Beam Therapeutics shared updated clinical data from the BEAM-302 program for alpha-1 antitrypsin deficiency, demonstrating durable correction of the disease-causing mutation, increased total and functional AAT, reduced mutant Z-AAT and toxic Z-polymers, and evidence that AAT production remains under normal physiologic control.
Published
Signal category
Research & Knowledge
Quote
“Today, we shared updated clinical data from our BEAM-302 program (for alpha-1 antitrypsin deficiency) which demonstrates durable correction of the disease-causing mutation, increased total and functional AAT, reduced mutant Z-AAT and toxic Z-polymers, and evidence that AAT production remains under normal physiologic control.”
— Gopi Shanker|Beam Therapeutics team
Company
Beam Therapeutics
Our vision is to provide life-long cures for patients suffering from serious diseases.
- Industry
- Biotechnology Research
- Location
- Cambridge, US
- Company size
- 598 employees
Beam Therapeutics, launched in 2018, is pioneering the use of CRISPR base editing to develop a broad portfolio of advanced genetic medicines. Our groundbreaking base editing technology allows us to make permanent, specific edits to single bases in DNA and RNA, without cutting the strands. Base editor therapeutics represent a new class of “precision genetic medicines,” combining precision targeting of the genome with precision control of editing outcomes. Our dream is to provide life-long cures for patients suffering from serious diseases. The Beam Team Is: • A community of fearless innovators • Rigorous and honest in our research • Listening with open minds • Committed to each other
Founded 2017