BEAM Beam Therapeutics Inc.

78.69
-3.22  -4%
Previous Close 81.91
Open 80
52 Week Low 39.71
52 Week High 138.5219
Market Cap $5,361,605,945
Shares 68,135,798
Float 45,106,243
Enterprise Value $4,793,038,214
Volume 461,283
Av. Daily Volume 588,090
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Drug Pipeline

Drug Stage Notes
BEAM-101 - BEACON
Sickle cell disease
Phase 1
Phase 1
IND cleared by FDA, noted November 8, 2021.

Latest News

  1. CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced new preclinical research demonstrating the ability of the company's multiplex edited CAR T cells to target CD5-positive tumors, leading to tumor clearance in vivo. The data are highlighted in a poster titled, "CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies," at the Society for Immunotherapy of Cancer's (SITC) 36th Annual Meeting, being held Nov. 10-14, 2021.

    CAR T therapies have the potential to address a number of T-cell malignancies, but their therapeutic potential…

    CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced new preclinical research demonstrating the ability of the company's multiplex edited CAR T cells to target CD5-positive tumors, leading to tumor clearance in vivo. The data are highlighted in a poster titled, "CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies," at the Society for Immunotherapy of Cancer's (SITC) 36th Annual Meeting, being held Nov. 10-14, 2021.

    CAR T therapies have the potential to address a number of T-cell malignancies, but their therapeutic potential is often hindered by technological limitations in their development and application. Beam has developed a process using base editing to simultaneously silence five target genes, including CD5 and PD1, to create allogeneic anti-CD5 CAR T-cells with enhanced effector function for potential use as off-the-shelf treatments for T-cell malignancies.

    "There is a significant unmet need in the treatment of T-cell lymphomas, as some indications have particularly poor prognoses and patients with relapsed or refractory disease have few treatment options that can produce deep and durable responses," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "We believe our approach using multiplex editing of T-cells to produce allogeneic anti-CD5 CAR T investigational therapies may offer enhanced potency and more durable responses for patients with T-cell malignancies. Importantly, our base editing approach is designed to avoid making double-stranded breaks in DNA that are produced by multiplex editing with nucleases, thereby decreasing the potential for translocations and other genomic aberrations with potential for unforeseen consequences, increasing their potential safety and tolerability." 

    Key findings reported in the poster include:

    • Multiplexed base editing resulted in anti-CD5 CAR T cells with 94-98% editing efficiency at all 5 target loci and transduction efficiency of over 85%, which Beam believes is an efficiency at which the likelihood of graft versus host disease, CAR T rejection, and immunosuppression would be greatly reduced;
    • CD5 knockout improved production of a combination of proinflammatory cytokines, including a greater than 300% increase in IL-2 production;
    • CD5 knockout greatly improved in vivo efficacy of anti-CD5 CAR T cells in a xenograft mouse model of T-ALL compared to unedited cells. CD5 edited anti-CD5 CAR T cells were able to clear established tumor in a dose dependent manner; however, unedited anti-CD5 CAR T cells failed to clear initial tumor at all doses despite demonstrating in vitro efficacy.
    • Mice previously cleared of tumor underwent additional tumor challenges to assess the persistence and functionality of anti-CD5 CAR T cells and were cleared of tumor both a second and third time, indicating extended persistence of functional anti-CD5 CAR T cells in vivo.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables 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.

    Cautionary Note Regarding 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 planned base editing data presentations at an upcoming scientific conference; the therapeutic applications and potential of our technology, including with respect to T-cell malignancies; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important 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 studies 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 headings "Risk Factors Summary" and "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements 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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  2. CAMBRIDGE, Mass., Nov. 11, 2021 (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 Jefferies 2021 Virtual London Healthcare Conference, being held November 16-19, 2021. The fireside chat will be available to registered participants on-demand between 8:00 a.m. GMT on November 18, 2021, and 5:00 p.m. GMT on November 19, 2021.

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

    About Beam Therapeutics
    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology…

    CAMBRIDGE, Mass., Nov. 11, 2021 (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 Jefferies 2021 Virtual London Healthcare Conference, being held November 16-19, 2021. The fireside chat will be available to registered participants on-demand between 8:00 a.m. GMT on November 18, 2021, and 5:00 p.m. GMT on November 19, 2021.

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

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables 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.

