Arbor Biotechnologies Secures $74M Funding Amid Gene Editing Industry Challenges

Arbor Biotechnologies, a Cambridge-based gene editing company, has successfully raised $74 million in a Series C funding round, demonstrating resilience in a challenging landscape for genetic medicine startups. The funding, led by ARCH Venture Partners and TCGX, comes at a time when many peers in the industry are struggling to secure venture investment.

Gene Editing's 'Gartner Hype Cycle' and Industry Turbulence

The gene editing sector has entered what Arbor's CEO Devyn Smith describes as a "show-me state" in the Gartner hype cycle. Recent setbacks in the industry include Editas Medicine's significant staff reduction and bluebird bio's sale for a fraction of its former valuation. Even CRISPR Therapeutics, despite achieving the first-ever approval for a CRISPR-based gene editor, has announced layoffs.

Despite these challenges, Smith remains optimistic about gene editing's potential. "I'm very bullish that gene editing is a modality that can be applied to many diseases successfully," he stated, noting that only about 200 patients worldwide have been dosed with gene editing therapies to date.

Advancing ABO-101 for Primary Hyperoxaluria Type 1

The majority of the new funding will support the clinical development of Arbor's lead candidate, ABO-101, targeting primary hyperoxaluria type 1 (PH1). This rare liver-and-kidney condition affects between 1 and 3 out of every million people in the U.S. and Europe.

ABO-101 has received orphan drug and rare pediatric disease designations, as well as IND clearance in December 2024. The Phase I/II trial, named RedePHine, is set to begin soon with an estimated primary completion date in 2029.

Unlike currently approved treatments from Alnylam Pharmaceuticals and Novo Nordisk that require regular injections, ABO-101 is designed as a "one-and-done" therapy. It aims to durably reduce oxalate levels by knocking down the HAO1 gene using a CRISPR-Cas12 system delivered via lipid nanoparticles.

Arbor's Technological Edge and Future Prospects

Arbor's platform utilizes Cas12, a smaller CRISPR protein than the more common Cas9. This approach potentially offers advantages in production costs and reduced off-target effects. The company has also employed AlphaFold to engineer improvements in Cas12's precision.

Beyond PH1, Arbor is exploring treatments for other liver diseases and neurological conditions such as amyotrophic lateral sclerosis (ALS). The company has formed collaborations with major players like Vertex Pharmaceuticals, which has committed up to $1.2 billion in milestone payments for various disease targets.

With this latest funding round, Arbor has secured a cash runway into 2027. As the gene editing field faces a pivotal year with numerous trial readouts on the horizon, Smith believes these results could help rekindle investment interest in the sector. "That will help build the safety database, as well as demonstrate the clinical benefit this technology can have for patients, and that's going to be a big boost," he concluded.