid-year 2021
    • End of Phase 2 Meeting with the U.S. Food and Drug Administration (FDA) is anticipated before year end 2020, to align on a final dose and pivotal study design
    • Presented data from the B-AMAZE trial at the 13th Annual Congress of the European Association for Haemophilia and Allied Disorder (EAHAD) on February 7, 2020 and the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting on July 13, 2020.
    • Announced an agreement with Thermo Fisher Scientific securing dedicated production capacity and resources for the manufacture of FLT180a from 2021 to 2027, to support the planned Phase 2b/3 pivotal clinical trial and potential commercialisation

    Fabry Disease

    • Dosed the first patient in the ongoing MARVEL1 study, an adaptive dose finding Phase 1/2 study to assess the safety and efficacy of FLT190 in adult males with classic Fabry Disease
    • On February 12, 2020, presented preliminary data at the 16th annual WORLD SymposiumTM showing that, in the first patient dosed, the infusion was well tolerated and resulted in a four-fold increase in plasma αGLA activity by week four and maintained through the data cutoff at week 20
    • Received Orphan Drug Designation from the European Commission and the U.S. FDA for FLT190 for the treatment of Fabry Disease

    Gaucher Disease

    • On February 12, 2020, presented preclinical data at the 16th annual WORLD SymposiumTM demonstrating that Freeline's proprietary, in-house engineered GCase enzyme, with enhanced plasma stability for a 20-fold longer half-life, provides long term sustained steady state levels of fully active GCase in the bloodstream after a single injection. Uptake by macrophages in key organs, substrate clearance and greater bioavailability than the enzyme replacement therapy (ERT) velaglucerase alfa were observed

    Haemophilia A

    • On May 13, 2020, presented preclinical data at the 23rd Annual American Society of Gene & Cell Therapy Meeting detailing highly potent gene therapy candidates for Haemophilia A utilising novel, proprietary Factor VIII constructs that fit within the natural capacity of the AAV capsid

    Manufacturing and CMC Platform

    • Continued development of CMC platform and production using Freeline's proprietary manufacturing processes and related analytics at four locations in three countries, including Freeline's current good manufacturing practice (GMP) suite at the Cell and Gene Therapy Catapult Manufacturing Centre in the U.K., as well as Brammer, Novasep and Pall Corporation
    • Further advanced industry leading, proprietary analytic methods including long-read next generation sequencing (LR-NGS) for optimization of vector integrity and safety of product candidates
    • On May 12, 2020, presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting an in-depth analysis of AAV vector packaging and proprietary analytical tools, as well as the differences between classical vector preparations and Freeline's split packaging plasmid system
    • On May 14, 2020, presented at the 23rd Annual ASGCT Meeting the development of Freeline's commercial scale adherent cell culture bioreactor platform process for production of high potency and quality AAV product
    • Developed design concept for a Freeline manufacturing facility capable of meeting mid to long term clinical and commercial needs

    Corporate

    • Completed initial public offering for total net proceeds of $166.6 million
    • Completed $120 million Series C financing
    • Appointed Theresa Heggie as Chief Executive Officer
    • Appointed Julie Krop, M.D. as Chief Medical Officer
    • Promoted Romuald Corbau, Ph.D. from Senior Vice President, Research to Chief Scientific Officer

    First Half 2020 Financial Highlights

    Total operating expenses increased by approximately $11.4 million from $29.7 million for the six months ended June 30, 2019 to $41.1 million for the six months ended June 30, 2020.

    Research and development expenses increased by approximately $5.8 million from $23.6 million for the six months ended June 30, 2019 to $29.4 million for the six months ended June 30, 2020. The increase in research and development expenses was primarily attributable to an increase in spending on FLT180a specifically related to the ongoing Phase 1/2 B-AMAZE clinical trial and planned pivotal clinical trial as well as unallocated costs for all research and development activities including increased discovery costs.

    General and administrative expenses increased by $5.3 million from $6.0 million for the six months ended June 30, 2019, to $11.3 million for the six months ended June 30, 2020. The increase in general and administrative expenses was primarily attributable to increased legal and professional fees, and personnel costs.

    Total other income, net was $7.8 million for the six months ended June 30, 2020, compared to $5.1 million for the six months ended June 30, 2019. During the six months ended June 30, 2020 and 2019, Freeline recognised an R&D tax credit from the U.K. as a benefit within other income for approximately $6.9 million and $5.1 million, respectively.

    In June 2020, Freeline received net cash proceeds of approximately $69.0 million from the sale of series C preferred shares. During the six months ended June 30, 2020, net cash provided by financing activities was $67.9 million, consisting of net cash proceeds from the sale and issuance of series C preferred shares.

    During the six months ended June 30, 2020, net cash used in operating activities was $30.7 million, primarily resulting from a net loss of $33.3 million, adjusted for non-cash charges of $0.9 million for depreciation and $0.6 million of non-cash share-based compensation. The net loss was also partially offset by $0.9 million related to changes in components of working capital, including a $9.0 million increase in prepaid expenses and other assets and a $9.9 million increase in accounts payable and accrued expenses, all of which relate to increased research and development spending on preclinical and clinical trials and increased general and administrative spending resulting from increased professional and legal expenses incurred in conjunction with preparation for becoming a public company.