    Contacts:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB



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  3. BEAM-101 IND Cleared by FDA for Evaluation as a Treatment for Sickle Cell Disease

    BEAM-102 IND-Enabling Studies Also Underway

    CAMBRIDGE, Mass., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business and pipeline updates, as well as third quarter 2021 financial results.

    As part of today's update, Beam announced that its Investigational New Drug (IND) application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration (FDA). BEAM-101, the company's lead ex vivo base editing product candidate, is a patient-specific, autologous hematopoietic investigational cell…

    BEAM-101 IND Cleared by FDA for Evaluation as a Treatment for Sickle Cell Disease

    BEAM-102 IND-Enabling Studies Also Underway

    CAMBRIDGE, Mass., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported recent business and pipeline updates, as well as third quarter 2021 financial results.

    As part of today's update, Beam announced that its Investigational New Drug (IND) application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration (FDA). BEAM-101, the company's lead ex vivo base editing product candidate, is a patient-specific, autologous hematopoietic investigational cell therapy which incorporates base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin (HPFH) to potentially alleviate the effects of mutations causing sickle cell disease or beta-thalassemia. This is the first open IND for base editing technology, a next-generation form of CRISPR capable of making single base changes without creating double strand breaks in the DNA. Beam is preparing to initiate a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which Beam refers to as the BEACON-101 trial.

    "We are thrilled to share that the FDA has cleared our first IND. BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables the important transition from a preclinical to a clinical-stage company, bringing us closer to our ultimate goal of helping patients," said John Evans, chief executive officer of Beam. "As leaders in the field of base editing, this milestone underscores the expertise of our team and the significant potential of our technology. We are grateful to the team members who have dedicated countless hours to this effort, and we look forward to this program's continued evaluation in the clinic. As we look toward 2022, we believe we are well-positioned both financially and organizationally to execute on our vision."

    "In addition to the clearance of our IND for BEAM-101, we are pleased to announce that we have initiated IND-enabling studies for BEAM-102, our Makassar base editing approach for the treatment of sickle cell disease," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "We have also made continued progress on BEAM-201, our multiplexed base editing approach for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic leukemia as well as continued advancement across our robust portfolio, as showcased by a number of recent and upcoming data presentations demonstrating our leadership in base editing as well as optimized delivery technologies for our programs. Importantly, we also expect to nominate our first development candidate for in vivo base editing in the liver using LNP delivery for the treatment of patients with glycogen storage disease type Ia (R83C mutation) by the end of the year. We've made extraordinary progress as a company this year and remain committed to advancing novel science so that we may reach as many patients as possible."

    Upcoming Base Editing Data Presentations

    • Preclinical data highlighting potential of CAR T multiplex base editing approach targeting CD5 at 2021 SITC Annual Meeting. At the Society for Immunotherapy of Cancer's (SITC) 36th Annual Meeting, Beam will present preclinical data from its multiplex edited allogeneic CAR T research targeting CD5-positive hematologic malignancies in a poster titled "CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies."
    • Multiple abstracts accepted for presentation at the 63rd American Society of Hematology Annual Meeting & Exposition (ASH). At this year's ASH meeting, Beam will present preclinical data highlighting its base editing approach to address sickle cell disease, including new preclinical data for BEAM-102, and data highlighting Beam's in vivo high-throughput lipid nanoparticle (LNP) screening approach to identify novel LNPs that can deliver base editors to tissues beyond the liver, such as hematopoietic stem cells. In addition, Beam will present an overview on the application of base editing for the treatment of beta-hemoglobinopathies and other genetic blood disorders during a scientific program session. Details of the presentations can be found here.

    Recent Base Editing Progress & Data Updates

    • BEAM-102 IND-enabling studies initiated. Beam has initiated IND-enabling studies for BEAM-102 for the treatment of sickle cell disease. BEAM-102 aims to directly correct the causative mutation in sickle cell disease by recreating a naturally-occurring normal human hemoglobin variant, HbG Makassar.
    • Preclinical data highlighting base editing approach to address GSDIa presented at European Society of Gene & Cell Therapy. Beam recently presented preclinical data demonstrating the ability of its liver-targeted base editing approach to directly correct R83C, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa), and the elimination of the disease phenotype in a novel in vivo model.
    • Optimized LNP delivery approaches for in vivo base editing to the liver and other tissues presented at TIDES 2021. Beam announced new preclinical data highlighting advancements with its approach to developing novel LNP formulations for increased in vivo liver editing potency, with high levels of editing at what Beam believes could be a clinically-relevant dose level of 1.0 mg/kg. Beam remains on track to nominate its first Development Candidate for in vivo base editing using LNP delivery by the end of 2021. Also at TIDES, Beam reported an update on its proprietary approach to developing LNPs to deliver base editors to tissues beyond the liver, including hematopoietic stem and progenitor cells.
    • Preclinical data highlighting potential of base editors to target disease drivers of chronic hepatitis B infection presented at 2021 International HBV Meeting. Beam recently presented data demonstrating the potential of its cytosine base editors to reduce viral markers, including hepatitis B surface antigen (HBsAg) expression, and prevent viral rebound of hepatitis B virus (HBV) in in vitro models.

