Beam Therapeutics Inc. (Beam), a biotechnology company, is establishing the fully integrated platform for precision genetic medicines.
The company’s suite of gene editing technologies is anchored by its proprietary base editing technology, which potentially enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. This approach uses a chemical reaction designed to create precise, predictable and effic...
Beam Therapeutics Inc. (Beam), a biotechnology company, is establishing the fully integrated platform for precision genetic medicines.
The company’s suite of gene editing technologies is anchored by its proprietary base editing technology, which potentially enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. This approach uses a chemical reaction designed to create precise, predictable and efficient genetic outcomes at the targeted sequence.
The company’s proprietary base editors have two principal components: (i) a clustered regularly interspaced short palindromic repeats, or CRISPR, protein, bound to a guide RNA, that leverages the established DNA-targeting ability of CRISPR, but is modified to not cause a double-stranded break; and (ii) a base editing enzyme, such as a deaminase, which carries out the desired chemical modification of the target DNA base.
The company is also pursuing a suite of delivery modalities, including both ex vivo and in vivo approaches, depending on tissue type.
The company is seeking to build the leading integrated platform for precision genetic medicine, which has broad therapeutic applicability and the potential to transform the field of precision genetic medicines.
The company is prioritizing the lead programs in its hematology and genetic disease portfolios, each of which have the potential to provide differentiated therapies for significant patient populations with high unmet medical need:
BEAM-101 is a patient-specific, autologous hematopoietic stem cell, or HSC, investigational therapy designed to offer a potentially best-in-class profile, incorporating base edits that are intended to alleviate the effects of sickle cell disease by increasing fetal hemoglobin, which is expected to increase functional hemoglobin production, and in the case of sickle cell disease, inhibit hemoglobin S polymerization. In December 2024, the company announced preliminary positive data from its Phase 1/2 clinical trial of BEAM-101, which the company refers to as the BEACON trial. The adult enrollment target for BEACON has been achieved, and the first adolescent patients have cleared screening and enrolled in the trial. The company expects to dose 30 patients in the BEACON trial by mid-2025. The company also plans to present updated data from the trial in mid-2025.
BEAM-302 is a liver-targeting lipid nanoparticle, or LNP, formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype), which is most commonly responsible for severe alpha-1 antitrypsin deficiency, or AATD. AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The company is evaluating BEAM-302 in a Phase 1/2 dose-escalation clinical trial and expects to report initial data from multiple cohorts in the trial in the first half of 2025.
The company is also continuing to advance its other hematology and genetic diseases programs:
Engineered Stem Cell Antibody Paired Evasion, or ESCAPE, is a potentially non-genotoxic approach to HSC transplantation, or HSCT, which combines antibody-based conditioning with multiplex gene edited HSCs. In the company’s ESCAPE program, it expects to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103, an anti-CD117 monoclonal antibody designed to suppress hematopoietic stem and progenitor cells that express CD117, by the end of 2025.
BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most prevalent disease-causing mutation for, and the mutation which results in the most severe form of, glycogen storage disease type 1a, or GSD1a. The company is evaluating BEAM-301 in a Phase 1/2 clinical trial, with patient dosing expected to commence in early 2025.
Base Editing Technology
The company’s proprietary DNA base editors have two principal components that be fused together or incorporated into one another to form a single protein. The first component is a CRISPR associated protein. These proteins have been adapted and engineered to target specific genomic locations in human cells. The targeting ability of the CRISPR protein has been preserved, but the cutting ability has been modified such that the CRISPR protein does not make a double-stranded break in the DNA. The second component of the company’s base editors is a human deaminase, a class of naturally occurring enzymes. The company’s Cytosine Base Editors, or CBEs, and its Adenine Base Editors, or ABEs, each use a different, engineered deaminase, which it has designed to act only on single-stranded DNA.
Base Editing Platform
The company’s unique advantages of base editing – single base editing precision, predictable editing outcome, high editing efficiency, and the avoidance of double-stranded breaks – make it a compelling approach for a wide range of therapeutic applications.
