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Curing serious diseases one letter at a time Spelling matters

The genetic information that makes you you is stored as a unique code in your DNA. The code is written with just four chemical bases, or letters, labeled A, C, G, and T. People have 3 billion of these letters in their DNA. The letters are arranged in a specific sequence that can spell the difference between health and disease.

Genetic Code 650 X 290Px

A single spelling error in the code—known as a point mutation—can lead to serious illness just as a single misspelled letter in a word can lead to an entirely different meaning: list or lost, batter or better.

Point Mutation 650 X 290Px

Correcting the spelling error

To correct the misspelling, we use a new tool called base editing, which works like an eraser and a pencil. By rewriting a single letter, base editors may correct disease-causing point mutations and potentially create life-long cures for patients suffering from serious diseases.

Base editing has the potential to correct a disease-causing point mutation with just a few treatments—or possibly with one treatment that may last a lifetime.

Base editor image
DNA Image DNA image for mobile

Educational resources*

Gene Therapy 101

Available from the American Society of Gene and Cell Therapy at

Gene Editing

Available from the American Society of Gene and Cell Therapy at

Understanding the Importance of Gene Therapy for Rare Diseases

Available from the National Organization for Rare Disorders at


* 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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An important part of the drug development process is recognizing and validating what matters to patients. To understand their needs, we collaborate with clinical experts, patient advocacy organizations, and disease communities to optimize our clinical trial design and the participant experience.

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