Massachusetts’ Homology Medicines Snags $83.5M to Advance AMEnDR into Human Trials

Massachusetts’ Homology Medicines Snags $83.5M to Advance AMEnDR into Human Trials August 1, 2017
By Mark Terry, Breaking News Staff

Bedford, Mass. – Homology Medicines closed on a Series B financing round worth $83.5 million. The round was led by Deerfield Management and joined by new investors Fidelity Management and Research Company, HBM Healthcare Investments, Maverick Ventures, Novartis , Rock Springs Capital, Vida Ventures, Vivo Capital and Alexandria Venture Investments. It was joined by existing investors 5AM Ventures, ARCH Venture Partners and Temasek. To date the company has raised $127 million.

Homology was founded and incubated in 2015 with a seed investment within the 4:59 Initiative, the company creation engine of 5AM Ventures. The company is building on the naturally occurring process of homologous recombination. The company believes it has a type of gene editing technology that is safer and more effective than other methods, specifically CRISPR-Cas9.

The money raised from this round is to be used to advance the company’s lead development candidate, which is presently in preclinical IND-enabling studies for an inborn error of metabolism disease. It will also support continued development of Homology’s AMEnDR (AAV-Mediated Editing by Direct Homologous Recombination) platform into the clinic for rare genetic diseases.

The company won’t specify yet what disease they’re working on, although it has indicated that it has ongoing projects in CD34 diseases, cystic fibrosis, CNS diseases, Duchenne muscular dystrophy (DMD) and others.

John Carroll, writing for Endpoints News, says, “Homology is one of the upstarts traveling one step behind the pioneering crews that booted up gene editing in a big way. That group includes Editas , CRISPR Therapeutics and Intellia —which all made virtually overnight debuts onto the public markets with a set of IPOs that attracted a tremendous amount of investor attention—and which sagged as the long clinical road to an actual therapy came into view.”

“This funding from leading biotechnology investment firms will enable us to rapidly bring our first development candidate toward the clinic and advance our expanding pipeline,” said Arthur Tzianabos, Homology’s chief executive officer, in a statement. “The strong support from investors who understand the space and appreciate the potential of our novel human-derived adeno-associated virus vectors to both edit and correct genes validates this technology for developing new therapeutic options and, ultimately, cures for patients.”

The company launched in May 2016 and had 12 employees. It was situated in Lexington, Mass., close to the U.S. headquarters of Shire Pharmaceuticals , where Tzianabos and several members of his executive team had worked in rare disease research. Now the company has 40 staffers and is at a larger facility across the Lexington-Bedford border.

Homology’s approach is different enough—and potentially better—than CRISPR. This also has allowed Homology to stay out of the ongoing patent lawsuits over CRISPR-Cas9 that the other companies are tangled in. Recent studies have also found that CRISPR-Cas9 may cause off-target gene editing mistakes.

“We root for the whole field,” Tzianabos told the Boston Business Journal. “Everybody is trying to help patients with rare genetic diseases. But as a group of former Shire executives, we saw this (technology) as very eminently developable. It can be used to precisely change the genome. That’s the big advantage here.”

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