To complement the company’s next-generation gene editing technologies, it also makes significant investments in a suite of delivery technologies designed to deliver gene editing or other nucleic acid payloads to the right cells and enable potentially curative therapy. These delivery technologies include ex vivo modalities, such as electroporation, as well as in vivo modalities, such as LNPs.
In the company’s pipeline, it has initially focus on applications of these technologies where their delivery capabilities have already been clinically validated by third parties, such as ex vivo editing of blood stem cells and LNP delivery to the liver. Longer term, the company is also investing in more innovative delivery options, including through its collaboration with Orbital it has also developed critical enabling capabilities such as mRNA manufacturing and cell processing for autologous and allogeneic cell therapy.
Due to the critical importance of high-quality manufacturing and control of production timing and know-how, it has also established a 100,000 square foot cGMP manufacturing facility in Research Triangle Park, North Carolina. The facility is designed to support manufacturing for the company’s ex vivo cell therapy programs in hematology and in vivo non-viral delivery programs for liver and liver-mediated diseases, with the capability to scale-up to support potential commercial supply. For the company’s initial clinical trials, it is relying primarily on the company’s internal manufacturing capabilities, along with CMOs with relevant manufacturing experience in genetic medicines. The company’s portfolio and capabilities, the probability of technical success of its programs, and the speed at which the company provides potentially life-long cures to patients.
Building an integrated platform combining the company’s gene editing capabilities with advanced delivery and manufacturing capabilities will give the company the flexibility to develop a sustainable portfolio, featuring rapid development of new programs and lifecycle improvements in its core programs.
In addition to the company’s internal pipeline, the breadth and depth of its integrated technology platform gives it the opportunity to create a hub for partnering with other companies, which is an important part of its business model. This model will help the company to unlock the full potential of precision genetic medicine across a wider array of possible applications, including many outside its core areas of focus.
Base Editing Portfolio
The company is advancing multiple programs through clinical development in parallel. The company’s lead programs are focused on sickle cell disease and AATD, and it is also advancing programs in other genetic diseases.
Hematology
The company is advancing hematology base editing programs in which HSCs are collected from a patient, edited using electroporation, a clinically validated technology for the delivery of therapeutic constructs into harvested cells, and then infused back into the patient following a conditioning regimen, such as treatment with busulfan, the standard of care in HSC transplantation today. Once reinfused, the HSCs begin repopulating a portion of the bone marrow in a process known as engraftment. The engrafted, edited HSCs give rise to progenitor cell types with the corrected gene sequences. The company is deploying this ex vivo approach in its BEAM-101 and ESCAPE base editing programs.
The company is pursuing a long-term, staged development strategy for the company’s base editing approach to treat hematological diseases that consists of advancing the company’s lead ex vivo program, BEAM-101, in Wave 1, improving patient conditioning regimens in Wave 2, and enabling in vivo base editing with delivery directly into HSCs of patients via LNPs in Wave 3. This suite of technologies – base editing, improved conditioning and in vivo delivery for editing HSCs – can maximize the potential applicability of the company’s sickle cell disease programs to patients, as well as create a platform for the treatment of many other severe genetic blood disorders.
Wave 1: Ex vivo base editing via autologous transplant with BEAM-101
The company is using base editing to pursue the development of BEAM-101 for the treatment of sickle cell disease. BEAM-101 is a patient-specific, autologous HSC investigational therapy designed to offer a potentially best-in-class profile, incorporating base edits that are intended to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin, or HPFH. The beneficial effects of the fetal form of hemoglobin, or HbF, to compensate for mutations in adult hemoglobin were first identified in individuals with HPFH. Individuals who carry mutations that would have typically caused them to be beta-thalassemia or sickle cell disease patients, but who also have HPFH, are asymptomatic or experience a much milder form of their disease.
BEAM-101 intends to alleviate the effects of sickle cell disease by increasing HbF, which is expected to increase functional hemoglobin production, and in the case of sickle cell disease, inhibit hemoglobin S polymerization.