    Business Highlights

    • Executed non-exclusive option and license agreement with Sana Biotechnology for Cas12b. Under the agreement, Beam granted Sana non-exclusive commercial rights to utilize its CRISPR Cas12b system with certain allogeneic T-cell and stem cell-derived programs. The agreement excludes any rights to base editing using Beam's Cas12b system, which rights remain exclusively with Beam. Sana agreed to pay Beam an upfront payment of $50 million, and Beam may also receive certain target option exercise fees, certain milestone payments upon the achievement of certain development and commercial sale milestones, and certain royalties on net sales of royalty-bearing products by Sana.

    Third Quarter 2021 Financial Results

    • Cash Position: Cash, cash equivalents and marketable securities were $933.4 million as of September 30, 2021, compared to $299.7 million as of December 31, 2020.
    • Research & Development (R&D) Expenses: R&D expenses were $54.6 million for the third quarter of 2021, compared to $29.8 million for the third quarter of 2020.
    • General & Administrative (G&A) Expenses: G&A expenses were $15.8 million for the third quarter of 2021, compared to $7.5 million for the third quarter of 2020.
    • Net Loss: Net loss attributable to common stockholders was $28.1 million, or $0.42 per share, for the third quarter of 2021, compared to $34.5 million, or $0.69 per share, for the third quarter of 2020.

    About Sickle Cell Disease and Beta-Thalassemia

    Sickle cell disease, a severe inherited blood disease, is caused by a single point mutation, E6V, in the beta globin gene. This mutation causes the mutated form of sickle hemoglobin (HbS) to aggregate into long, rigid molecules that bend red blood cells into a sickle shape under conditions of low oxygen. Sickled cells obstruct blood vessels and die prematurely, ultimately resulting in anemia, severe pain (crises), infections, stroke, organ failure, and early death. Sickle cell disease is the most common inherited blood disorder in the United States, affecting an estimated 100,000 individuals, of which a significant proportion are of African-American descent. Beta-thalassemia is another inherited blood disorder characterized by severe anemia caused by reduced production of functional hemoglobin due to insufficient expression of the beta globin protein. Transfusion-dependent beta-thalassemia (TDBT) is the most severe form of this disease, often requiring multiple transfusions per year. Patients with TDBT suffer from failure to thrive, persistent infections, and life-threatening anemia.

    About BEAM-101

    BEAM-101 is a patient-specific, autologous hematopoietic cell therapy under investigation for the treatment of sickle cell disease. BEAM-101 incorporates ex vivo base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin (HPFH) to potentially alleviate the effects of mutations causing sickle cell disease or beta-thalassemia by increasing the levels of fetal hemoglobin (HbF).

    About Beam Therapeutics

    Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables 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 potentially life-long cures to patients suffering from serious diseases.

    Cautionary Note Regarding 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 commencement of clinical trials for BEAM-101, including our BEACON-101 trial; our expectation that we will nominate our first development candidate for in vivo base editing in the liver using LNP delivery for the treatment of patients with GSDIa disease (R83C mutation) by the end of the year; any future payments we may receive under our agreement with Sana; our planned base editing data presentations at upcoming scientific conferences; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important 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 studies 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 headings "Risk Factors Summary" and "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, our Quarterly Report on Form 10-Q that we will file for the quarter ended September 30, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements 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 (unaudited)    
    (in thousands)    
             
      September 30, 2021 December 31, 2020    
    Cash, cash equivalents, and marketable securities $933,405  $299,671     
    Total assets  1,156,555   451,677     
    Total liabilities  302,741   206,116     
    Total stockholders' equity  853,814   245,561     
             
    Condensed Consolidated Statement of Operations (unaudited)    
    (in thousands, except share and per share data)    
             