    On July 1, 2020, Freeline received an aggregate of approximately $10.0 million in additional proceeds from the sale of series C preferred shares, bringing the total net proceeds of the Series C financing to $79.0 million.

    On August 11, 2020, the Company completed a Nasdaq IPO of 8,823,529 American Depositary shares ("ADSs") representing the same number of ordinary shares at an IPO price of $18.00, for total net proceeds of approximately $147.7 million. On August 20, 2020, the underwriters of the IPO exercised a portion of their overallotment option by purchasing an additional 1,128,062 ADSs representing the same number of ordinary shares for additional net proceeds of $18.9 million. The total net proceeds from the IPO was $166.6 million.

    As of June 30, 2020 and September 30, 2020, Freeline had cash and cash equivalents of $106.6 million and $252.6 million, respectively.

    As of September 30, 2020, the Company had 35,872,749 ordinary shares outstanding.

    Freeline anticipates transitioning to quarterly reporting of financial results in 2021.

    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 U.K. and has operations in Germany and the U.S.

    About Haemophilia

    Haemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In Haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in Haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be ‘carriers' of the affected gene and may experience symptoms. Haemophilia A is the most common type of Haemophilia affecting about one in every 5,000 males, while Haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

    About FLT180a

    The Freeline Haemophilia B programme, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial, B-AMAZE, with the goal of normalising FIX activity in patients with moderate and severe Haemophilia.

    About Fabry Disease

    Fabry Disease is an inherited, X-linked disease characterised 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 enzyme (αGLA) 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 Enzyme Replacement Therapy and chaperone therapy to temporarily clear Gb3 accumulation and alleviate symptoms.

    About FLT190

    FLT190 is an investigational liver-directed adeno-associated viral (AAV) gene therapy for the treatment of Fabry Disease. We believe the programme is the first clinical-stage AAV gene therapy international study in Fabry Disease. FLT190 is an in-vivo gene therapy administered by a one-time intravenous infusion.

    The study, named MARVEL-1, is a multi-centre, 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 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 Type 1 Gaucher Disease, 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 programme is to deliver a one-time treatment of a long-lasting gene therapy that will provide a consistent and therapeutically relevant level of constant endogenous GCase, thus eliminating the need for ERT.

    About FLT201

    FLT201 is an investigational liver-directed adeno-associated viral (AAV) gene therapy in preclinical development for the treatment of Type 1 Gaucher Disease. By using Freeline's high-transducing AAVS3 capsid, our goal is to deliver GBA endogenously at normal levels with a one-time treatment. FLT201 contains a liver-specific promoter and a GBA sequence which expresses our novel, proprietary GBA variant with a greater than 20-fold longer half-life than wild-type GBA protein in purified systems. To our knowledge, we are the only company to date that has announced a programme for the development of an AAV gene therapy for the treatment of Type 1 Gaucher Disease and plan to file an IND for this programme in 2021.

    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, 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 an 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 commercialisation of its product candidates; and the Company's ability to identify or discover additional product candidates, or failure to capitalise on programmes 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

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877

    The following tables summarize our results of operations for the six months ended June 30, 2020 and 2019, as well as certain other financial information (U.S. dollar amounts in thousands):

      Six Months Ended June 30, 
    Selected Key Financial Measures 2020  2019  Change 
    Operating expenses:            
    Research and development $29,445  $23,616  $5,829 
    General and administrative  11,284   6,016   5,268 
    General and administrative - fees due to related parties  379   119   260 
    Total operating expenses  41,108   29,751   11,357 
    Loss from operations  (41,108)  (29,751)  (11,357)
    Other income, net:            
    Other income, net  862   22   840 
    Interest income, net  88      88 
    Benefit from R&D tax credit  6,894   5,084   1,810 
    Total other income, net  7,844   5,106   2,738 
    Loss before income taxes  (33,264)  (24,645)  (8,619)
    Income tax expense     (9)  9 
    Net loss $(33,264) $(24,654) $(8,610)



     
      September 30, 
      2020 
      (in thousands, except share amounts) 
    Ordinary shares  35,872,749 
    Cash and cash equivalents $252,639 

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  5. LONDON, Sept. 29, 2020 (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 Theresa Heggie, Chief Executive Officer, will present at Chardan's 4th Annual Genetic Medicines Conference at the revised time of 3.45 pm EDT on 6 October.

    A live audio webcast of the presentation will be available on the investor relations section of Freeline's website at https://www.freeline.life/investors-media/. An archived replay will be available on the Company's website for a period of 30…

    LONDON, Sept. 29, 2020 (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 Theresa Heggie, Chief Executive Officer, will present at Chardan's 4th Annual Genetic Medicines Conference at the revised time of 3.45 pm EDT on 6 October.

    A live audio webcast of the presentation will be available on the investor relations section of Freeline's website at https://www.freeline.life/investors-media/. An archived replay will be available on the Company's website for a period of 30 days after the conference.

    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, 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 an 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 programmes 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

    JW Communications

    Julia Wilson

    +44 (0) 7818 430877

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