The company is conducting a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which the company refers to as its BEACON trial. The BEACON trial includes up to 45 patients ages 18 to 35 with severe sickle cell disease who have received prior treatment with at least one disease-modifying agent with inadequate response or intolerance. Following mobilization, conditioning and treatment with BEAM-101, patients are assessed for safety and tolerability, with safety endpoints including neutrophil and platelet engraftment. Patients are also assessed for efficacy, with efficacy endpoints, including the change from baseline in severe vaso-occlusive events, transfusion requirements, HbF levels, and quality of life assessments. The trial’s Data Monitoring Committee and the U.S. Food & Drug Administration have cleared the trial to enroll adolescents from 12 to 17 years old. The adult enrollment target for BEACON has been achieved, and the first adolescent patients have cleared screening and enrolled in the trial. The company expects to dose 30 patients in the BEACON trial by mid-2025. The company also plans to present updated data from the trial in mid-2025.
In December 2024, the company presented initial data from the BEACON trial at the 2024 American Society of Hematology Annual Meeting and Exposition, or ASH. The presentation contained preliminary data as of October 28, 2024 from seven patients in the trial, with follow up ranging from one to 11 months. The presentation data included the following:
All patients achieved endogenous HbF levels exceeding 60% and reduction in corresponding sickle hemoglobin S below 40% that was durable through the data cutoff date. A pancellular distribution of HbF was also observed after the elimination of transfused blood. Total hemoglobin levels increased rapidly with resolution of anemia in all patients after elimination of the transfused blood.
All patients achieved the minimum target cell dose in either 1 or 2 cycles of mobilization (average: 1.4). The mean time to neutrophil engraftment was 17.1 days (range: 15–21), with a low mean duration of neutropenia (6.3 days). The mean time to platelet engraftment was 19.1 days (range: 11–34).
Key markers of hemolysis, including indirect bilirubin, haptoglobin, lactate dehydrogenase and reticulocytes, normalized or improved in all patients following BEAM-101 treatment.
The initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous HSCT. The most common treatment-emergent adverse events were consistent with busulfan conditioning, including febrile neutropenia, stomatitis and anemia. One patient died four months after BEAM-101 infusion due to respiratory failure that was determined by the investigator to be likely related to busulfan conditioning and deemed unrelated to BEAM-101. No vaso-occlusive crises were reported post-engraftment.
Wave 2: Non-genotoxic Conditioning
In parallel with Wave 1 development, the company also intends to improve the transplant conditioning regimen for patients undergoing HSCT, reducing toxicity challenges associated with HSCT standard of care. As a potential alternative to genotoxic conditioning regimens in HSCT, the company is advancing its ESCAPE program. ESCAPE aims to avoid toxicity challenges associated with currently available conditioning regimens for patients with sickle cell disease and beta-thalassemia ahead of autologous HSCT, by combining antibody-based conditioning with multiplex gene edited HSCs. ESCAPE may also have applications in other diseases of the blood and immune system where HSCT could deliver potential benefits but has been limited by toxicities associated with current standard of care conditioning regimens.
The company has nominated a development candidate for its ESCAPE technology consisted of two investigational drug products: BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy that includes the same therapeutic edit as BEAM-101 (editing the HBG1/2 genes to elevate fetal hemoglobin), plus an additional edit to CD117 designed to block binding of BEAM-103, allowing the edited cells to evade suppression by the antibody. The company intends to advance BEAM-103 and BEAM-104 for development in sickle cell disease and beta-thalassemia, potentially building on the same regulatory, manufacturing, clinical and commercial foundations being established with BEAM-101. The company expects to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103 by the end of 2025.
Wave 3: In vivo base editing via HSC-targeted LNPs
The company is also exploring the potential for in vivo base editing programs for sickle cell disease, 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. In preclinical studies, the company achieved in vivo validation of the company’s most potent HSC-directed LNP, demonstrating:
Durable, dose-dependent mRNA transfection in HSCs, resulting in fluorescent reporter expression in more than 40% of cells, maintained out to 16 weeks post-delivery;
Efficient transfection of human CD34+ cells in vitro; and
Efficient transfection of nearly 20% of CD34+ HSCs in humanized mice and NHPs at a dose of 1.0 mg/kg.
