Key PointsBeam Therapeutics develops precision gene-editing and genetic medication for severe sickle cell disease (SCD).Beam Therapeutics performs a precision form of gene-editing called base-editing using CRISPR/Cas to make a single letter change in the DNA.Prime Medicine pioneered its Prime Editor platform to perform prime-editing, an alternative to CRISPR/Cas9, which allows for the precise replacement of a specific DNA sequence without causing insertion and deletions (indels).5 stocks we like better than ...

The promise of gene editing is moving closer to being a reality. If you’re an investor with a speculative appetite and appropriate risk tolerance, you may want to consider a long position in gene editing stocks. The idea at the core of gene editing is personalized medicine. Tools like 23-and-Me can help you understand your DNA. While knowledge is power, in the case of your personal health, that knowledge has limitations. After all, knowing that you are more genetically predisposed to a certain disease is li ...

Shares of Beam Therapeutics Inc. (BEAM) have rallied 27.3% in the past three months compared with the industry’s increase of 9.8%.Beam’s pipeline of gene editing therapies is based on its proprietary base editing technology. The company is advancing its base editing technology across three disease area portfolios, namely, hematology, immunology/oncology and genetic diseases.The company is developing its leading ex-vivo genome-editing candidate, BEAM-101, for the treatment of sickle cell disease (SCD). The c ...

CAMBRIDGE, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that management will present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 11:15 a.m. PT in San Francisco. 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 The ...

Beam Therapeutics Inc. (Exact name of Registrant as specified in its Charter) ☐ Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 | --- | --- | --- | |-----------------------------------------|-------------------|-----------------------------------------------------| | Title of each class | Trading Symbol(s) | Name of each exchange on which registered | | Common Stock, par value $0.01 per share | BEAM | Nasdaq Global Select Market | This Quarterly Report on Form 10-Q ...

