Beam Therapeutics Inc 2024年度报告

At Beam, our vision is to develop lifelong cures for patients suffering from serious diseases. Thishas never felt more tangible than it does today, having achieved clinical proof-of-concept acrossboth of our core franchises in hematology and liver genetic diseases, further establishing ourleadership in base editing and our commitment to transforming genetic medicine. We believe base editing can enable one-time curative therapies, fundamentally reshaping howdiseases are treated while significantly reducing the burdens of today’s chronic diseasemanagement. With a pioneering platform, world-class capabilities, and a highly skilled team,Beam is positioned to drive innovation and lead the next generation of genetic medicine. Fully Integrated, Clinically Validated Base Editing Platform •Delivery Excellence:We have successfully delivered therapiesex vivo, using CD34 and Tcells, andin vivo, using our lipid nanoparticle (LNP) technology for liver-directedtreatments.•Manufacturing Strength: Our North Carolina facility has completed over 100 GMPbatches, reinforcing our robust production and scaling capabilities.•Regulatory and Clinical Execution: With seven investigational new drug (IND) orclinical trial authorization (CTA) approvals across five countries and 30+ active clinicaltrial sites, our programs continue to advance rapidly. High-Value Franchises with Best-in-Class Profiles HematologyClinical Proof-of-Concept with BEAM-101:Our lead Wave 1 base editor, BEAM-101, is •an investigational autologous cell therapy designed to efficiently and uniformly increasefetal hemoglobin without using double-stranded DNA breaks. BEAM-101 delivers agenetically modified cell product through stem cell transplant, enabled by chemotherapyconditioning, for patients with severe sickle cell disease (SCD).In our ongoing BEACONPhase 1/2 clinical trial of BEAM-101, initial data demonstrated a robust increase in fetalhemoglobin of >60%, a decrease in hemoglobin S to <40%, resolution of anemia in allpatients, a low number of cell collection cycles, and a rapid time to engraftment. Thesedata underscore the potential for BEAM-101 to offer a differentiated, best-in-classtreatment for patients with SCD.•Proof-of-Concept with ESCAPE in Non-Human Primates (NHPs): Our Wave 2innovation, ESCAPE (Engineered Stem Cell Antibody Evasion), is developing a non-genotoxic alternative to traditional myeloablative conditioning, which is associated withchallenging side effects and risks. As a result, ESCAPE could open up the potential tobring the benefits of curative therapy to the vast majority of SCD patients who areineligible for or unwilling to undergo a chemotherapy-based transplant. Proof-of-conceptfor ESCAPE in NHPs demonstrated successful engraftment of base-edited cells usingonly antibody conditioning and no chemotherapy. These data represent a potentialparadigm shift in transplant medicine, providing a strong foundation for advancingESCAPE into the clinic this year. •In VivoEditingto Expand the Reach of Our Medicines: Our Wave 3 innovation aims todeliver base editors intravenously via LNPs targeted to hematopoietic stem cells,eliminating the need for transplantation altogether. In so doing, ourin vivoeditingtechnology has further potential to simplify delivery and expand the application of baseediting to reach many more patients. Liver Genetic DiseasesClinical Proof-of-Concept for BEAM-302 and Correction of a Disease-Causing •Mutation: BEAM-302 is designed to correct the PiZ allele, the most common genevariant linked to severe alpha-1 antitrypsin deficiency (AATD). BEAM-302 has thepotential to simultaneously reduce the aggregation of mutant, misfolded AAT protein thatcauses toxicity to the liver and increase circulating levels of corrected and functional AATprotein, addressing the underlying pathophysiology of both the liver and lung disease. Inour ongoing Phase 1/2 clinical trial of BEAM-302, initial data demonstrated a favorablesafety profile, durable, dose-dependent correction of the PiZ mutation, and a clinicallymeaningful increase in total AAT in patients. To our knowledge, these are the first data todemonstrate a genetic correction of a disease-causing mutation in DNA, representing apotential breakthrough treatment for patients. •Advancing BEAM-301 into Clinical Development: BEAM-301 is designed to correct theR83C mutation, the most common disease-causing mutation that results in the mostsevere form of glycogen storage disease type 1a (GSD1a), a severe metabolic disorderwith potentially life-threatening and long-term complications. BEAM-301 aims tonormalize blood glucose without continuous supplementation to improve metabolicparameters. Preclinical data have shown that a single administration of BEAM-301directly and durably corrected the R83C mutationin vivo, with an ongoing significantsurvival benefit at one-year post-treatment. Executing with Speed and Precision •BEAM-101:In our BEACON trial, we have achieved our target enrollment o