BEAM Beam Therapeutics Inc.

41.56
-0.91  -2%
Previous Close 42.47
Open 42.49
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Market Cap $2,409,066,586
Shares 57,965,991
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Volume 397,686
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  1. CAMBRIDGE, Mass., Nov. 20, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today has been named one of the Top Places to Work in Massachusetts in the 13th annual employee-based survey project from The Boston Globe for the second year in a row. Top Places to Work recognizes the most admired workplaces in the state voted on by the people who know them best: their employees. The survey measures employee opinions about their company's direction, execution, connection, management, work, pay and benefits, and engagement.

    The employers are placed into one of four groups: small, with 50 to 99 employees; medium, with 100 to 249 workers; large, with…

    CAMBRIDGE, Mass., Nov. 20, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today has been named one of the Top Places to Work in Massachusetts in the 13th annual employee-based survey project from The Boston Globe for the second year in a row. Top Places to Work recognizes the most admired workplaces in the state voted on by the people who know them best: their employees. The survey measures employee opinions about their company's direction, execution, connection, management, work, pay and benefits, and engagement.

    The employers are placed into one of four groups: small, with 50 to 99 employees; medium, with 100 to 249 workers; large, with 250 to 999; and largest, with 1,000 or more. Beam was ranked as the top biotechnology company in the medium category and ranked 18 out of 55 in the category overall.

    "Beam was undoubtedly a great place to work when we received this honor last year, and despite the unprecedented circumstances as a result of the global pandemic, it remains true in 2020," said John Evans, chief executive officer of Beam Therapeutics. "COVID-19 has constantly challenged our teams to collaborate, innovate and stay connected in new ways. Our growth and progress over the course of 2020 speaks volumes to the exceptional values and commitment of every member of the Beam team."

    The rankings in Top Places to Work are based on confidential survey information collected by Energage, a Pennsylvania-based employee research and consulting firm specializing in employee engagement and retention, from over 80,000 employees at 285 Massachusetts organizations. The winners share a few key traits, including offering progressive benefits, giving their employees a voice, and encouraging them to have some fun while they're at it.

    "This was a particularly challenging year to be a great place to work, and the companies that made our list went above and beyond to keep their employees safe, engaged, and cared for," said Katie Johnston, the Globe's Top Places to Work editor. "From offering help with childcare to making the workplace more equitable to holding virtual talent shows, these employers showed that the best get better in crisis."

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization focused on its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.Beamtx.com.

    Contacts:

    Media:

    Dan Budwick

    1AB

     

    Investors:

    Monique Allaire

    THRUST Strategic Communications

     



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  2. Novel Base Editors for Two Most Common GSDIa Mutations Demonstrate Significantly Higher Levels of In Vivo Mutation Correction than Required to Restore Glucose Homeostasis

    Previously Presented Data from Alpha-1 Program to be Reviewed in Encore Presentation

    CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present preclinical data from its liver-focused programs, including the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa), during the American Association for the Study of Liver Diseases…

    Novel Base Editors for Two Most Common GSDIa Mutations Demonstrate Significantly Higher Levels of In Vivo Mutation Correction than Required to Restore Glucose Homeostasis

    Previously Presented Data from Alpha-1 Program to be Reviewed in Encore Presentation

    CAMBRIDGE, Mass., Nov. 13, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present preclinical data from its liver-focused programs, including the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa), during the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting Digital Experience being held virtually November 13-16, 2020.

    GSDIa, also known as Von Gierke disease, is an inborn disorder of glucose metabolism caused by mutations in the G6PC gene that disrupt a key enzyme, Glucose-6- Phosphatase (G6Pase), which is involved in glucose homeostasis. This disruption results in low blood glucose levels that can be fatal if patients do not adhere to a strict regimen of slow-release forms of glucose, administered every one to four hours (including overnight). There are currently no pharmacological therapies approved for patients with GSDIa. Beam has engineered novel adenine base editors (ABE) that, in preclinical models, have achieved high levels of precise correction of the two most prevalent GSD1a mutations, R83C and Q347X, in both in vitro and in vivo settings.