[PART I Financial Information](index=5&type=section&id=PART%20I%20Financial%20Information) [Item 1. Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for Beam Therapeutics Inc. as of June 30, 2023, including balance sheets, statements of operations, statements of stockholders' equity, and statements of cash flows, along with detailed notes [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets increased slightly to **$1.35 billion** as of June 30, 2023, from **$1.34 billion** at year-end 2022, primarily due to increased additional paid-in capital offsetting the accumulated deficit, with cash and marketable securities remaining the largest asset components at approximately **$1.1 billion** Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $225,544 | $232,767 | | Marketable securities | $847,472 | $845,367 | | Total current assets | $1,094,805 | $1,092,896 | | **Total assets** | **$1,353,887** | **$1,341,714** | | **Liabilities & Equity** | | | | Total current liabilities | $209,536 | $223,595 | | Total liabilities | $542,244 | $608,240 | | Accumulated deficit | $(1,236,598) | $(1,057,362) | | **Total stockholders' equity** | **$811,643** | **$733,474** | [Condensed Consolidated Statements of Operations and Other Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Other%20Comprehensive%20Loss) The company reported a net loss of **$179.2 million** for the six months ended June 30, 2023, an increase from **$141.2 million** in the prior year, driven by higher research and development expenses which rose to **$197.3 million** Statement of Operations Highlights (in thousands, except per share data) | Metric | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | License and collaboration revenue | $44,324 | $25,084 | | Research and development | $197,254 | $139,966 | | General and administrative | $48,146 | $43,309 | | Loss from operations | $(201,076) | $(158,191) | | **Net loss** | **$(179,236)** | **$(141,164)** | | **Net loss per share, basic and diluted** | **$(2.41)** | **$(2.03)** | [Condensed Consolidated Statements of Stockholders' Equity](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity) Total stockholders' equity increased from **$733.5 million** at December 31, 2022, to **$811.6 million** at June 30, 2023, primarily due to proceeds from the At-the-Market (ATM) stock offering and other stock-based activities, which offset the **$179.2 million** net loss Changes in Stockholders' Equity (Six Months Ended June 30, 2023, in thousands) | Description | Amount | | :--- | :--- | | Balance at December 31, 2022 | $733,474 | | Issuance of common stock from ATM offering, net | $200,993 | | Stock-based compensation | $50,195 | | Net loss | $(179,236) | | **Balance at June 30, 2023** | **$811,643** | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities was **$194.5 million** for the six months ended June 30, 2023, while financing activities provided **$206.0 million**, resulting in a net decrease in cash, cash equivalents, and restricted cash of **$4.6 million** Cash Flow Summary (Six Months Ended June 30, in thousands) | Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash provided by (used in) operating activities | $(194,484) | $166,214 | | Net cash provided by (used in) investing activities | $(16,029) | $(538,601) | | Net cash provided by (used in) financing activities | $205,958 | $78,016 | | **Net change in cash, cash equivalents and restricted cash** | **$(4,555)** | **$(294,371)** | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes provide detailed information on the company's business, liquidity, accounting policies, fair value measurements, marketable securities, accrued liabilities including a legal contingency, significant license and collaboration agreements, common stock transactions, and stock-based compensation plans - The company expects its cash, cash equivalents, and marketable securities of **$1.1 billion** as of June 30, 2023, to be sufficient to fund operations for at least the next 12 months[217](index=217&type=chunk) - The company has accrued a **$3.4 million** liability related to a dispute with a research institution over an alleged breach of a confidentiality agreement, which the institution has rejected[2](index=2&type=chunk) Revenue Recognition from Key Collaborations (Six Months Ended June 30, 2023, in millions) | Agreement | Revenue Recognized | | :--- | :--- | | Pfizer Agreement | $26.3 | | Apellis Agreement | $12.2 | | Orbital Agreement | $4.2 | - As of June 30, 2023, the company has sold an additional **9,727,417 shares** under its amended At-the-Market (ATM) sales agreement for aggregate gross proceeds of **$528.2 million**[17](index=17&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's business strategy, clinical pipeline progress, financial results, and liquidity, highlighting advancements in BEAM-101 and BEAM-201 trials, plans for BEAM-301 and BEAM-302 regulatory submissions, and the significant increase in R&D expenses, while confirming a cash runway of at least 12 months [Overview and Pipeline](index=25&type=section&id=Overview%20and%20Pipeline) Beam is a biotechnology company focused on precision genetic medicines using proprietary base editing technology, advancing a diversified portfolio across hematology, immunology/oncology, and genetic diseases, with key programs including BEAM-101 and BEAM-201 in clinical trials and regulatory submissions planned for BEAM-302 and BEAM-301 - BEACON trial for BEAM-101 (sickle cell disease) is ongoing, with data from multiple patients planned for release in 2024[77](index=77&type=chunk)[97](index=97&type=chunk) - The first patient in the Phase 1/2 trial for BEAM-201 (T-ALL/T-LL) is expected to be dosed in Q3 2023[132](index=132&type=chunk) - The company plans to submit a regulatory application for BEAM-302 (AATD) in Q1 2024 and for BEAM-301 (GSDIa) in H1 2024 to initiate clinical trials[103](index=103&type=chunk)[102](index=102&type=chunk) - The company's **100,000 sq. ft.