Arbor Scores $215 Million for Next-Gen CRISPR Toolbox

Piggy Bank Money

CRISPR technology is the printing press of our age. By rewriting our very code to cure disease, the potential is nearly limitless. As with all groundbreaking technology, visionaries in the field are already asking, “How can we make it better?” 

Arbor Biotechnologies has been working quietly since its 2018 uncloaking, building its world-changing, gene-editing toolbox that, according to CEO Devyn Smith, Ph.D., is the industry’s “most extensive.” Co-founded by CRISPR pioneer Feng Zhang, Arbor moves beyond the initial discovery of Cas9, utilizing Cas13d, a much smaller molecule with implications for RNA manipulation.  

Today, the Cambridge biotech announced an oversubscribed Series B raise totaling $215 million, a significant increase from their initial, modest $15.6 million Series A round. Arbor will move toward the clinic to put its initial candidates to test for liver and CNS diseases. The company also hopes to move into other CRISPR spaces explore more options in the field.  

“While our primary focus has been on developing our bespoke CRISPR nucleases, we are also looking to progress our other precision editing innovations, such as CRISPR transposases,” said Smith.  

Starting in the liver is not uncommon for CRISPR companies. It’s easier to deliver the gene-editing technology there. With Arbor’s new enzymes, the company hopes to either knock out a gene and replace it with a new one or knock out multiple genes to cure disease.  

The company has three liver disease programs in the works, one targeting primary hyperoxaluria, a rare genetic disease present at birth that often results in end-stage renal disease.  The only hope for a cure for PH type 1 at this point is a liver transplant. IND filing is slated for the end of 2023 to start trials the next year.  

One goal for Arbor’s central nervous system focus is a gene edit treatment that will cure Huntington’s disease, a fatal condition that breaks down the nerve cells in the brain over time. There is no cure for Huntington’s or any way to stop it from getting worse.  

The challenge with delivering CRISPR to the CNS is the delivery system. The adeno-associated virus used for delivery is too small for most CRISPR systems. Arbor is developing alternatives that would create smaller gene-editing tools to address this roadblock.  

Temasek, Ally Bridge Group, and TCG Crossover led the charge, while a handful of new investors also joined the cause. Arbor’s latest strategic partner, Vertex Pharmaceuticals, invested as well.  

Vertex and Arbor recently struck a $1.2 billion deal, building on a three-year old partnership. In the original agreement, Vertex forked over $30 million for Arbor’s RNA-altering enzyme, Cas13d, to help the company with its cystic fibrosis treatments.  

The new agreement will help Vertex develop ex vivo cell therapies for next-gen approaches to treating blood diseases—sickle cell and beta thalassemia—and insulin-producing hypo-immune islet cells to treatof type 1 diabetes. An undisclosed upfront cash payment was made with the bulk promised in milestone payments across seven potential programs. 

Arbor is also looking to double its headcount from 60 staffers to 120 to support their push toward the clinic.  

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