• Four-year research collaboration combines Pfizer's deep experience in global drug development, including programs utilizing messenger RNA (mRNA), lipid nanoparticles (LNP), and gene therapy, with Beam's leadership in base editing and mRNA/LNP delivery technologies
    • Beam will receive an upfront payment of $300 million, be eligible to receive future milestone payments of up to $1.05 billion for a potential total consideration of up to $1.35 billion
    • Beam may opt into a global co-development and co-commercialization agreement for one program
    • Research and development activities aim to advance potentially transformative therapies for patients living with rare genetic diseases

    NEW YORK and CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Pfizer

    • Four-year research collaboration combines Pfizer's deep experience in global drug development, including programs utilizing messenger RNA (mRNA), lipid nanoparticles (LNP), and gene therapy, with Beam's leadership in base editing and mRNA/LNP delivery technologies

    • Beam will receive an upfront payment of $300 million, be eligible to receive future milestone payments of up to $1.05 billion for a potential total consideration of up to $1.35 billion
    • Beam may opt into a global co-development and co-commercialization agreement for one program
    • Research and development activities aim to advance potentially transformative therapies for patients living with rare genetic diseases

    NEW YORK and CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Pfizer Inc. (NYSE:PFE) and Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced an exclusive four-year research collaboration focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system.  

    The base editing programs to be evaluated as part of the collaboration will leverage Beam's proprietary in vivo delivery technologies, which use messenger RNA (mRNA) and lipid nanoparticles (LNP) to deliver base editors to target organs. Combining these technologies with Pfizer's proven experience in developing and manufacturing medicines and vaccines, this collaboration seeks to advance potentially transformative therapies for patients living with rare diseases.

    Beam's proprietary base editing technologies are designed to enable a new class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. This approach aims to create a more precise and efficient edit compared to traditional gene editing methods, which operate by creating targeted double-stranded breaks in the DNA, resulting in potential challenges associated with unwanted DNA modifications.

    "At Pfizer, we believe in the powerful potential of mRNA and LNP technologies to address the greatest unmet needs for patients, as evidenced by the beneficial impact our mRNA/LNP-based COVID-19 vaccine is having on the pandemic," said Mikael Dolsten, M.D., Ph.D., Chief Scientific Officer and President, Worldwide Research, Development and Medical of Pfizer. "We have a strong history in developing gene replacement therapies for rare diseases, and we see this collaboration with Beam as an opportunity to advance the next generation of gene editing therapies – an exciting scientific frontier – potentially leading to transformation for people living with rare genetic diseases."

    "We are thrilled to partner with Pfizer, a global leader in the design, development, and commercialization of novel medicines," said John Evans, Chief Executive Officer of Beam. "Our leading platform for precision genetic medicine has greatly evolved over the last few years, and we are committed to ensuring the broadest reach of these potentially life-changing technologies. This collaboration will provide a unique opportunity to create potentially transformative base editing programs for indications with critical unmet needs, leveraging our proprietary base editing technology and expanding delivery capabilities. We look forward to working together with Pfizer to advance these technologies and potentially expand our impact for people suffering from serious diseases."

    Under the terms of the collaboration agreement, Beam will conduct all research activities through development candidate selection for three undisclosed targets, which are not included in Beam's existing programs. Pfizer may opt in to exclusive, worldwide licenses to each development candidate, after which it will be responsible for all development activities, as well as potential regulatory approvals and commercialization, for each such candidate. Beam has a right to opt in, at the end of Phase 1/2 studies, upon the payment of an option exercise fee, to a global co-development and co-commercialization agreement with respect to one program licensed under the collaboration pursuant to which Pfizer and Beam would share net profits as well as development and commercialization costs in a 65%/35% ratio (Pfizer/Beam).

    Beam will receive an upfront payment of $300 million and, assuming Pfizer exercises its opt-in license rights for all three targets, is eligible for development, regulatory and commercial milestone payments for potential total deal consideration of up to $1.35 billion. Beam is also eligible to receive royalties on global net sales for each licensed program. The collaboration has an initial term of four years and may be extended up to one additional year.