** manufacturing facility in North Carolina became operational in Q1 2023 and is expected to initiate cGMP operations in late 2023[106](index=106&type=chunk) [Collaborations](index=28&type=section&id=Collaborations) The company leverages strategic collaborations to expand its base editing technology, including partnerships with Pfizer, Apellis, Verve, Sana, and Orbital, which provide upfront payments, potential milestones, and royalties, contributing to the company's revenue stream - Collaboration with Pfizer focuses on in vivo base editing for three rare genetic disease targets in the liver, muscle, and central nervous system[108](index=108&type=chunk) - Collaboration with Apellis focuses on discovering new treatments for complement system-driven diseases across six preclinical programs[109](index=109&type=chunk) - Collaboration with Verve Therapeutics granted Verve exclusive licenses for base editing technology against three liver-mediated cardiovascular disease targets, with Verve's lead product, VERVE-101, currently in a global Phase 1b clinical trial[110](index=110&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) The company's financial results show a significant year-over-year increase in operating expenses, primarily due to higher R&D costs, which rose by **$57.3 million** to **$197.3 million** for the six months ended June 30, 2023, driven by clinical trial costs, IND-enabling studies, and increased personnel, while license and collaboration revenue grew to **$44.3 million** Comparison of Operating Expenses (Six Months Ended June 30, in thousands) | Expense Category | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | Research and development | $197,254 | $139,966 | $57,288 | | General and administrative | $48,146 | $43,309 | $4,837 | | **Total operating expenses** | **$245,400** | **$183,275** | **$62,125** | - The **$57.3 million** increase in R&D expenses for the first half of 2023 was primarily due to a **$30.9 million** increase in external costs (manufacturing, clinical expenses for BEAM-101/201, IND-enabling studies for BEAM-301/302) and a **$14.4 million** increase in employee-related expenses due to headcount growth[179](index=179&type=chunk)[125](index=125&type=chunk) [Liquidity and Capital Resources](index=36&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2023, Beam had **$1.1 billion** in cash, cash equivalents, and marketable securities, primarily funded through equity offerings, including an amended At-the-Market (ATM) sales agreement allowing for the sale of up to an additional **$800.0 million** in common stock, which management believes is sufficient to fund operations for at least the next 12 months - The company had **$1.1 billion** in cash, cash equivalents, and marketable securities as of June 30, 2023[159](index=159&type=chunk) - The company received a **$300.0 million** upfront payment from Pfizer in January 2022 and a **$25.0 million** payment from Apellis in June 2022[159](index=159&type=chunk) - The company expects its current cash position will fund operating expenses and capital expenditures for at least the next 12 months from the report's issuance date[186](index=186&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk exposure is interest rate sensitivity on its **$1.1 billion** portfolio of cash, cash equivalents, and marketable securities, though due to the short-term duration and low-risk profile of these investments, a 10% change in interest rates is not expected to have a material effect, and the company has minimal foreign currency exchange or inflation risk - The company's main market risk is interest rate changes affecting its **$1.1 billion** in cash and investments[166](index=166&type=chunk) - Due to the short-term nature of its investment portfolio, a **10%** change in interest rates is not expected to have a material impact on the portfolio's fair market value[166](index=166&type=chunk) [Item 4. Controls and Procedures](index=40&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of June 30, 2023, with no material changes in internal control over financial reporting during the quarter - The Principal Executive Officer and Principal Financial Officer concluded that the company's disclosure controls and procedures were effective as of June 30, 2023[167](index=167&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended June 30, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[167](index=167&type=chunk) [PART II Other Information](index=41&type=section&id=PART%20II%20Other%20Information) [Item 1. Legal Proceedings](index=41&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently subject to any material legal proceedings, though Note 6 of the financial statements describes a pre-litigation dispute with a research institution - The company states it is not currently subject to any material legal proceedings[197](index=197&type=chunk) [Item 1A. Risk Factors](index=41&type=section&id=Item%201A.%20Risk%20Factors) This section highlights key risks to the company's business, including the dynamic and litigious intellectual property landscape for gene editing, reliance on third-party manufacturers for critical supplies (citing a recent DOJ subpoena of a key NHP supplier), and potential difficulties in enrolling patients in clinical trials for rare diseases - The intellectual property landscape for gene editing is highly dynamic, and the company faces risk of infringement lawsuits from third parties, which could be costly and delay development[168](index=168&type=chunk) - The company relies on third parties for manufacturing and supply, which increases risks of delays and cost overruns, and a key supplier of non-human primates (NHPs), Charles River, received a DOJ subpoena regarding NHP importation, which could affect preclinical development[199](index=199&type=chunk) - The company may face challenges in enrolling patients for clinical trials, particularly for rare diseases, which could delay or prevent regulatory approvals and increase development costs[170](index=170&type=chunk)[200](index=200&type=chunk) [Item 5. Other Information](index=46&type=section&id=Item%205.%20Other%20Information) This section discloses the adoption of Rule 10b5-1 trading arrangements by several of the company's directors and officers, including the CEO, CFO, President, Chief Legal Officer, and Chief Medical Officer, to facilitate the orderly sale of company securities, including sales to cover tax withholding obligations on vesting restricted stock units (RSUs) - Several executives, including CEO John Evans, CFO Terry-Ann Burrell, and President Giuseppe Ciaramella, adopted Rule 10b5-1 trading plans during the quarter[228](index=228&type=chunk) - The plans include arrangements for the sale of shares to cover tax withholding obligations upon the vesting of RSU equity awards[238](index=238&type=chunk) [Item 6. Exhibits](index=48&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the quarterly report, including an amendment to the Sales Agreement with Jefferies LLC, certifications from the CEO and CFO as required by the Sarbanes-Oxley Act, and XBRL data files - Filed exhibits include Amendment No. 2 to the At-the-Market (ATM) Sales Agreement with Jefferies LLC, dated May 10, 2023[232](index=232&type=chunk) - Certifications by the Principal Executive Officer and Principal Financial Officer pursuant to Sarbanes-Oxley Sections 302 and 906 are included as exhibits[232](index=232&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Delaware 81-5238376 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 238 Main Street Cambridge, MA 02142 (Address of principal executive of ices) (Zip Code) Registrant's telephone number, including area code: (857) 327-8775 Securities registered pursuant to Section 12(b) of the Act: (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For ...

