Company Overview - Beam is focused on next-generation gene editing utilizing CRISPR tools, specifically through a technology called base editing, which allows for precise genome targeting and editing without double-stranded breaks [2]. Key Programs - The company has two major franchises, one of which is in hematology, targeting sickle cell disease with its lead program BEAM-101, which is considered a best-in-class option for patients suffering from this condition [3]. - The next-generation versions of the program aim to provide functional cures in more accessible ways, including non-genotoxic conditioning and in vivo delivery directly to the bone marrow [3].

Summary of Beam Therapeutics Conference Call Company Overview - Beam Therapeutics is focused on next-generation gene editing using CRISPR tools, specifically base editing, which allows precise genome targeting without double-stranded breaks [4][5] Key Programs - **Hematology Franchise**: - Lead program BIM-101 targets severe sickle cell disease, aiming to provide a best-in-class option for patients [5] - Future versions aim to deliver functional cures with less invasive methods, avoiding chemotherapy [5] - **Liver Franchise**: - Lead program BEAM-302 targets alpha-one antitrypsin deficiency, showing strong evidence of correcting the causative mutation [6][8] - Additional programs include MUM-301 for glycogen storage disease and other liver-targeted genetic medicine programs [6] Financial Position - The company is well-financed with $1.2 billion in cash, sufficient to support its pipeline through significant milestones until 2028 [6] Clinical Data and Efficacy - **BEAM-302**: - Initial Phase I trial data showed a dose response with total alpha-one levels reaching approximately 12.4 micromolar, with 90% being the corrected form (M) and only 10% being the toxic form (Z) [12][13] - The Z protein was reduced by about 80%, indicating a significant therapeutic correction [13] - The treatment is designed to be a one-time durable correction, potentially providing lifelong benefits [16] Safety and Tolerability - The LNP delivery system and base editing have shown good safety profiles, with no significant adverse effects reported [15][20] - Ongoing studies will further explore dosing and safety in patients with liver involvement [31] Future Development Plans - The company plans to explore higher single doses and multi-dose cohorts to optimize efficacy while maintaining safety [18][20] - An update on data and future steps is expected in early 2026 [21][80] Competitive Landscape - Beam Therapeutics positions BEAM-302 as a potentially best-in-class option compared to augmentation therapies and RNA editing approaches, emphasizing its one-time, permanent solution [56][57] - The company acknowledges the presence of competitors but believes its approach offers significant advantages in terms of efficacy and durability [60] Market Considerations - The market for sickle cell disease treatments is complex, with reimbursement processes still being established [84][86] - Beam Therapeutics is optimistic about its market entry, drawing parallels to the CAR T-cell therapy market's evolution [88] Patient Demand and Community Response - There is strong demand from patients for a one-time curative approach, especially given the severity of diseases like alpha-one antitrypsin deficiency [69][70] - The company is actively working to identify and educate more patients about genetic testing and treatment options [72] Regulatory Pathway - Beam Therapeutics is considering accelerated approval pathways due to the clear therapeutic signal from its data [77][79] - The company is preparing for potential regulatory discussions to expedite patient access to its therapies [79] Conclusion - Beam Therapeutics is making significant progress in gene editing therapies, with promising clinical data and a strong financial position to support its innovative programs aimed at treating severe genetic diseases [99]

Group 1 - Beam Therapeutics Inc. is a biotechnology company focused on developing precision genetic medicines through base editing technology [2] - The company has a fully integrated platform that includes gene editing, delivery, and internal manufacturing capabilities [2] - Beam's proprietary base editing technology allows for precise and efficient single base changes in targeted genomic sequences without causing double-stranded breaks in DNA [2] Group 2 - Management will participate in several upcoming investor conferences, including Citi's 2025 Biopharma Back to School Conference and the Wells Fargo Healthcare Conference [3] - The live webcasts of these events will be available on the company's website and archived for 60 days [1]