    About Pfizer: Breakthroughs That Change Patients' Lives

    At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

    Pfizer Disclosure Notice

    The information contained in this release is as of January 10, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

    This release contains forward-looking information about the potential of mRNA and LNP technology and a research collaboration between Pfizer and Beam focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle, and central nervous system, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any applications may be filed for any drug or vaccine candidates in any jurisdictions; whether and when regulatory authorities may approve any potential applications that may be filed for any drug or vaccine candidates in any jurisdictions, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether any such drug or vaccine candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of any drug or vaccine candidates; whether the collaboration between Pfizer and Beam will be successful; uncertainties regarding the impact of COVID-19 on Pfizer's business, operations and financial results; and competitive developments.

    A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2020 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

    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.

    Beam 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 potential benefits of our collaboration with Pfizer, any future payments we may receive under our research collaboration agreement with Pfizer, the therapeutic applications and potential of our technology, including our ability to deliver base editors to target organs in and beyond the liver 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 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:

    Pfizer:

    Media Relations

    +1 (212) 733-1226

    Investor Relations

    +1 (212) 733-4848

    Beam:

    Investors:

    Chelcie Lister

    THRUST Strategic Communications

    Media:

    Dan Budwick

    1AB



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  1. First Subject Anticipated to be Enrolled in BEAM-101 Phase 1/2 Clinical Trial for the Treatment of Sickle Cell Disease in the Second Half of 2022

    BEAM-301 Named as Fourth Development Candidate for the Treatment of Glycogen Storage Disease Type Ia

    Nomination of Two Additional Development Candidates Anticipated in 2022

    Company to Present Pipeline and Business Updates at 40th Annual J.P. Morgan Healthcare Conference on January 10, 2022, at 2:15 p.m. ET

    CAMBRIDGE, Mass., Jan. 09, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today outlined anticipated 2022 milestones across its ex vivo programs targeting editing of hematopoietic stem cells…

    First Subject Anticipated to be Enrolled in BEAM-101 Phase 1/2 Clinical Trial for the Treatment of Sickle Cell Disease in the Second Half of 2022

    BEAM-301 Named as Fourth Development Candidate for the Treatment of Glycogen Storage Disease Type Ia

    Nomination of Two Additional Development Candidates Anticipated in 2022

    Company to Present Pipeline and Business Updates at 40th Annual J.P. Morgan Healthcare Conference on January 10, 2022, at 2:15 p.m. ET

    CAMBRIDGE, Mass., Jan. 09, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today outlined anticipated 2022 milestones across its ex vivo programs targeting editing of hematopoietic stem cells (HSCs) and T cells and in vivo programs targeting editing of liver cells leveraging lipid nanoparticles (LNPs) for delivery. Updates include that the company has selected its fourth development candidate and first in vivo base editing candidate, BEAM-301, which aims to correct the R83C mutation for the potential treatment of patients with glycogen storage disorder Ia (GSDIa).

    "We made significant progress across our base editing portfolio in 2021, which culminated in U.S. Food and Drug Administration clearance of the first investigational new drug application of a base editing therapeutic, BEAM-101. We also further expanded our platform, particularly with LNP delivery of base editors to the liver and our proprietary technology for accelerating LNP delivery to other tissues, including HSCs," said John Evans, chief executive officer of Beam. "We believe 2022 is set to be our most important year yet, with preparations underway to launch the BEACON-101 clinical trial with BEAM-101 for the treatment of sickle cell disease and to complete our transition to becoming a clinical-stage company. We believe we are well positioned today, with four development candidates, a rich pipeline of earlier stage programs, and an industry-leading platform of editing and delivery technologies enabling us to bring forward a new class of precision genetic medicines. None of this would be possible without the commitment of our remarkable team of fearless innovators. We look forward to the year ahead and continuing our work to bring potentially life-changing medicines to as many patients as possible."

    Ex Vivo HSC Programs

    • BEAM-101 is a patient-specific, autologous HSC investigational therapy, which incorporates base edits that are designed to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin. BEAM-101 aims to potentially alleviate the effects of mutations causing sickle cell disease (SCD) or beta-thalassemia by leading to increases in fetal hemoglobin, which inhibits hemoglobin S (HbS) polymerization. The BEACON-101 trial is a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of SCD. The trial is expected to include an initial "sentinel" cohort of three patients, treated one at a time to confirm successful engraftment, followed by dosing in up to a total of 45 patients. Beam has begun site selection and the institutional review board approval processes for the BEACON-101 trial and plans to enroll the first subject in the second half of 2022.
    • BEAM-102 is designed to treat SCD by directly editing the causative HbS point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant has been reported to have the same function as the more common HbA variant and does not cause SCD. Beam plans to submit an investigational new drug (IND) application for BEAM-102 in the second half of 2022.