    "Base editing represents an exciting new opportunity for the treatment of serious diseases, including GSDIa, where patients are in need of a disease-modifying, life-long treatment," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "These first preclinical data from our GSDIa program demonstrate significant levels of precise correction of the disease-causing R83C and Q347X point mutations, well above the level that we believe is needed to have a clinically relevant treatment effect for patients with GSD1a. We look forward to presenting these data, as well as our encore presentation of our Alpha-1 data from ASGCT, as we continue to advance our liver base editing programs toward the clinic."

    Details of the GSDIa presentation are as follows:

    Title: Base-Editing as a Therapeutic Approach for the Direct Correction of Disease-Causing Mutations Underlying Glycogen Storage Disease Type Ia

    Publication Number: 0589

    Session Title: Genomics and Precision Medicine

    Data Summary: Beam's approach to treating patients with GSDIa is to deliver an ABE via lipid nanoparticle (LNP) to the liver to repair either the R83C or the Q347X mutations in G6PC. It is estimated that these two-point mutations account for 900 and 500 patients, respectively, in the United States, representing approximately 60% of all GSDIa patients. Animal studies suggest that a critical therapeutic threshold of approximately 11% of normal G6Pase activity in liver cells is sufficient to restore fasting glucose; however, this level must be maintained in order to preserve glucose control and alleviate other serious, and potentially fatal, GSDIa symptoms. In vivo correction of both mutations by ABEs was observed in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts reveal significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpass those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing. Further, these levels of in vivo correction for GSDIa by base-editing are achieved without creation of double-stranded breaks. In total, these data support base-editing technology as a promising approach for precise correction of causative mutations in GSDIa.

    Beam will also report data during an oral presentation at AASLD from its Alpha-1 Antitrypsin Deficiency (Alpha-1) program, which were previously presented at American Society of Gene & Cell Therapy 2020 Annual Meeting. Details of the Alpha-1 presentation are as follows:

    Title: Evaluation of Adenine Base Editing as a Potential Treatment for Alpha-1 Antitrypsin Deficiency

    Publication Number: 0032

    Session Title: Parallel 3: Metabolic and Genetic Diseases

    Session Broadcast Date and Time: Saturday, November 14, 2020, 2:00 p.m. ET

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.beamtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our plans for scientific publications; our plans to advance our liver base editing programs toward the clinic; the expected levels of GSDIa mutation correction that are required to be clinically relevant and disease-modifying in humans; and the therapeutic applications and potential of our technology, including our ability to develop precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and potentially commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarters ended March 31, 2020, June 30, 2020, and September 30, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

    Contacts:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

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  3. BEAM-201, an Off the Shelf Allogeneic CD7-Targeting CAR-T, Named as Development Candidate for Treatment of T-ALL; First Cell Therapy Featuring Four Simultaneous Genetic Edits; Demonstrates 96-99% On-target Editing and In Vivo Proof of Concept of Tumor Clearance

    Multiple Upcoming Data Presentations Demonstrate Strength and Breadth of Base Editing Platform, Including First Preclinical Data from GSDIa Program

    $135 Million in Capital Raised through Successful Follow-on Offering

    CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported pipeline updates, recent business highlights and third quarter 2020 financial…

    BEAM-201, an Off the Shelf Allogeneic CD7-Targeting CAR-T, Named as Development Candidate for Treatment of T-ALL; First Cell Therapy Featuring Four Simultaneous Genetic Edits; Demonstrates 96-99% On-target Editing and In Vivo Proof of Concept of Tumor Clearance

    Multiple Upcoming Data Presentations Demonstrate Strength and Breadth of Base Editing Platform, Including First Preclinical Data from GSDIa Program

    $135 Million in Capital Raised through Successful Follow-on Offering

    CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported pipeline updates, recent business highlights and third quarter 2020 financial results.

    "2020 has been a year of significant progress for Beam," said John Evans, chief executive officer of Beam. "Since the start of the year, we've named three development candidates from our portfolio, now including BEAM-201, our multiplex editing program for the treatment of T-cell acute lymphoblastic leukemia. We are also pleased to report that we're on track to submit our first IND in the second half of 2021, with BEAM-101 for the treatment of sickle cell disease. The continued advancement of our pipeline is a testament to both the strength and breadth of our base editing platform and our exceptional team. Combined with the capital we've added to our balance sheet, we are well positioned to continue our strategy of advancing multiple programs to the clinic in parallel, in the hope of providing much-needed new treatment options for patients with serious diseases."

