Gene editing has spent the last decade proving what's possible in the lab. The next decade is about proving it can reach the patients who need it most, from ultra-rare liver diseases to conditions like ALS, where no truly effective treatments exist today. 

Arbor Biotechnologies is one of the companies pushing that frontier, built on a diverse toolbox of editing technologies that goes beyond the limitations of first-generation CRISPR tools. Its lead clinical program, ABO-101, targets PH1, and last October the company struck a partnership with Chiesi Group worth up to two billion dollars in milestones, with European regulators granting the program orphan drug designation just this summer. 

Arbor is also building out a CNS pipeline focused initially on ALS. Here to talk through the science, the strategy, and what's next is Devyn Smith, CEO of Arbor Biotechnologies.

01:36 – Meet Devyn Smith

07:48 – Arbor's toolbox approach versus single-editor CRISPR companies

12:16 – What PH1 is and how ABO-101 works

15:19 – Inside the Chiesi partnership for ABO-101

20:14 – Why ALS anchors Arbor's CNS pipeline

26:48 – Arbor's three-year outlook and long-term vision

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To dive deeper into the topic: 

• What’s on the horizon for gene editing-based therapies?
• CRISPR technology’s next wave: Ten companies to watch in 2025 
• Beyond CRISPR: What CAST could bring to genome editing