BioTech IP Review

Publication Number: US20240252550A1
Filing Date: April 26, 2024
Publication Date: August 1, 2024
Assignee: Beam Therapeutics Inc.
Inventors: Giuseppe Ciaramella (and possibly others as listed on the application)

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What This Patent Covers

This patent application describes compositions and methods for genetically modifying hepatocytes (liver cells) using base editing technology — Beam’s proprietary precision genome‑editing approach:

• Base editor systems tailored to target specific genomic sequences in hepatocytes (liver cells). These systems typically comprise a programmable DNA‑binding protein, a nucleobase editing enzyme, and a guide RNA (gRNA) to direct the edit to the desired DNA site.
• Treating hereditary diseases: The methods involve introducing targeted edits associated with beneficial therapeutic outcomes — for example, reducing expression or activity of KLKB1 (kallikrein B1) to treat conditions linked to its activity.
• Therapeutic application: While specific disease claims aren’t fully disclosed in the abstract, the underlying concept positions these methods as therapies for genetic diseases of the liver or systemic diseases corrected via liver gene editing.
• Delivery and editing tools: The invention contemplates using programmable DNA base editors (e.g., cytosine or adenine base editors) that install precise single‑base changes without creating double‑strand DNA breaks — Beam’s hallmark advantage over traditional CRISPR/Cas9.

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Why This Is Important

• Central to Beam’s clinical programs: Beam’s lead in‑vivo base‑editing therapies (e.g., BEAM‑302 for alpha‑1 antitrypsin deficiency and BEAM‑301 for glycogen storage disease type Ia) aim to correct disease‑causing liver gene mutations — the genes expressed primarily in hepatocytes. Progress toward commercial gene‑editing medicines requires strong IP covering liver cell base editing methods.
• Therapeutic breadth: A patent like this can serve as platform protection for a range of liver‑targeted base editing therapies — a major part of Beam’s pipeline.
• Strategic value: Covering methods of gene editing in hepatocytes adds IP depth beyond specific products, supporting exclusive rights to use base editing in a key organ system manipulated by multiple programs in Beam’s pipeline.

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Summary

The US20240252550A1 patent application covers compositions and methods for genetically modifying hepatocytes using base editors — essentially enabling precise therapeutic editing of liver cell genomes. This application is strategically important for Beam because it underpins key in‑vivo gene editing programs designed to treat liver‑associated genetic diseases using the company’s base editing platform.