      Three Months Ended September 30, Six Months Ended September 30,
       2021   2020   2021   2020 
    License revenue $763  $6  $775  $18 
    Operating expenses:        
    Research and development  54,623   29,825   290,306   70,728 
    General and administrative  15,774   7,502   39,450   21,251 
    Total operating expenses  70,397   37,327   329,756   91,979 
    Loss from operations  (69,634)  (37,321)  (328,981)  (91,961)
    Other income (expense):        
    Change in fair value of derivative liabilities  35,800   2,700   (8,400)  (8,700)
    Change in fair value of long-term investments  (4,892)  -   21,960   517 
    Change in fair value of contingent consideration liabilities  10,599   -   9,553   - 
    Interest and other income (expense), net  9   169   (63)  1,016 
    Total other income (expense)  41,516   2,869   23,050   (7,167)
    Net loss $(28,118) $(34,452) $(305,931) $(99,128)
    Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock  -   -   -   (1,277)
    Net loss attributable to common stockholders $(28,118) $(34,452) $(305,931) $(100,405)
    Net loss per common share attributable to common stockholders, basic and diluted $(0.42) $(0.69) $(4.86) $(2.31)
    Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted  66,377,611   50,087,747   62,960,219   43,438,919 
             



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  4. CAMBRIDGE, Mass., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that John Maraganore, Ph.D., founding chief executive officer and director of Alnylam Pharmaceuticals, has joined the company's board of directors.

    "John is a pioneer and leader in the biopharma community and has been responsible for building some of the most successful biotech companies and patient-centric drug development programs of our time. He brings tremendous experience leading companies from drug discovery stages through commercialization, and we are thrilled to welcome him to the Beam board of directors," said John Evans, chief executive officer…

    CAMBRIDGE, Mass., Nov. 08, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that John Maraganore, Ph.D., founding chief executive officer and director of Alnylam Pharmaceuticals, has joined the company's board of directors.

    "John is a pioneer and leader in the biopharma community and has been responsible for building some of the most successful biotech companies and patient-centric drug development programs of our time. He brings tremendous experience leading companies from drug discovery stages through commercialization, and we are thrilled to welcome him to the Beam board of directors," said John Evans, chief executive officer of Beam Therapeutics. "2021 has been a significant year for Beam, including the recent clearance of our Investigational New Drug application for BEAM-101, enabling our transition to a clinical-stage organization. Along with the rest of the board, I look forward to partnering with John as we continue building the leading company in the field of precision genetic medicines."

    "Beam has positioned itself as a true innovator in the field of gene editing, which has the potential to change both the treatment approach and the course of disease for patients with a wide range of severe genetically-driven diseases," said Dr. Maraganore. "The company is at an important inflection point as it prepares to advance the first-ever base editor into clinical development for people with sickle cell disease. I look forward to contributing to these efforts and working with the great team at Beam as they pursue their goal of bringing important new gene editing medicines to patients."

    Dr. Maraganore has served as the founding CEO and a director of Alnylam since 2002, where he has built and led the company from early platform research on RNA interference through global approval and commercialization of the first three RNAi medicines including ONPATTRO®, GIVLAARI®, and OXLUMO®. Prior to Alnylam, Dr. Maraganore served as an officer and a member of the management team for Millennium Pharmaceuticals, Inc., most recently as senior vice president, strategic product development where he was responsible for the company's product franchises in oncology, and cardiovascular, inflammatory, and metabolic diseases. Before Millennium, he served as director of molecular biology and director of market and business development at Biogen, Inc. At Biogen, Dr. Maraganore invented and led the discovery and development of ANGIOMAX® (bivalirudin) for injection, formerly HIRULOG™ and currently marketed by The Medicines Company. Prior to Biogen, Dr. Maraganore was a scientist at ZymoGenetics, Inc., and the Upjohn Company. Dr. Maraganore received his M.S. and Ph.D. in biochemistry and molecular biology at the University of Chicago. He is a member of the board of Agios Pharmaceuticals and Biotechnology Innovation Organization (BIO), where he was Chair from 2017-2019.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables 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.