Genetic Diseases
The company is using LNPs to advance BEAM-302 and BEAM-301.
BEAM-302: In vivo LNP liver-targeting for AATD
BEAM-302 is a liver-targeting LNP formulation of base editing reagents designed to offer a one-time treatment to correct the E342K point mutation (PiZZ genotype) predominantly responsible for the severe form of AATD. AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the ‘Z’ allele). This point mutation causes Alpha-1 antitrypsin, or AAT, to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the United States have two copies of the Z allele. There are no curative treatments for patients with AATD.
The company is conducting a Phase 1/2 open label, dose escalation clinical trial of BEAM-302 at sites located in the United Kingdom for the treatment of AATD. The trial will evaluate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 initially in patients with AATD-associated lung disease. The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study. The company continues to progress enrollment and global site activation and expect to report initial clinical data from multiple cohorts in the trial in the first half of 2025.
BEAM-301: In vivo LNP liver-targeting for GSD1a
BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most prevalent disease-causing mutation for, and the mutation which results in the most severe form of, GSD1a. GSD1a is an autosomal recessive disorder caused by mutations in the G6PC gene that disrupts a key enzyme, G6Pase, critical for maintaining glucose homeostasis. Inhibition of G6Pase activity results in low fasting blood glucose levels that can result in seizures and be fatal. Patients with this mutation typically require ongoing corn starch administration, without which they may enter into hypoglycemic shock within one to three hours.
The company is conducting a Phase 1/2 clinical trial of BEAM-301 at a select number of sites in the United States. The trial is an open-label, multi-cohort, single-ascending dose evaluation of BEAM-301 for the treatment of GSD1a in patients with the R83C mutation. Key endpoints of the trial include safety and tolerability, time to hypoglycemia during fasting, and changes from baseline in corn starch supplementation. In January 2025, the company activated the first clinical trial site for the Phase 1/2 clinical trial of BEAM-301. The company is continuing site activation activities and expect dosing to commence in early 2025.
The company’s portfolio of precision gene editing technologies
The company has licensed a portfolio of three additional complementary gene editing technologies – prime editing, Cas12b nuclease editing and RNA base editing – for certain fields. Combined with base editing, the company has assembled a broad and versatile portfolio of next generation gene editing technologies for the potential treatment of many severe diseases.
The company has a license to prime editing from Prime Medicine, Inc. Prime editing may be able to achieve the rewriting of short sequences of DNA at a target location. Prime editing utilizes a CRISPR protein to target a mutation site in DNA and to nick a single strand of the target DNA. The guide RNA allows the CRISPR protein to recognize a DNA sequence that is complementary to the guide RNA and also carries a primer for reverse transcription and a replacement template. The reverse transcriptase copies the template sequence in the nicked site, installing the edit. As with base editing, prime editing does not cause double-stranded breaks in the target DNA, resulting in lower insertion and deletion rates than gene editing technologies that rely on double stranded breaks.
The company has the exclusive right to develop prime editing technology for the creation or modification of any single base transition mutations, as well as any edits made for the treatment of sickle cell disease. Transition mutations (i.e., A-to-G, G-to-A, C-to-T, or T-to-C) are the largest single class of disease-associated genetic mutations and include all of the company’s current targets for base editing programs.
The company also has a license agreement with The Broad Institute, Inc., or Broad Institute, that gives the company access to the Cas12b nuclease family, which allows the company to make ‘cut’ edits, which may be appropriate for some applications that require a double stranded break, or to use the general gene targeting ability of Cas12b for other gene editing applications.
The company’s Broad Institute license also gives the company access to RNA base editing technology, a two-part modular system using an RNA-directed CRISPR protein for targeting RNA strands and a deaminase for editing. This CRISPR protein, known as Cas13, is modified so that it cannot break the RNA strand, and is fused to a deaminase capable of making a single base edit at a specific target location within the RNA strand.
