Our CommitmentDisease Areas
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 options.
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. SCD is the most common inherited blood disorder in the United States (U.S.), affecting an estimated 100,000 individuals within the U.S. and approximately eight million people worldwide.
Beam is advancing multiple base editing programs for sickle cell disease, including ex vivo and in vivo approaches. Learn more here.
Alpha-1 antitrypsin deficiency
Alpha-1 antitrypsin deficiency (AATD) is a potentially life-limiting genetic disorder that may cause disease in both lung and liver function. AATD is caused by changes in the SERPINA1 gene, leading to lower levels of circulating alpha-1 antitrypsin protein in the blood, which is needed to protect the lungs from damage, and the build-up of toxic mutant protein in the liver. The vast majority of AATD diagnoses are in individuals with two copies of the PiZ variant (PiZZ), a single letter misspelling in the SERPINA1 gene. People with AATD usually develop symptoms of lung disease between 20 and 50 years old. Many individuals with AATD remain undiagnosed, specifically people with obstructive pulmonary disease (COPD) or emphysema. Liver disease can develop from infancy through adulthood. The severity, signs, and symptoms of the disease as well as the age at which they appear vary among individuals even within the same family. It is estimated that approximately 100,000 individuals in the U.S. have two copies of the Z allele, known as the PiZZ genotype, although only about 10% of all patients are thought to have been diagnosed. There are currently no curative treatments approved for patients with AATD, and the only approved therapy in the U.S., intravenous AAT protein replacement, has not been shown to prevent ongoing lung function decline and destruction in patients.
Beam is advancing BEAM-302, an in vivo base editing approach to treating both lung and liver manifestations of AATD. Learn more here.
Glycogen storage disease Ia
Glycogen storage disease type Ia (GSDIa) is a serious, potentially life-threatening genetic disease with no approved disease-modifying treatments available to date. In patients who have GSDIa, 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 is advancing BEAM-301, an in vivo base editing approach to treating GSDIa. Learn more here.
Resources
For more information on diseases, find here additional resources and links to advocacy organizations.
Sickle Cell Disease
Sickle Cell Disease Association of America
www.sicklecelldisease.org
Sick Cells
sickcells.org
Sickle Cell Community Consortium
sicklecellconsortium.org
Sickle Cell Disease Coalition
www.scdcoalition.org
Alpha-1 Antitrypsin Deficiency
Alpha-1 Foundation
alpha1.org
Alpha-1 Europe Alliance
alpha1europe.org
Alpha-1 Association of New Zealand
www.alpha1.org.nz
Alpha-1 Association of Australia
alpha1.org.au
Alpha-1 Organisation Australia
www.a1oa.org.au
Glycogen Storage Disease
Association for Glycogen Storage Diseases
www.agsdus.org
Global Center for Glycogen Storage Disease
globalcenterforgsd.com
Children’s Fund for GSD Research
www.curegsd.org
*Â These resources are for information only. Inclusion of these resources does not indicate endorsement by Beam Therapeutics of an organization or its communications. The list is not intended as a comprehensive list of resources, nor are the resources intended to provide medical advice.
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Our Science
Beam is a values-driven organization with a vision of delivering life-long cures to patients suffering from serious diseases. Â
Our Pipeline
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 options.
Our Disease Areas
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 options.Â