Core Insights - Beam Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, a genetically modified cell therapy aimed at treating sickle cell disease (SCD) [1][2] - The RMAT designation follows an earlier orphan drug designation and is expected to facilitate collaboration with the FDA as the company progresses towards a Biologics License Application (BLA) filing [2][4] - The BEACON Phase 1/2 trial has dosed 30 patients, with promising clinical data indicating robust increases in fetal hemoglobin and reductions in sickle hemoglobin [3][5] Company Overview - Beam Therapeutics is focused on developing precision genetic medicines using its proprietary base editing technology, which allows for precise genetic modifications without double-stranded breaks in DNA [7][8] - The company aims to create a fully integrated platform for gene editing, delivery, and manufacturing to provide long-term cures for serious diseases [7][8] Product Details - BEAM-101 is an investigational therapy that utilizes autologous CD34+ hematopoietic stem and progenitor cells, which are base-edited to enhance fetal hemoglobin production [5] - The therapy is designed to mimic naturally occurring variants that lead to increased fetal hemoglobin, potentially alleviating symptoms of SCD [5][6] Clinical Trial Insights - Updated clinical data from the BEACON trial presented at the EHA 2025 Congress showed durable increases in fetal hemoglobin and no vaso-occlusive crises reported post-engraftment [3][4] - The trial is ongoing, with additional data expected by the end of 2025, further supporting the potential of BEAM-101 as a transformative treatment for SCD [2][4]

Financial Performance - Beam Therapeutics reported a loss of $1.00 per share in Q2 2025, which was better than the Zacks Consensus Estimate of a loss of $1.04 and improved from a loss of $1.11 per share in the same quarter last year [1][6] - Total revenues for the second quarter were $8.5 million, down from $11.8 million year-over-year, and missed the consensus estimate of $14 million [1][6] - Research and development expenses increased by approximately 17% to $101.8 million compared to the previous year, while general and administrative expenses decreased by around 9.1% to $26.9 million [3] Cash Position - As of June 30, 2025, Beam Therapeutics had cash, cash equivalents, and marketable securities totaling $1.2 billion, unchanged from March 31, 2025, and is expected to fund operations into 2028 [3] Pipeline Developments - The company is advancing its ex-vivo genome-editing candidate BEAM-101 in a phase I/II BEACON study for sickle cell disease, with enrollment completed for both adult and adolescent cohorts [7] - BEAM-302 is being developed for alpha-1 antitrypsin deficiency (AATD) and is currently in a phase I/II dose-escalation study, with positive initial safety and efficacy data reported [8][9] - The phase I/II study for BEAM-302 consists of two parts, with ongoing dosing and enrollment in Part B for AATD patients with mild-to-moderate liver disease [10][11] Market Performance - Shares of Beam Therapeutics have declined by 24.6% year-to-date, contrasting with a 1.9% rise in the industry [2]