Collaborations
The company’s collection of base editing, gene editing and delivery technologies has significant potential across a broad array of genetic diseases. To fully realize this potential, the company has established and plans to continue to seek out innovative collaborations, licenses, and strategic alliances with pioneering companies and with leading academic and research institutions. Additionally, the company has and intends to continue to pursue relationships that potentially allow the company to accelerate its preclinical research and development efforts.
Pfizer
In December 2021, the company entered into a four-year research collaboration agreement with Pfizer Inc., or Pfizer, focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. Under the terms of the agreement, the company will conduct all research activities through development candidate selection for three pre-specified, undisclosed targets. 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 development candidate. The company has a right to opt in, at the end of Phase 1/2 clinical trials, 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 the company and Pfizer would share net profits as well as development and commercialization costs in a 35%/65% ratio (Beam/Pfizer).
Apellis Pharmaceuticals
In June 2021, the company entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of the company’s base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, the company will conduct preclinical research on six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis has an exclusive option to license any or all of the six programs and will assume responsibility for subsequent development. The company may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.
Verve Therapeutics and Eli Lilly and Company
In April 2019, the company entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, a company focused on gene editing for cardiovascular disease treatments, and in July 2022, the company and Verve amended the Verve Agreement. Under the terms of the Verve Agreement, as amended, the company granted Verve exclusive worldwide licenses under certain of the company’s editing technologies for human therapeutic applications against a total of three liver-mediated, cardiovascular disease targets, including use of the company’s base editing technology for each of these targets and use of certain of the company’s gene editing technology for two of such targets. In exchange, the company received shares of Verve common stock. In October 2023, the company entered into a transfer and delegation agreement, or the Lilly Agreement, with Eli Lilly and Company, or Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement, including the company’s opt-in rights to co-develop and co-commercialize each of Verve’s base editing programs for cardiovascular disease, which consist of programs targeting PCSK9, ANGPTL3 and an undisclosed liver-mediated, cardiovascular target. In addition, Lilly acquired the right to receive any future milestone or royalty payments payable to the company under the Verve Agreement.
Sana Biotechnology
In October 2021, the company entered into an option and license agreement, or the Sana Agreement, with Sana Biotechnology, Inc., or Sana, pursuant to which the company granted Sana non-exclusive research and development and commercial rights to the company’s CRISPR Cas12b technology to perform nuclease editing for certain ex vivo engineered cell therapy programs. Under the terms of the Sana Agreement, licensed products include certain specified allogeneic T cell and stem cell-derived products directed at specified genetic targets, with certain limited rights for Sana to add and substitute such products and targets. The Sana Agreement excludes the grant of any Beam-controlled rights to perform base editing. Sana is conducting a first-in-human trial of SC291, its CD19-targeted allogeneic CAR-T cell therapy, in patients with various B-cell mediated autoimmune diseases. Sana is also conducting a first-in-human trial of SC262, its CD22-directed allogeneic CAR-T cell therapy, in patients with relapsed or refractory B-cell malignancies.
Orbital Therapeutics
In September 2022, the company entered into a license and research collaboration agreement, or the Orbital Agreement, with Orbital, pursuant to which each of the company granted the other licenses to certain technology controlled during the three years after entry into the Orbital Agreement that are necessary or reasonably useful for the non-viral delivery or the design or manufacture of RNA for the prevention, treatment or diagnosis of human disease. The company’s license to Orbital is for all fields other than its exclusive field and also excludes the targets and substantially all of the indications that are the subject of the company’s existing programs. The company’s exclusive field consists of all products and biologics that function in the process of gene editing or conditioning for use in cell transplantation, or that act in combination with any such products or biologics. Orbital’s license to the company is for all fields other than Orbital’s exclusive field. Orbital’s exclusive field consists of products and biologics that function as vaccines and also of therapeutic proteins, other than therapeutic proteins (i) that use gene editing, (ii) for use in conditioning, (iii) for use in regenerative medicine, (iv) for use as a CAR immune therapy, including CAR-T, CAR-NK and CAR-macrophage compositions, (v) for use as a T-cell receptor therapy or (vi) that modulate certain immune responses. The licenses are exclusive in each party’s exclusive field for three years and non-exclusive in those fields thereafter. The company and Orbital agreed that for a period of three years after entry into the Orbital Agreement, subject to limited exceptions, the company would not research, develop and commercialize, or grant licenses to research, develop and commercialize, products or biologics within the other party’s exclusive field.
