Welcome to our dedicated page for Beam Therapeutics news (Ticker: BEAM), a resource for investors and traders seeking the latest updates and insights on Beam Therapeutics stock.
Beam Therapeutics Inc. (BEAM) is a pioneering biotechnology company developing precision genetic medicines through its innovative base editing platform. This page serves as the definitive source for official company announcements, clinical trial updates, and strategic developments in gene editing technology.
Investors and researchers will find curated press releases covering therapeutic advancements, financial disclosures, and scientific partnerships. Our collection includes updates on BEAM's hematology programs, in vivo editing initiatives, and manufacturing capabilities - all essential for understanding the company's position in genetic medicine.
Key content categories include clinical trial progress for sickle cell disease therapies, intellectual property developments in base editing, and strategic collaborations advancing CRISPR-based treatments. Each update is carefully verified to ensure accuracy and compliance with financial reporting standards.
Bookmark this page for direct access to Beam Therapeutics' latest milestones in creating single-edit genetic therapies. Check regularly for authoritative updates on one of biotechnology's most promising precision medicine platforms.
Beam Therapeutics (NASDAQ: BEAM) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-302, a genetic medicine designed to treat Alpha-1 Antitrypsin Deficiency (AATD). This follows the recent clearance of their U.S. IND application in March 2025.
The Phase 1/2 trial has shown promising initial results, with BEAM-302 demonstrating good tolerability and dose-dependent mutation correction. In the 60 mg dose cohort, total AAT protein levels exceeded therapeutic threshold. The company has begun dosing in the fourth cohort (75 mg) and plans to initiate Part B of the trial in H2 2025, targeting AATD patients with mild to moderate liver disease.
The RMAT designation provides benefits including accelerated approval pathways, increased FDA interaction, and potential priority review, recognizing BEAM-302's potential as a transformative, one-time treatment for AATD patients.
Beam Therapeutics (NASDAQ: BEAM) reported significant progress in Q1 2025 and key financial results. The company achieved several clinical milestones, including: dosing the first patient in the Phase 1/2 study of BEAM-301 for Glycogen Storage Disease Type Ia, advancing BEAM-302 to its fourth cohort for Alpha-1 Antitrypsin Deficiency after positive initial data, and receiving FDA IND clearance. The BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease is progressing, with updated data to be presented at EHA 2025.
Financially, Beam ended Q1 2025 with $1.2 billion in cash, cash equivalents, and marketable securities, bolstered by a $500 million financing. Q1 2025 showed a net loss of $109.3 million ($1.24 per share), with R&D expenses at $98.8 million and G&A expenses at $27.9 million. The company's cash runway is expected to extend into 2028.
Beam Therapeutics (BEAM) presented additional data from its Phase 1/2 clinical trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation Conference in Lisbon. Key findings from the 60 mg cohort (n=3) showed:
- Corrected M-AAT reached 91% of total AAT in circulation at Day 28
- 79% mean decrease in mutant Z-AAT observed at Day 28
- Single doses were well-tolerated with durable dose-dependent correction
The company has initiated a fourth cohort evaluating 75 mg of BEAM-302 and plans to report additional data in H2 2025. Beam also received FDA clearance for BEAM-302's IND, allowing U.S. site activation. The company plans to begin Part B of the trial, including AATD patients with mild to moderate liver disease, in H2 2025.
Beam Therapeutics (Nasdaq: BEAM) announced FDA clearance of its investigational new drug (IND) application for BEAM-302, targeting alpha-1 antitrypsin deficiency (AATD) treatment. This marks the company's second in vivo base editing program with an open IND in the U.S.
BEAM-302, a liver-targeting lipid-nanoparticle formulation, is designed to correct the PiZ mutation in AATD patients. The ongoing Phase 1/2 trial includes Part A for patients with lung disease and Part B for those with mild to moderate liver disease. Initial safety and efficacy data reported in March 2025 demonstrated proof of concept as a potential one-time treatment.
The treatment has received clinical trial authorization across six countries. Beam plans to continue dose-escalation in Part A, including a fourth dose cohort, and expects to begin dosing Part B patients in H2 2025, with updated data presentation scheduled for the same period.