    Base Editing Progress

    • First CAR-T Base Editing Development Candidate, BEAM-201, Named for Treatment of T-ALL; Data Presented at Society for Immunotherapy of Cancer's 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020): Beam is advancing BEAM-201, a potent and specific anti-CD7 CAR-T multiplex editing program for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), a severe disease affecting children and adults with five-year overall survival of less than 25%. BEAM-201 is produced using a GMP-compliant, clinical-scale process in which T-cells derived from healthy donors are simultaneously base edited at four genomic loci then transduced with a lentivirus coding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic ("off the shelf") CD7-targeting CAR-T cells resistant to both fratricide and immunosuppression. Preclinical data from this approach were reported in a poster as part of SITC 2020, which is being held virtually from November 9-14, 2020. Editing with BEAM-201 led to potent, dose-dependent tumor control in vitro and in an in vivo xenograft mouse model. Details of the presentation are as follows:
      • Title: Highly Efficient Multiplexed Base Editing Enables the Development of Investigational Universal CD7-targeting CAR-T Cells to Treat T-ALL

        Publication Number: 111

        Category: Cellular Therapies

    Upcoming Base Editing Data Presentations

    • First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia to be Presented at American Association for the Study of Liver Diseases' (AASLD) The Liver Meeting Digital Experience™: Beam will present the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa) during a poster session at AASLD's The Liver Meeting Digital Experience, which is being held virtually November 13-16, 2020. Details of the presentation are as follows:
      • Title: Base-Editing as a Therapeutic Approach for the Direct Correction of Disease-Causing Mutations Underlying Glycogen Storage Disease Type Ia

        Publication Number: 0589

        Session Title: Genomics and Precision Medicine

    Beam will also report data during an oral presentation at AASLD from its Alpha-1 Antitrypsin Deficiency (Alpha-1) program. Details of the presentation are as follows:

    • Title: Evaluation of Adenine Base Editing as a Potential Treatment for Alpha-1 Antitrypsin Deficiency

      Publication Number: 0032 

      Session Title: Parallel 3: Metabolic and Genetic Diseases 

      Session Broadcast Date and Time: Saturday, November 14, 2020, 2:00 p.m. ET
    • Updated Data from Novel Sickle Cell Disease Approaches to be Presented at 62nd American Society of Hematology Annual Meeting and Exposition (ASH 2020): Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102, and will present updated preclinical data from these two complementary editing programs during poster sessions at ASH 2020, being held virtually December 5-8, 2020. Details of the presentations are as follows:
      • Title: Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

        Session: 801. Gene Editing, Therapy and Transfer: Poster I

        Date: Saturday, December 5, 2020

        Publication Number: 1543
    • Title: Adenine Base Editing of Gamma Globin Gene Promoters Shows No Detectable Off-Target RNA or DNA Editing

      Session: 801. Gene Editing, Therapy and Transfer: Poster I

      Date: Saturday, December 5, 2020

      Publication Number: 1545

    Recent Business Highlights

    • Successful $135 Million Follow-on Offering Completed: In October 2020, Beam sold 5,000,000 shares of common stock at a public offering price of $23.50 per share. Underwriters exercised in full their option to purchase up to an additional 750,000 shares of common stock at the public offering price, less the underwriting discounts and commissions. The gross proceeds of the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Beam, were approximately $135.1 million. The net proceeds of the offering were $126.6 million.
    • Leadership Update – Courtney Wallace Promoted to Chief Business Officer: In November 2020, Beam promoted Courtney Wallace to chief business officer. Ms. Wallace previously served as senior vice president, head of business development and strategy at Beam. Ms. Wallace has served as Beam's head of corporate strategy and business development since 2018.

    Upcoming Investor Conference Presentation

    John Evans, chief executive officer, will participate in a fireside chat during the Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 4:25 p.m. GMT/11:25 a.m. ET.

    The live webcast will be available in the investor section of the company's website at www.beamtx.com. The webcast will be archived for 60 days following the presentation.