Hematology and Gene Editing Programs - The company is advancing hematology base editing programs, specifically targeting sickle cell disease and beta-thalassemia, with an estimated 100,000 individuals affected by sickle cell disease in the U.S. alone[115] - The BEACON trial for BEAM-101 has dosed 30 patients, achieving a median time to neutrophil engraftment of 16.5 days and a median time to platelet engraftment of 19.5 days[119] - All patients in the BEACON trial achieved endogenous HbF levels exceeding 60% and reduced sickle HbS below 40%, with total hemoglobin levels increasing rapidly[123] - The company plans to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103 by the end of 2025, which includes an anti-CD117 monoclonal antibody[124] - The Phase 1/2 trial of BEAM-302 for Alpha-1 Antitrypsin Deficiency (AATD) showed positive initial safety and efficacy data, with no serious adverse events reported[129] - Following treatment with BEAM-302, rapid and durable increases in total AAT were observed, plateauing around Day 21 and maintained for the duration of follow-up[130] - The company is exploring in vivo base editing programs using lipid nanoparticles (LNPs) to deliver base editors directly to hematopoietic stem cells, potentially eliminating the need for transplantation[125] - The ESCAPE program aims to improve transplant conditioning regimens, reducing toxicity challenges associated with current standard of care[121] - The company’s proprietary base editing technology allows for precise genetic modifications without double-stranded breaks, enhancing the efficiency of gene editing[112] - The company is committed to building a leading integrated platform for precision genetic medicine, with broad therapeutic applicability[113] Financial Performance - The net losses for the six months ended June 30, 2025, were $211.6 million, compared to $189.7 million for the same period in 2024, with an accumulated deficit of $1.8 billion as of June 30, 2025[155] - License and collaboration revenue for the three months ended June 30, 2025, was $8.5 million, a decrease of $3.3 million from $11.8 million in 2024, attributed to reduced research activities[168] - Research and development expenses increased to $101.8 million for the three months ended June 30, 2025, up by $14.7 million from $87.0 million in 2024, primarily due to higher external research costs and increased employee-related expenses[169] - General and administrative expenses decreased to $26.9 million for the three months ended June 30, 2025, down by $2.8 million from $29.6 million in 2024, despite an increase in employee-related costs[171] - The net loss for the three months ended June 30, 2025, was $102.3 million, compared to a net loss of $91.1 million in 2024, reflecting an increase of $11.2 million[167] - For the six months ended June 30, 2025, license and collaboration revenue was $15.9 million, down from $19.2 million in 2024, a decrease of $3.3 million due to reduced research activities[177] - Total operating expenses for the six months ended June 30, 2025, were $255.4 million, an increase of $27.2 million from $228.2 million in 2024, driven by higher research and development costs[176] - The loss from operations for the six months ended June 30, 2025, was $239.4 million, compared to a loss of $209.0 million in 2024, an increase of $30.4 million[176] Cash Flow and Funding - Net cash used in operating activities was $180.3 million for the six months ended June 30, 2025, compared to $182.8 million in 2024[192] - Cash used in investing activities for the six months ended June 30, 2025, was $295.5 million, primarily due to net purchases of marketable securities[197] - Net cash provided by financing activities was $474.5 million for the six months ended June 30, 2025, mainly from the issuance of common stock[198] - As of June 30, 2025, the company had $1.2 billion in cash, cash equivalents, and marketable securities[190] - Future funding requirements may reach up to $90.0 million each for success payments to Harvard and Broad Institute based on stock performance[190] - The company expects operating expenses to increase over the next twelve months due to ongoing clinical development and readiness activities for potential product launches[200] Manufacturing and Collaborations - The company has established a 100,000 square foot cGMP manufacturing facility in North Carolina to support clinical programs and potential commercial supply[152] - The company has entered into a collaboration agreement with Pfizer for in vivo base editing programs, with a profit-sharing ratio of 35% for the company and 65% for Pfizer[145] - Under the Lilly Agreement, the company received a $200 million payment and is eligible for up to $350 million in potential future development-stage payments[147] - The company has licensed a portfolio of three additional gene editing technologies, enhancing its capabilities in treating severe diseases[139] Market Risks and Economic Factors - The company does not anticipate significant market risk from interest rate changes, as a 10% change would not materially affect the fair market value of its investment portfolio[210] - There have been no material changes to the company's contractual obligations and commitments in the first half of 2025[209] - The company is currently not exposed to significant foreign currency exchange risk, but future contracts with international vendors may increase this risk[211] - Inflation has not materially affected the company's financial statements to date, but future operations may be adversely impacted[212]

Exhibit 99.1 Beam Therapeutics Reports Second Quarter 2025 Financial Results and Provides Update on BEAM-302 Development Progress in Alpha-1 Antitrypsin Deficiency (AATD) With 17 Patients Dosed in the Phase 1/2 Trial, BEAM-302 Continues to Demonstrate Durable Correction of the Disease-causing Mutation, Restoration of AAT Physiology, and a Well Tolerated Safety Profile BEAM-302 Development Progress and Clinical Update Positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previou ...