Prime Medicine
In September 2019, the company entered into a collaboration and license agreement with Prime Medicine to research and develop a novel gene editing technology developed by one of the company’s founders. Under the terms of the agreement, the company granted Prime Medicine a non-exclusive license to certain of its CRISPR technology (including Cas12b), delivery technology and certain other technology controlled by the company to develop and commercialize gene editing products for the treatment of human diseases. Prime Medicine granted the company an exclusive license to develop and commercialize prime gene editing technology for the creation or modification of any single base transition mutations, as well as any edits made for the treatment of sickle cell disease.
Competition
Within the disease areas that the company focuses on, the company is also aware of competing companies that have approved therapies, those with therapies in development, and others that may emerge in the future. For sickle cell disease, these companies include CRISPR Therapeutics, Vertex Pharmaceuticals, bluebird bio, Novartis Pharmaceuticals, Kamau Therapeutics, Fulcrum Therapeutics, Tessera Therapeutics, Cimeio Therapeutics and Agios Pharmaceuticals. For the company’s AATD targeted therapies, these include Wave Life Sciences, Moderna, Korro Bio, Tessera Therapeutics and Arrowhead Pharmaceuticals.
Intellectual Property
The company’s wholly owned and its in-licensed patents and patent applications cover various aspects of its base editing platform and its programs, including: C-to-T DNA base editors; A-to-G DNA base editors; A-to-I RNA base editors, or REPAIR; C-to-U RNA base editors, or RESCUE; Dual editing C-to-T and A-to-G DNA base editors; CRISPR/Cas12b systems for nuclease editing; Novel guide RNA sequences; Systems and methods for increasing the specificity of base editing; Multiplex base editing in immune cells ex vivo; Methods for evaluating base editing specificity; Therapeutic methods; and Delivery modality.
The company also has an option to license patents and patent applications relating to CRISPR/Cas9 systems. The company intends to continue to pursue, when possible, additional patent protection, including composition of matter, method of use, and process claims, directed to each component of the company’s platform technology and the programs in the company’s portfolio. As of December 31, 2024, the company’s wholly-owned patent portfolio consisted of eight issued U.S. patents, and 21 issued patents in jurisdictions outside the United States. The company also has approximately 539 pending patent applications, including PCT applications, provisional patent applications and counterparts to the foregoing U.S. and foreign patents. In addition, Beam co-owns one issued U.S. patent and one issued patent in a jurisdiction outside the United States. The company also has approximately 26 pending patent applications between the Broad Institute, Inc., UCL Business, Ltd., and Apellis Pharmaceuticals, Inc. The patents and patent applications outside of the United States were filed in numerous jurisdictions, including Australia, Brazil, Canada, China, Europe, Hong Kong, India, Japan, Korea, Singapore and South Africa. Many of the company’s owned patents and patent applications are related to the company’s DNA base editing technology, including claims to base editor variants with enhanced activities or novel properties, methods of using such base editors, methods of using such base editors for therapeutic indications, multiplex base editing in immune cells ex vivo, guide RNAs that target base editors to therapeutically relevant DNA sequences, and methods for evaluating base editing specificity. Certain of the company’s owned patents and patent applications are related to viral and non-viral delivery technologies. If issued as U.S. patents, and if the appropriate maintenance fees are paid, the U.S. patents would be expected to expire between 2039 and 2045, excluding any additional term for patent term adjustments or patent term extensions.
