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.
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.
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.
Educational resources*
Gene Therapy 101
Available from the American Society of Gene and Cell Therapy at
https://patienteducation.asgct.org/gene-therapy-101
Gene Editing
Available from the American Society of Gene and Cell Therapy at
https://patienteducation.asgct.org/gene-therapy-101/gene-editing
Understanding the Importance of Gene Therapy for Rare Diseases
Available from the National Organization for Rare Disorders at
https://rarediseases.org/gene-therapy/
* 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.
The Beam Story
Our Research
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.