    Third Quarter 2020 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $202.2 million as of September 30, 2020. This cash balance does not include the proceeds of the October 2020 offering.
    • Research & Development (R&D) Expenses: R&D expenses were $29.8 million for the quarter ended September 30, 2020, compared to $12.5 million for the quarter ended September 30, 2019.
    • General & Administrative (G&A) Expenses: G&A expenses were $7.5 million for the quarter ended September 30, 2020, compared to $5.5 million for the quarter ended September 30, 2019.
    • Net Loss: Net loss attributable to common stockholders was $34.5 million, or $0.69 per share, for the quarter ended September 30, 2020, compared to $22.3 million, or $3.31 per share, for the quarter ended September 30, 2019.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.beamtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the expected timing of filing investigational new drug applications; our ability to advance programs to the clinic; the sufficiency of our cash position; expected presentations at upcoming conferences; and the therapeutic applications and potential of our technology, including our ability to develop precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarters ended March 31, 2020, June 30, 2020, and September 30, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

    Contacts:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

    Condensed Consolidated Balance Sheet Data 
    (in thousands) 
             
      September 30,

    2020
      December 31,

    2019
     
    Cash, cash equivalents, and marketable securities $202,220  $91,848 
    Total assets  278,273   156,099 
    Redeemable convertible preferred stock     302,049 
    Total stockholders' equity (deficit)  200,071   (201,104)



    Condensed Consolidated Statement of Operations 
    (in thousands, except share and per share data) 
                     
      Three Months Ended

    September
     30,
      Nine Months Ended

    September
     30,
     
      2020  2019  2020  2019 
    License revenue $6  $6  $18  $12 
    Operating expenses:                
    Research and development  29,825   12,543   70,728   34,402 
    General and administrative  7,502   5,487   21,251   14,393 
    Total operating expenses  37,327   18,030   91,979   48,795 
    Loss from operations  (37,321)  (18,024)  (91,961)  (48,783)
    Other income (expense):                
    Change in fair value of derivative liabilities  2,700   (1,600)  (8,700)  (3,600)
    Interest and other income (expense), net  169   619   1,533   1,907 
    Total other income (expense)  2,869   (981)  (7,167)  (1,693)
    Net loss $(34,452) $(19,005) $(99,128) $(50,476)
    Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock     (3,262)  (1,277)  (9,451)
    Net loss attributable to common stockholders $(34,452) $(22,267) $(100,405) $(59,927)
    Net loss per common share attributable to common stockholders, basic and diluted $(0.69) $(3.31) $(2.31) $(9.58)
    Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted  50,087,747   6,717,792   43,438,919   6,254,069 

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  4. BEAM-201, an Off the Shelf Allogeneic CD7-Targeting CAR-T, Named as Development Candidate for Treatment of T-Cell Acute Lymphoblastic Leukemia

    First Cell Therapy Featuring Simultaneous Edits to Four Genes; Demonstrates 96-99% On-target Editing at Clinical Scale and In Vivo Proof of Concept of Tumor Clearance

    CAMBRIDGE, Mass., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will advance BEAM-201, a potent and specific anti-CD7, multiplex edited, allogeneic CAR-T therapy, as a development candidate for the treatment of T-cell acute lymphoblastic leukemia (T-ALL). Preclinical data on BEAM-201 are being…

    BEAM-201, an Off the Shelf Allogeneic CD7-Targeting CAR-T, Named as Development Candidate for Treatment of T-Cell Acute Lymphoblastic Leukemia

    First Cell Therapy Featuring Simultaneous Edits to Four Genes; Demonstrates 96-99% On-target Editing at Clinical Scale and In Vivo Proof of Concept of Tumor Clearance

    CAMBRIDGE, Mass., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will advance BEAM-201, a potent and specific anti-CD7, multiplex edited, allogeneic CAR-T therapy, as a development candidate for the treatment of T-cell acute lymphoblastic leukemia (T-ALL). Preclinical data on BEAM-201 are being presented in a poster session during the Society for Immunotherapy of Cancer's 35th Anniversary Annual Meeting & Pre-Conference Programs (SITC 2020), and demonstrate potent, dose-dependent tumor control in vitro and in an in vivo xenograft model.