    Cautionary Note Regarding 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 commencement of clinical trials for BEAM-101; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important 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 studies 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 headings "Risk Factors Summary" and "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements 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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  5. Preclinical Data Demonstrates that BEAM-102 Makassar Base Editing Converts Sickle Hemoglobin to Functional Hemoglobin for Potential Treatment of Sickle Cell Disease

    New In Vivo Data Highlight Novel LNP Screening Technology to Identify LNPs for Delivery of Base Editors to Tissues Beyond the Liver, Including to Blood Stem Cells

    CAMBRIDGE, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that preclinical data highlighting the company's Makassar base editing approach for the potential treatment of sickle cell disease (SCD) and its proprietary lipid nanoparticle (LNP) screening capabilities will be presented during…

    Preclinical Data Demonstrates that BEAM-102 Makassar Base Editing Converts Sickle Hemoglobin to Functional Hemoglobin for Potential Treatment of Sickle Cell Disease

    New In Vivo Data Highlight Novel LNP Screening Technology to Identify LNPs for Delivery of Base Editors to Tissues Beyond the Liver, Including to Blood Stem Cells

    CAMBRIDGE, Mass., Nov. 04, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that preclinical data highlighting the company's Makassar base editing approach for the potential treatment of sickle cell disease (SCD) and its proprietary lipid nanoparticle (LNP) screening capabilities will be presented during two poster sessions at the 63rd American Society of Hematology (ASH) Annual Meeting. In addition, Beam will present an overview on the application of base editing for the treatment of beta-hemoglobinopathies and other genetic blood disorders during a Scientific Program session titled, "Gene Editing 2.0: Advances in Gene Editing to Treat Genetic Blood Disorders." The ASH Annual Meeting is being held December 11-14, 2021, virtually and in Atlanta.

    "We look forward to presenting these new data at ASH, which further support our leadership position in the field of base editing and the significant opportunity we have to potentially make a difference for patients in need of meaningful treatment options," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "The additional preclinical data being presented from our Makassar program continue to validate the potential of our base editing approach to treating SCD, which is designed to correct sickle globin to a normally functioning hemoglobin variant. In addition, the ability of our in vivo high-throughput LNP screening technology to identify novel LNPs capable of delivering our editors to stem cells is exciting and supports a promising delivery platform that could eventually eliminate the need for transplant in sickle cell disease and other genetic blood disorders. Taken together, these data, coupled with our ongoing work around non-genotoxic conditioning, give us hope to be able to provide a more holistic treatment approach for patients with SCD and beyond."

    Details of the poster presentations are as follows:

    Poster Title: Conversion of HbS to Hb G-Makassar by Adenine Base Editing is Compatible with Normal Hemoglobin Function

    Session Name: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster I

    Date: Saturday, December 11, 2021

    Presentation Time: 5:30p.m - 7:30p.m.

    Location: Georgia World Congress Center, Hall B5

    Abstract Summary: Beam is advancing BEAM-102, its Makassar base editing approach, as a potential treatment for SCD that is constructed to directly edit the causative HbS point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant has been reported to be a functional beta-hemoglobin that is not pathogenic. In preclinical studies, Beam sought to further characterize Makassar hemoglobin, evaluating its size, molecular weight, oligomerization and polymerization potential, oxygen binding properties, as well as solving its crystal structure. Altogether, Beam's biophysical and biochemical characterization shows that Makassar globin behaves as a functional hemoglobin and represents a promising investigational approach for the treatment of sickle cell disease.

    Poster Title: Screening of Chemically Distinct Lipid Nanoparticles In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells

    Session Name: 801. Gene Therapies: Poster II

    Date: Sunday, December 12, 2021

    Presentation Time: 6:00p.m. - 8:00p.m.

    Location: Georgia World Congress Center, Hall B5

    Abstract Summary: Beam is developing an approach to directly deliver base editors to hematopoietic stem and progenitor cells (HSPCs) in vivo through non-viral delivery methods, such as LNPs for the potential delivery of base editors as a treatment for a range of hemoglobinopathies. To identify optimal LNPs for delivery to HSPCs, leveraging its proprietary DNA barcoding technology, Beam screened more than 1,000 LNPs and identified LNP-HSC1 as the most potent. LNP-HSC1 was validated in vivo, leading to durable, dose-dependent mRNA transfection in HSPCs of more than 40%, which was maintained for 10 weeks post-delivery. Further, LNP-HSC1 efficiently transfected mice at doses ranging from 0.3mg/kg-1.0mg/kg, and in non-human primates, a dose-dependent increase in mRNA in bone marrow-derived CD34+ HSPCs was observed.

    About Beam Therapeutics

    Beam Therapeutics (NASDAQ:BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that enables 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.

    Cautionary Note Regarding 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 planned base editing and LNP screening data presentations at an upcoming scientific conference; the therapeutic applications and potential of our technology, including with respect to SCD and LNP screening; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important 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 studies 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 headings "Risk Factors Summary" and "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2020, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements 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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