    Ex Vivo T Cell Programs

    • BEAM-201 is a multiplex base edited anti-CD7 CAR-T cell investigational therapy designed to treat relapsed/refractory T cell acute lymphoblastic leukemia, a severe disease affecting children and adults. Beam plans to submit an IND application for BEAM-201 in the second half of 2022.
    • Beam plans to nominate a second CAR-T development candidate in 2022.

    In Vivo LNP Liver-targeting Programs

    • BEAM-301, the company's newest development candidate, is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation. R83C is the most common disease-causing mutation of GSDIa, a life-altering genetic disease with no approved disease-modifying treatments available today. Beam anticipates initiating IND-enabling studies for BEAM-301 in 2022.
    • Beam plans to nominate a second liver-targeted development candidate in 2022.

    J.P. Morgan Healthcare Conference

    Mr. Evans will present Beam's pipeline and business updates during a presentation at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022, at 2:15 p.m. ET. A live 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.

    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 plans, and anticipated timing, to nominate additional development candidates, initiate IND-enabling studies, and submit IND applications; the therapeutic applications and potential of our technology, including with respect to sickle cell disease, beta-thalassemia, T-ALL, GSDIa, and LNPs; the planned initiation and design of our BEACON-101 clinical trial, including the timing of enrolling the first subject in the trial; our planned presentations at an upcoming conference; 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 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., Jan. 03, 2022 (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 present at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 2:15 p.m. ET.

    A live 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…

    CAMBRIDGE, Mass., Jan. 03, 2022 (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 present at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022 at 2:15 p.m. ET.

    A live 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

     



    Primary Logo

    View Full Article Hide Full Article
  3. CAMBRIDGE, Mass., Dec. 12, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today shared a three-stage, long-term development strategy for its base editing approach to treat sickle cell disease (SCD). The plan, and supportive data, are being presented during a Scientific Program Session oral presentation and two poster sessions at the 63rd American Society for Hematology (ASH) Annual Meeting & Exposition.

    SCD is a severe, hereditary monogenic blood disorder that alters the structure and function of oxygen-carrying hemoglobin in red blood cells and is caused by a single letter spelling error in the beta globin gene. To fully address SCD and…

    CAMBRIDGE, Mass., Dec. 12, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today shared a three-stage, long-term development strategy for its base editing approach to treat sickle cell disease (SCD). The plan, and supportive data, are being presented during a Scientific Program Session oral presentation and two poster sessions at the 63rd American Society for Hematology (ASH) Annual Meeting & Exposition.

    SCD is a severe, hereditary monogenic blood disorder that alters the structure and function of oxygen-carrying hemoglobin in red blood cells and is caused by a single letter spelling error in the beta globin gene. To fully address SCD and support broad accessibility, Beam is deploying a stepwise strategy that includes advancements of its ex vivo programs, BEAM-101 and BEAM-102, improvements in patient conditioning regimens, and enablement of in vivo base editing with delivery directly into patients via lipid nanoparticles (LNPs).

    "Our goal is to make disease-altering, lifelong treatments with base editing broadly accessible to SCD patients around the globe, and, over time, improve on the delivery of these therapies so patients everywhere can opt for a potentially curative therapy," said John Evans, chief executive officer of Beam. "We have focused our initial SCD efforts on our ex vivo programs, BEAM-101 and BEAM-102, which leverage established clinical and regulatory strategies and have the potential to offer transformative therapeutic options for SCD patients. To further improve the therapeutic options for SCD patients, we also aim to develop lower-toxicity conditioning methods for improved transplant, as well as pursuing in vivo delivery of our base editors using our innovative LNP delivery capabilities. We believe this suite of technologies – base editing, improved conditioning and in vivo delivery for editing HSCs – can maximize the potential of our SCD programs as well as create a powerful platform for the treatment of many other severe genetic blood disorders."

