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Beam Therapeutics Provides Business and Pipeline Updates

Company On-track to Submit First IND for BEAM-101 in the Second Half of 2021 Continued Progress Across Base Editing Portfolio, Including Initiation of. ....

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A Base Hit: How Base Editing Is Changing Genomics

For growth investors seeking disruptive exposure in the healthcare sector, genomics is often a favored destination. ....

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CRISPR 2.0: Base Editing in the Groove

CRISPR 2.0: Base Editing in the Groove
A series of exciting preclinical and animal model results show that base editing is making rapid strides toward the clinic
March 4, 2021
Researchers from the Broad Institute, the NIH, and Vanderbilt University have used an adenine base editor (ABE) to treat progeria in mice. Correcting the mutation that causes progeria led to strong symptom reduction and longer lifespan. An ABE is shown here bound to a guide RNA and a targeted piece of DNA. [Aditya Raguram, Liu Laboratory, Broad Institute]
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A reasonable idea, suggests Sekar Kathiresan, MD, co-founder and CEO of Verve Therapeutics, is the development of a one-time treatment a single spelling change in the DNA of a liver gene in an adult person that would “turn off the gene and lower blood cholesterol levels for the rest of the person’s life.” Although this idea would not have been taken very seriously 10 years ago, it is now being investigated. It is a potent ill ....

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Progeria study finds base-editing therapy lengthens lifespan in mice

Luke Koblan sat at his desk, staring at some data. Excited but baffled, the grad student thought, there’s no way those numbers could be right. He shared the results with his boss, Professor David Liu, and today, that data has spurred a team of scientists from the Broad Institute of MIT and Harvard (including Koblan and Liu), the National Institutes of Health, and Vanderbilt University Medical Center to achieve an almost unbelievable leap for the CRISPR gene-editing tool known as base editing, moving the technology a critical step closer to being a treatment for progeria, a deadly premature aging disease. ....

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