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Pipeline Broad and diversified portfolio

We seek to potentially cure serious diseases and address significant unmet medical need so that people can reach their full potential. We believe leveraging ex vivo and in vivo approaches, different delivery modalities, and multiple editing approaches will help us target a broad range of diseases with limited or no disease-modifying treatment.

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Lead programs Sickle cell disease

Sickle cell disease (SCD) is a group of genetic disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disease inherit two copies of a gene, each with a single letter misspelling. This misspelling causes production of atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape, blocking the flow of blood and oxygen throughout the body. The characteristic features of SCD begin in childhood and may include anemia, repeated infections, and episodes of pain. The severity of symptoms varies from person to person and throughout life. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, such as stroke and organ damage. Chronic organ damage is the leading cause of death in adults with SCD.

Beam is advancing two ex vivo base editing programs, Beam-101 and Beam-102, in which cells are collected from a patient, edited, and then infused back into the patient. To create space for the edited cells to take hold in the bone marrow, patients undergo a conditioning regimen, such as treatment with busulfan, the standard of care in hematopoietic stem cell (HSC) transplantation today.

BEAM-101

Edit type: Activation

Activation

Delivery modality: Electroporation | Ex vivo

Approach: Activation of fetal hemoglobin

BEAM-101 is an investigational therapy that produces base edits designed to potentially alleviate the effects of sickle cell disease by mimicking genetic variants seen in individuals who have hereditary persistence of fetal hemoglobin.

BEAM-102

Edit type: Gene correction

Correction

Delivery modality: Electroporation | Ex vivo

Approach: HbG-Makassar

BEAM-102 is an investigational therapy designed to edit the causative hemoglobin S point mutation directly to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant is believed to be a normal- functioning variant of hemoglobin.

LONG-TERM COMMITMENT TO SCD

Today’s conditioning regimens are associated with significant toxic effects. Future improved conditioning regimens that produce fewer toxic effects could potentially be paired with BEAM-101 and BEAM-102 and with other future programs.

Beam is also exploring the potential for in vivo base editing programs for SCD, in which base editors would be delivered to a patient through an infusion of lipid nanoparticles (LNPs) targeted to HSCs, eliminating the need for transplantation. This approach has the potential to provide a more accessible option for patients, particularly in regions where ex vivo treatment and transplantation are not widely accessible.

T-cell cancers

Chimeric antigen receptor T-cell (CAR-T) therapies have the potential to address a number of T-cell cancers, but their therapeutic potential is often hindered by technological limitations in their development and application. For example, approved CAR-T therapies are created from a patient’s own cells, which creates both technical and logistical challenges. To address these limitations, we are using multiplex base editing to create donor-derived CAR-T cells by simultaneously silencing multiple target genes, thereby potentially enabling universal compatibility and resistance to host rejection, fratricide, and immunosuppression.

BEAM-201

Edit type: Multiplex editing

Multiplexing

Delivery modality: Electroporation | Ex vivo

Approach: Gene silencing

BEAM-201 is a multiplex base edited anti-CD7 CAR-T cell investigational therapy for relapsed and refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma, a severe disease affecting children and adults.

Glycogen storage disease 1a

Glycogen storage disease type 1a (GSD1a) is a serious, potentially life-threatening genetic disease with no approved disease-modifying treatments available to date. In patients who have GSD1a, glycogen accumulates in organs and tissues such as the liver, kidneys, and small intestine. The accumulation of glycogen (and sometimes fat) and inability to generate glucose during fasting can lead to severe and life-threatening hypoglycemia, seizures, metabolic dysfunction in lipids, short stature, liver adenoma, and renal disease.

BEAM-301

Edit type: Gene correction

Correction

Delivery modality: LNP | In vivo

Approach: Correction of R83C mutation

BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation. R83C is the most common mutation responsible for causing GSD1a.

Base Editing Applications

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Base editing is an emerging class of precision genetic medicines designed to overcome the limitations of existing approaches and expand the potential of genetic medicine.

Our delivery strategy is to establish a suite of clinically validated technologies, including electroporation and nonviral and viral delivery modalities.

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