As of December 31, 2024, the company’s in-licensed patent portfolio consisted of approximately 64 issued U.S. patents, and approximately 265 issued patents in jurisdictions outside the United States. The company also has approximately 350 pending patent applications, including PCT applications, provisional patent applications and counterparts to the foregoing U.S. and foreign patents. The patents and patent applications outside of the United States were filed in numerous jurisdictions, including Australia, Canada, China, Europe, Hong Kong, India, Israel, Japan, Korea, New Zealand, Russia and Singapore. The patents and applications from the company’s in-licensed portfolio for DNA base editing include claims to novel base editors, claims to engineered deaminase enzymes (e.g., evolved TadA) used in the base editors, compositions including the base editor or engineered deaminase as a component, methods of using such base editors, including methods of using such base editors for therapeutic indications, and guide RNAs that target base editors to therapeutically relevant DNA sequences. The in-licensed patents and applications also cover various aspects related to the platform technology, including base editing systems that employ S. pyogenes Cas9, S. aureus Cas9, Cas9 PAM variants, inactive forms of Cas9, and/or Cas9 nickases, and systems for delivery of base editors. The patents and applications from the company’s in-licensed portfolio for RNA base editing include claims to novel base editors, compositions including the base editor as a component, guide RNAs that target base editors to therapeutically relevant RNA sequences, and methods of using such base editors, including methods of using such base editors for therapeutic indications. The patents and applications from the company’s in-licensed portfolio for Cas12b editing include claims to methods of using Cas12b to modify DNA (e.g., nuclease cleavage of DNA) and engineered and/or non-naturally occurring compositions including Cas12b as a component. The patents and applications from the company’s in-licensed portfolio for delivery technologies include claims to novel lipid-based delivery systems and compositions, viral-based delivery systems and compositions, and methods of using such systems and compositions to deliver base editors. The patents and applications from the company’s in-licensed portfolio for the balance of the company’s platform include claims to compositions and methods for delivery of charged base editor proteins into cells, modification and improvements to the base editing systems including improvements to the nucleotide binding protein component, guide RNA component and base editing enzyme component of the base editing complex, methods for evaluating gene targeting and base editing efficiency and compositions and methods for prime editing. The company’s in-licensed patents and patent applications, if the appropriate maintenance fees are paid, are expected to expire between 2034 and 2040, excluding any additional term for patent term adjustments or patent term extensions (or the corresponding foreign equivalent).
The company also has a nonexclusive license to conduct research activities and an option to exclusively license certain patents and patent applications directed to Cas9 and Cas12a from Editas, who in turn has licensed such patents from various academic institutions. In the case of Cas9, a number of the U.S. patents are subject to an interference declared by the Patent and Trademark office, and a number of the European patents are the subject of one or more oppositions.
As of December 31, 2024, the company’s registered trademark portfolio contained approximately 33 registered/allowed trademarks and pending trademark applications in the United States and in certain overseas jurisdictions.
Intellectual Property Licenses
License Agreement with The President and Fellows of Harvard College
In June 2017, the company entered into a license agreement with Harvard, as amended, or the Harvard License Agreement, pursuant to which it received an exclusive, worldwide, royalty-bearing, sublicensable license under certain patent rights owned or controlled by Harvard to make, have made, offer for sale, sell, have sold and import licensed products in the field of the prevention or treatment of any and all human diseases and conditions, excluding human germline modification and products for non-human animal and plant applications. The company also received a non-exclusive, worldwide, royalty-bearing, sublicensable license to research, have researched, develop and have developed ‘enabled’ products related to the Harvard patent rights which are not licensed products.
License Agreement with Editas Medicine, Inc.
