As other gene editing programs fold or get sold, Arbor Biotechnologies has secured $73.9 million to advance its lead liver-targeting gene therapy into human trials.
The series C was led by Arch Venture Partners and TCGX, with new investors QIA, Partners Investment, Revelation Partners and Kerna Ventures joining alongside other existing investors, according to a March 18 release.
The raise will support ABO-101, a CRISPR-based gene editing therapy meant to treat primary hyperoxaluria type 1 (PH1). The FDA granted the therapy both orphan drug and rare pediatric disease designations in early February.
A two-part phase 1 trial of ABO-101 is slated to start this month, according to ClinicalTrials.gov. The first part of the study will test single ascending doses of the gene therapy in adults with PH1, while the second portion will test a recommended dose in children with the disease.
ABO-101 is Arbor’s first program to enter human trials.
The new cash will also help other branches of Arbor’s pipeline, according to the release, including an editing program for an undisclosed rare liver disease and another program targeting amyotrophic lateral sclerosis.
“Arbor is developing a differentiated portfolio with first-in-class potential to deliver on the promise of CRISPR-based genetic medicines,” Keith Crandell, partner and cofounder at Arch Venture Partners, said in the release. “Arbor has established a track record of pipeline focus, coupled with execution and capital efficiency, to yield strong preclinical data supporting its pipeline.”
PH1 is caused by a malfunctioning copy of the AGXT gene, which makes an enzyme called alanine-glyoxylate aminotransferase (AGT). Though AGT is created in the liver, its absence causes the buildup of oxalate downstream in the kidneys. This buildup causes symptoms like frequent kidney stones, loss of bladder control and painful urination, and, in serious cases, it can lead to kidney failure.
Arbor sprouted out of the shadows in 2018 with a $15.6 million series A and a newfound CRISPR-associated enzyme called Cas13d. This enzyme’s smaller size makes packaging it into gene therapy vectors easier.
Arbor followed up its debut with much bigger money at the end of 2021, signing a deal with Vertex worth up to $1.2 billion and closing a $215 million series B.
Arbor’s $73.9 million series C raise comes as the gene editing field undergoes a period of self-reflection. Pfizer recently discontinued its hemophilia gene therapy Beqvez, which carried a list price of $3.5 million per person and seemingly has not been given to any patients since its April 2024 approval. And bluebird bio, a gene therapy darling once valued at $10 billion, was sold to a pair of venture capital firms last month for a comparatively paltry $29 million.
Massachusetts-based Arbor is forging ahead into the clinic without Chief Scientific Officer John Murphy, Ph.D., who plans to retire. The firm is currently searching for his replacement.