Beam Therapeutics (Nasdaq: BEAM) has announced the pricing of an underwritten offering of 16,151,686 shares of common stock at $28.48 per share, along with pre-funded warrants for 1,404,988 shares at $28.47 per warrant. The offering is expected to generate approximately $500.0 million in gross proceeds.
The company plans to use the proceeds for advancing its platform technology, research and development activities, and pre-commercialization efforts. Key focus areas include the biologics license application for BEAM-101, advancement of a potential pivotal trial for BEAM-302 targeting alpha-1 antitrypsin deficiency, and development of a Phase 1/2 clinical trial for the ESCAPE conditioning platform in sickle cell disease patients.
The offering is expected to close around March 11, 2025, with J.P. Morgan, Jefferies, Cantor, Citigroup, and Wells Fargo Securities serving as joint book-running managers.
Beam Therapeutics (NASDAQ: BEAM) announced breakthrough initial data from its Phase 1/2 trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD). The trial demonstrated the first-ever clinical genetic correction of a disease-causing mutation through base editing.
Key findings from the first three single-ascending dose cohorts (15mg, 30mg, and 60mg) showed:
- The third dose level (60mg) achieved mean total AAT of 12.4µM at Day 28, exceeding the protective therapeutic threshold
- Reduction of mutant Z-AAT up to 78%
- Durable, dose-dependent increases in total and functional Alpha-1 Antitrypsin (AAT)
- Favorable safety profile with no serious adverse events or dose-limiting toxicities
The treatment demonstrated potential as a one-time therapy addressing both liver and lung disease in AATD patients. The company plans to continue dose escalation and expects to present updated data from Part A of the trial at a medical conference in H2 2025.
Beam Therapeutics reported its Q4 and full year 2024 financial results and reiterated key milestones for 2025. The company achieved its adult enrollment target in the BEACON trial for BEAM-101 in sickle cell disease, with dosing and updated data expected by mid-2025. Initial data from the Phase 1/2 trial of BEAM-302 in alpha-1 antitrypsin deficiency is anticipated in the first half of 2025, and dosing in the Phase 1/2 trial of BEAM-301 in glycogen storage disease type 1a is expected to start in early 2025. Beam ended Q4 2024 with $850.7 million in cash and equivalents, projecting a cash runway into 2027.
Financial highlights include:
- Q4 2024 R&D expenses: $101.4 million
- Full year 2024 R&D expenses: $367.6 million
- Q4 2024 G&A expenses: $28.7 million
- Full year 2024 G&A expenses: $111.5 million
- Q4 2024 net loss: $90.4 million, or $1.09 per share
- Full year 2024 net loss: $376.7 million, or $4.58 per share
Beam's strong financial position supports its ongoing and upcoming clinical trials and regulatory activities. The company's focus remains on delivering life-long cures for serious diseases through precision genetic medicines.
Beam Therapeutics (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines through base editing, has announced its management's upcoming participation in three major investor conferences in March 2025:
• The TD Cowen 45th Annual Health Care Conference on March 3 at 1:50 p.m. ET
• The Leerink Partners Global Biopharma Conference on March 10 at 1:40 p.m. ET
• The Barclays Global Healthcare Conference on March 11 at 3:30 p.m. ET
The company will participate in fireside chats at each event. Live webcasts will be accessible through Beam's investor website section at www.beamtx.com and will remain archived for 60 days after the presentations.
Beam Therapeutics (Nasdaq: BEAM), a biotechnology company focused on developing precision genetic medicines through base editing, has announced its participation in the upcoming Guggenheim SMID Cap Biotech Conference. John Evans, the company's CEO, will engage in a fireside chat scheduled for Thursday, February 6, 2025, at 2:30 p.m. ET in New York.
The event will be accessible to interested parties through a live webcast on the investor section of Beam's website (www.beamtx.com). The presentation recording will remain available on the website for 60 days following the event.
Beam Therapeutics (Nasdaq: BEAM) announced it will present data from the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease at the 2025 Tandem Meetings in Hawaii. The presentation will feature results previously shown at the ASH Annual Meeting in December 2024, covering seven treated patients. Key findings include robust and durable increases in fetal hemoglobin (HbF), reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and improved markers of hemolysis. The safety profile aligned with expected outcomes for busulfan conditioning and autologous hematopoietic stem cell transplantation. The company plans to present updated trial data in mid-2025.
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