Core Insights - Beam Therapeutics has made significant progress in its clinical trials, particularly with BEAM-101 for sickle cell disease and BEAM-302 for alpha-1 antitrypsin deficiency, with key data updates expected by early 2026 [2][11][7] Financial Performance - As of June 30, 2025, the company reported cash, cash equivalents, and marketable securities totaling $1.2 billion, an increase from $850.7 million at the end of 2024, providing a cash runway expected to support operations into 2028 [10][11] - Research and Development (R&D) expenses for Q2 2025 were $101.8 million, up from $87.0 million in Q2 2024, while General & Administrative (G&A) expenses decreased to $26.9 million from $29.6 million in the same period [11][16] - The net loss for Q2 2025 was $102.3 million, or $1.00 per share, compared to a net loss of $91.1 million, or $1.11 per share, in Q2 2024 [11][16] Clinical Development Progress - The BEACON Phase 1/2 trial of BEAM-101 has completed dosing for 30 patients, including the first adolescent patient, with plans for further data presentation at the end of 2025 [11][2] - The BEAM-302 trial has dosed 17 patients, showing promising results in restoring alpha-1 antitrypsin levels and reducing disease-causing mutations, with a full program update expected in early 2026 [3][5][7] - The company is expanding the dose exploration phase of BEAM-302 and has initiated enrollment in Part B of the trial, targeting patients with mild to moderate liver disease [4][5] Regulatory Designations - BEAM-101 received orphan drug designation from the FDA, aimed at supporting the development of treatments for rare diseases [11] - BEAM-302 was granted both Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations by the FDA, facilitating its development for serious unmet medical needs [11]

Company Overview - Beam Therapeutics Inc. (BEAM) shares increased by 5.7% to $21.46 in the last trading session, with a notable trading volume, and have gained 12.3% over the past four weeks [1][2] - The recent price rise is linked to investor optimism regarding the company's pipeline, particularly after the FDA granted orphan drug designation to BEAM-101 for treating sickle cell disease [2] Financial Performance - The company is projected to report a quarterly loss of $1.04 per share, reflecting a year-over-year increase of 6.3%, with expected revenues of $13.79 million, up 17.1% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without earnings estimate revisions [4] Industry Context - Beam Therapeutics operates within the Zacks Medical - Biomedical and Genetics industry, which includes other companies like Exact Sciences (EXAS) [5] - Exact Sciences has seen a 29.7% increase in its consensus EPS estimate over the past month, indicating a significant change from the previous year [6]

Core Viewpoint - The healthcare sector is highlighted as a critical area in the stock market, driven by the need for innovative therapies and medical technologies to address an aging population and rising chronic diseases. There are ten under-the-radar healthcare stocks identified as potential investment opportunities. Group 1: Company Highlights - **Certara**: Positioned to benefit from AI-driven transformations in medicine, providing bio-simulation software that accelerates drug development, with over 90% of novel drugs approved by the FDA since 2014 utilizing its technology [3][4]. - **Beam Therapeutics**: A clinical-stage biotech focusing on precision gene editing therapies for genetic diseases, with its lead candidate in phase 1/2 trials for sickle cell disease, showing promise for long-term growth [5][6]. - **Inspire Medical Systems**: Develops implantable devices for obstructive sleep apnea, reporting a 23% year-over-year revenue increase to $201 million, indicating strong market demand [8]. - **Insulet**: Specializes in tubeless insulin pump technology, targeting a revenue growth of 19% to 22% by 2025, with significant expansion opportunities in international markets [9]. - **Krystal Biotech**: Focuses on rare skin diseases, with its gene therapy expected to generate approximately $400 million in revenue this year, reinforcing its market position [10]. - **LifeMD**: Operates a telehealth platform with a 49% year-over-year revenue increase, driven by a partnership with Novo Nordisk, indicating potential for further growth [11]. - **Option Care Health**: Leading provider of home infusion services, positioned to benefit from the shift towards personalized healthcare [12]. - **Tempus AI**: Utilizes AI for precision medicine, projecting over 80% revenue growth this year to about $1.3 billion, highlighting its expansive data ecosystem [13]. - **TransMedics Group**: Innovates in organ transplantation with its FDA-approved Organ Care System, projecting a 30% revenue growth this year [14][15]. - **Veeva Systems**: Offers cloud-based software for life sciences, serving over 1,000 customers and positioned to capitalize on the industry's digital transformation [16].