Expenses and Compensation - Research and development expenses increased by $25.4 million due to additional stock-based compensation from more stock options and restricted stock units granted to employees[170] - General and administrative expenses increased by $30.6 million, primarily due to a $15.4 million increase in stock-based compensation and a $10.9 million increase in personnel-related costs[171] Income and Investments - The company recorded $23.9 million of other income related to the change in fair value of success payment liabilities, compared to $1.0 million of expense in the previous year[174] - Other income from non-controlling equity investments was $20.2 million, driven by changes in the value of investments in Prime and Verve[174] - The company recorded $18.9 million of other income related to the change in fair value of contingent consideration liabilities due to updated project timelines[174] - Interest and other income increased to $15.3 million, primarily due to higher interest income from increased market rates[174] Revenue and Payments - The company received $300.0 million in upfront payment from the Pfizer Agreement for in vivo base editing programs[178] - The company sold 3,908,289 additional shares under the amended Sales Agreement, generating $322.0 million in gross proceeds[178] - The company received $25.0 million as the First Anniversary Payment under the Apellis Agreement in June 2022[178] - Net cash provided by operating activities for the year ended December 31, 2022 was $22.5 million, primarily driven by the collection of collaboration receivables of $300.0 million related to the Pfizer Agreement[180] - Net cash used in operating activities for the year ended December 31, 2021 was $66.3 million, primarily due to a net loss of $370.6 million and an increase in collaboration receivable of $300.0 million[180] Future Financial Commitments - The company may owe Harvard and Broad Institute success payments of up to an additional $90.0 million each, contingent upon future events[185] - The company agreed to pay Guide's former stockholders and optionholders up to an additional $100.0 million in technology milestone payments and $220.0 million in product milestone payments, payable in common stock[185] - The company leases certain assets with aggregate future minimum commitments of $268.0 million for office and laboratory leases and $2.5 million for equipment leases as of December 31, 2022[185] - The company may be required to make milestone payments to former stockholders and optionholders of Guide in the form of common stock based on the achievement of certain product and technology milestones, with payments accounted for under ASC 480 and contingent consideration liabilities carried at fair value estimated using a probability-based model[190] - The estimated fair value of contingent consideration liabilities is determined using probability-adjusted discounted cash flow models, with significant changes in probabilities of success or milestone timing potentially resulting in significantly higher or lower fair value measurements[190] Cash and Investments - As of December 31, 2022, the company had $1.1 billion in cash, cash equivalents, and marketable securities[179] - The company had cash, cash equivalents, and marketable securities totaling $1.1 billion as of December 31, 2022, with primary market risk exposure related to interest rate sensitivity[196] - The company believes an immediate 10% change in interest rates would not have a material effect on the fair market value of its investment portfolio due to the short-term duration and low-risk profile of its investments[196] - The company is not currently exposed to significant foreign currency exchange rate risk but may face increased risk from future contracts with vendors located outside the United States[196] - Inflation has not had a material effect on the company's financial statements, but future operations may be adversely affected by rising labor, research, manufacturing, and development costs[196] Leases and Accounting - The company assesses whether contracts entered into on or after the effective date contain a lease based on criteria such as the use of a distinct identified asset, the right to economic benefits, and the right to direct the use of the asset[194] - Leases are classified as either finance leases or operating leases, with finance leases meeting specific criteria such as transfer of ownership, purchase options, or lease term covering a major part of the asset's useful life[195] Future Financial Needs - The company expects to finance its cash needs through equity offerings, debt financings, collaborations, strategic alliances, and licensing arrangements, as it does not have any committed external source of capital[184] - The company's operating expenses are expected to increase substantially as it advances clinical trials, research programs, and preclinical development of product candidates[183] Investments and Write-Downs - The company's investment in Orbital has been written down to zero as of December 31, 2022, under the equity method of accounting[189]

| --- | --- | |-------|-------| | | | | | | | | | | | | Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding: the initiation, timing, progress and results of preclinical studies and research and development programs, including the initiation and progress of clinical trials, including our BEACON trial and our BEAM-201 tr ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number 001-39208 Beam Therapeutics Inc. (Exact name of Registrant as specified in its Charter) Delaware 81-5238376 (State or other juri ...