Can 5AM upstart Homology leapfrog the gene editing pioneers?

In biotech, every big tech revolution is haunted by scientists who believe they can do the same thing more efficiently and with greater efficacy than the pioneers. And 5AM Ventures has pieced together a syndicate to back a new biotech which they believe can forge a new way forward in gene editing, one of the hottest fields in biotech today.

The company is called Homology Medicines, which has now garnered $43.5 million in Series A cash to herald its exit from stealth status and into the race to develop a new gene-editing technology. 5AM, which seeded the venture, arranged for Arch Venture to join as co-lead, with Temasek, Deerfield Management and Arch Overage Fund coming in as well. 

Handling the money is a team of Shire ($SHPG) vets experienced in the management of rare disease programs.

New CEO Arthur Tzianabos will be working with a process gleaned from nature, in which similar sequences of DNA from different chromosomes flip from one chromosome to another. In nature, that process broadens genetic diversity. But at a biotech like Homology, it points a way to accomplish gene editing using adeno-associated virus vectors ID’d from human CD34+ cells.

The tech offers a “very high level of efficiency of transduction (viral transfer) in vivo,” says Tzianabos, “in contrast to nucleases (at rival companies), which are not efficient.”

"In-Vivo gene editing, with high fidelity … has arrived," Tweeted Arch's Robert Nelsen this morning. "Not using Crispr or talens." 

If it works in humans, the company has a shot at pioneering a radical new form of gene therapy, which is exactly what 5AM’s Dr. Kush Parmar went searching for several years ago. The immediate task is to try it out on rare monogenic diseases, looking at the effect in small patient groups.

In some ways, Homology is late to the game, and in other ways it’s still very early. A variety of pioneering companies have been working on editing genes and gene therapy--reaping hundreds of millions of dollars of investors’ cash--but they’re still a considerable distance from seeing products hit the market. Homology’s team will be tasked with a near-term goal of providing some solid proof-of-concept data in humans, focusing on rare diseases triggered by genetic mutations, though there’s no timeline being offered for public review right now.

The tech pioneer here is Saswati Chatterjee, a professor of virology at the Beckman Research Institute at the City of Hope in California and a member of the Recombinant DNA Advisory Committee at the NIH.

Tzianabos, a former associate professor at Harvard Med who handled programs for rare genetic disorders at Shire, will be working with Sam Rasty as chief operating officer and Albert Seymour as chief scientific officer. Rasty had been vice president and head of new products at the Rare Diseases Business Unit of Shire, and Seymour is the former SVP and global head of research and nonclinical development at Shire.

Tzianabos recently left his job as the top scientist at the struggling OvaScience ($OVAS).

- read the release