    "BEAM-201 is the third development candidate from our pipeline of base editing programs named this year, an incredible milestone for our company and a testament to the strength of our platform and the dedication of our team," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "BEAM-201 is a highly differentiated editing program designed to provide an ‘off-the-shelf' CD7-targeting CAR-T cell therapy that may enable a one-time treatment option for patients with T-ALL. To our knowledge, BEAM-201 is the first cell therapy featuring simultaneous edits to four genes. The preclinical data being presented at SITC 2020 demonstrate 96-99% editing efficiencies across four targets without genomic rearrangements, as well as strong in vivo proof of concept of tumor clearance in a xenograft model. We are actively advancing BEAM-201 to assess its potential impact in treating people living with this devastating disease."

    BEAM-201 is a potent and specific anti-CD7, multiplex edited, allogeneic CAR-T development candidate for the treatment of relapsed/refractory T-ALL, a severe disease affecting children and adults with a five-year overall survival of less than 25%. BEAM-201 is produced using a GMP-compliant, clinical-scale process in which T cells derived from healthy donors are simultaneously base edited at four genomic loci then transduced with a lentivirus coding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic ("off the shelf") CD7-targeting CAR-T cells resistant to both fratricide and immunosuppression.

    Details of Beam's SITC 2020 presentation of BEAM-201 are as follows:

    Title: Highly efficient multiplexed base editing enables the development of investigational universal CD7-targeting CAR-T Cells to treat T-ALL

    Publication Number: 111

    Category: Cellular Therapies

    Data Summary:

    • In vitro characterization of the effects of base editing in BEAM-201 demonstrated:
      • Simultaneous base editing by a cytosine base editor (CBE) at four target loci in primary human T cells using a clinical-scale process produced 96-99% on-target editing of each of the four genes as measured by next-generation sequencing and flow cytometry;
      • Simultaneous quad base editing of T cells resulted in no detected genomic rearrangements resulting from the editing process;
      • Multiplex base editing did not negatively affect cell expansion during manufacturing;
      • CBE-edited cells decreased expression of the four target genes with minimal effect on other genes, including key members of the p53 pathway that are upregulated in response to DNA double-stranded breaks produced by multiplex editing with nucleases.
    • Further characterization of BEAM-201 in vitro and in a tumor mouse model demonstrated:
      • Beam's GMP-compliant, clinical-scale process resulted in final BEAM-201 CAR-T cell populations with on-target editing efficiencies between 96-99.9% at each of the four target loci, and 85% CAR-expressing cells. As a result, Beam estimates that 91% of cells are bi-allelically quad base edited and 77% of cells have all 5 genetic modifications. The company believes this is the highest level and uniformity of CAR expression and simultaneous editing across four target sites reported at clinical scale to date.
      • BEAM-201 cells demonstrated robust in vitro CD7-dependent cytokine production, and rapid in vitro cytotoxicity.
      • BEAM-201 cells also demonstrated dose-dependent clearance or control, across a 25-fold dose range, of an aggressive disseminated CCRF-CEM T-ALL tumor mouse model. 

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.beamtx.com.

    Forward-Looking Statements

    This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our progress advancing BEAM-201; and the therapeutic applications and potential of our technology, including our ability to develop precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the heading "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarters ended March 31, 2020 and June 30, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

    Contacts:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

    Primary Logo

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  5. CAMBRIDGE, Mass., Oct. 02, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that John Evans, chief executive officer, will participate in a fireside chat during the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, Oct. 5, 2020 at 8:00 a.m. ET.

    The live webcast will be available in the investor section of the company's website at www.beamtx.com. The webcast will be archived for 60 days following the presentation.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise…

    CAMBRIDGE, Mass., Oct. 02, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that John Evans, chief executive officer, will participate in a fireside chat during the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, Oct. 5, 2020 at 8:00 a.m. ET.

    The live webcast will be available in the investor section of the company's website at www.beamtx.com. The webcast will be archived for 60 days following the presentation.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam's proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.beamtx.com.

    Contacts:

    Investors:

    Monique Allaire

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB

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