The company has a license agreement, or the Editas License Agreement, with Editas pursuant to which it received an exclusive (even as to Editas), royalty-bearing, sublicensable, worldwide license under certain patent rights owned or controlled by Editas related to certain base editing technologies and CRISPR technology to develop, commercialize, make, have made, use, offer for sale, sell and import certain base editing products for the treatment of human diseases or conditions. The license the company received is non-exclusive with respect to certain specified targets. The company’s licensed field excludes the use of certain gene editing technologies in certain fields of use that have already been licensed to other partners of Editas, provided that its licensed field expand if the fields licensed to other Editas partners are reduced or are otherwise modified as a result of any termination, expiration, or amendment to Editas’ agreements with such partners. In addition, the company received a royalty-free, non-sublicensable, non-exclusive license under a separate set of patent rights owned or controlled by Editas to conduct research activities in its licensed field and for which the company has an option to obtain an exclusive license from Editas.
License Agreement with The Broad Institute, Inc.
The company's affiliate, Blink Therapeutics Inc., or Blink, has a license agreement, as amended, or the Broad License Agreement, with Broad Institute. In 2021, Blink merged with and into Beam, such that Blink's separate corporate existence ceased, and Beam continued as the surviving corporation and the successor by merger to the Broad License Agreement with respect to Blink. Under the Broad License Agreement, and as further detailed below, the company received certain rights to RNA base editing technology, including the RNA editor platforms RESCUE and REPAIR, which use Cas13 linked to a deaminase to deliver single base A-to-I or C-to-U editing of RNA transcripts, respectively, as well as the Cas12b nuclease family of gene editing enzymes.
Under the Broad License Agreement, the company has also been granted a non-exclusive, royalty-bearing and sublicensable license under all patents exclusively licensed to it under the Broad License Agreement to make, have made, offer for sale, sell, have sold and import certain products in its field that were made, discovered, developed or determined to have utility through the use of such patents in a research or discovery program commencing before May 2021 or through the use of transferred materials from Broad Institute but that are not covered by the licensed patents and a non-exclusive, worldwide, royalty-bearing and sublicensable internal research license under all patents exclusively licensed to it.
The company is permitted to sublicense the licensed patents to affiliates and third parties, provided that any such sublicense agreement must remain in compliance with and be consistent with the terms of the Broad License Agreement. In addition, any such sublicense agreement must include certain customary provisions to ensure the company’s ability to comply with the Broad License Agreement. The company is also responsible for any breaches of a sublicense agreement by the applicable sublicensee and are responsible for all payments due under the Broad License Agreement by operation of any such sublicense.
License agreement with Bio Palette Co., Ltd.
The company has a license agreement, or the Bio Palette License Agreement, with Bio Palette Co., Ltd., or Bio Palette, pursuant to which it received an exclusive (even as to Bio Palette and its affiliates), sublicensable license under certain patent rights related to base editing owned or controlled by Bio Palette to research, make, have made, import, export, distribute, use, have used, sell, have sold or offer for sale, and otherwise exploit products for the treatment of human disease throughout the world, but excluding products in the microbiome field in Asia. In addition, the company granted Bio Palette an exclusive license under certain patent rights related to base editing and gene editing owned or controlled by it to research, make, have made, import, export, distribute, use, have used, sell, have sold or offer for sale, and otherwise exploit products in the microbiome field in Asia, subject to the company’s right, in its sole discretion, to expand Bio Palette’s license (and the applicable royalty obligations) to the entire territory. Each party to the Bio Palette Agreement retains non-exclusive rights to develop and manufacture products in the microbiome field worldwide for the sole purpose of exploiting those products in its own territory.
As part of the Bio Palette License Agreement, if the company’s form a Scientific Advisory Board, then Bio Palette has the right to appoint two representatives to such board until the conclusion of the period ending five years after the effective date of the Bio Palette License Agreement. Additionally, the company and Bio Palette agree to communicate with each other regarding potential base editing collaborations in Japan.
Research and Development
The company’s research and development expenses were $367.6 million for the year ended December 31, 2024.
Government regulation
In the United States, the company’s candidate products are regulated as biological products, or biologics, under the Public Health Service Act, or the PHSA, and the Federal Food, Drug and Cosmetic Act, or the FDCA, the implementing regulations of the FDA and other federal, state and local statutes and regulations.
History
Beam Therapeutics Inc. was founded in 2017. The company was incorporated in 2017 as a Delaware corporation.