    During the presentations at ASH, Beam is highlighting its stepwise strategy for treating SCD and is presenting complementary data supporting several components of its approach:

    Wave 1: Ex Vivo Base Editing via Autologous Transplant

    Beam is advancing ex vivo base editing programs, in which cells are collected from a patient, edited and then infused back into the patient following a conditioning regimen, such as treatment with busulfan, the standard of care in hematopoietic stem cell (HSC) transplantation today. This approach is being deployed in the company's BEAM-101 and BEAM-102 base editing programs, and allows the company to pursue an efficient path for development using increasingly validated clinical endpoints and regulatory strategies.

    BEAM-101 is an investigational, patient-specific, autologous HSC therapy which incorporates base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin to potentially alleviate the effects of mutations causing SCD or beta-thalassemia. The company recently received clearance of its Investigational New Drug application for this program and is working to initiate its Phase 1/2 BEACON-101 trial.

    BEAM-102 aims to treat SCD by directly editing the causative HbS point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant has been reported to have the same function as the wild-type variant and does not cause SCD.

    During ASH, Beam is presenting updated preclinical data further characterizing Makassar hemoglobin created by BEAM-102 and demonstrating biophysical and biochemical properties consistent with normal hemoglobin, as expected:

    • Makassar globin did not polymerize in vitro
    • Cells co-expressing Makassar globin and sickle globin had properties similar to sickle trait cells, which are cells with one normal globin gene and one sickle globin gene and which do not sickle
    • Oxygen binding of Makassar globin was comparable to the most abundant normal adult hemoglobin (HbA)
    • The crystal structures of the Makassar globin and of HbA were superimposable, suggesting no meaningful structural differences

    Wave 2: Improved Conditioning

    In parallel with Wave 1 development, Beam also aims to improve the transplant conditioning regimen for SCD patients, reducing toxicity challenges associated with standard of care. Conditioning is a critical component necessary to prepare a patient's body to receive the ex vivo edited cells that must engraft in the patient's bone marrow in order to be effective. Today's conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. Beam has a collaboration with Magenta Therapeutics to evaluate the potential utility of MGTA-117, Magenta's novel antibody drug conjugate that is designed to precisely target only hematopoietic stem and progenitor cells, sparing immune cells. Importantly, improved conditioning regimens could potentially be paired with BEAM-101 and BEAM-102, as well as other base editing programs in hematology.

    Wave 3: In Vivo Base Editing via HSC-targeted LNPs

    Beam is also exploring the potential for in vivo base editing programs for SCD, in which base editors would be delivered to the patient through an infusion of LNPs targeted to HSCs, eliminating the need for transplantation altogether. This approach could provide a more accessible option for patients, particularly in regions where ex vivo treatment is challenging. Building on its acquisition of Guide Therapeutics, Beam has established a DNA-barcoded LNP screening technology to enable high-throughput in vivo identification of LNPs with novel biodistribution and selectivity for target organs beyond the liver.

    During ASH, Beam is presenting updated preclinical data using this technology to screen more than 1,000 LNPs for potential to deliver to HSCs and identified LNP-HSC1 as the most potent, with efficient transfection in both mice and non-human primates. Updated findings in the poster showed:

    • LNP-HSC1 was validated in vivo, leading to durable, dose-dependent mRNA transfection in HSCs and resulting in fluorescent reporter expression in more than 40% of cells, now maintained out to 16 weeks post-delivery
    • LNP-HSC1 efficiently transfected human CD34+ cells in vitro
    • LNP-HSC1 efficiently transfected nearly 20% of CD34+ HSCs in humanized mice and non-human primates at a dose of 1.0 mg/kg.

    Beam is continuing work to identify more potent LNPs for applications in in vivo base editing of HSCs. As with Wave 2, Beam can potentially leverage the same base editing payloads used in BEAM-101 and BEAM-102 for future in vivo programs, as well as expanding the use of the technology to other indications in hematology.

    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 strategic initiatives for our base editing program targeting SCD; 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, conditioning and LNP screening; the planned initiation of our BEACON-101 trial; 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 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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  4. 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.

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    Chelcie Lister

    THRUST Strategic Communications

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    Dan Budwick

    1AB



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