A Boston-area genetics medicine firm has attracted $43.5 million in initial capital to move forward in testing its proprietary gene editing technology platform to eventually treat patients suffering rare genetic disorders.
Lexington, Massachusetts-based Homology Medicines CEO Arthur Tzianabos announced the Series A Preferred Stock financing this week. He also revealed the new firm’s advisory board and management team as they moved out of stealth mode.
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Tzianabos, who characterized the funding as “a huge leap forward for a new way to gene-edit for patients suffering with rare disorders,” said he expects the funding will allow Homology Medicines to demonstrate proof of concept of utility in disease models that can be translated to clinical treatment.
Tzianabos said Homology’s management also includes Chief Operating Officer Sam Rasty and Chief Scientific Officer Albert Seymour. He said the team, who previously held executive positions with global pharmaceutical firm, Shire, has wide experience in translating scientific innovation to clinical application.
The preferred stock financing effort was led by next-generation life sciences venture capital firm 5AM Ventures and technology startup funders ARCH Venture Partners. It also included Singapore-based investment firm Temasek, healthcare investment management company Deerfield Management and the ARCH Overage Fund.
Homology’s next move is to develop therapeutics for commercial use, moving from animal experiments and studies, to validation, journal publication and treatment, Tzianabos said.
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“We initially want to focus on rare blood disorders, but feel this has a very broad application,” he said.
He said the technology platform offers a completely new and independent approach to the treatment of genetic disorders by harnessing the normal repair mechanism of the human body’s DNA — naturally occurring agents that can induce a process called homologous recombination — a way to deliver a correct gene to replace a defective gene.
Tzianabos said the technology is based on the pioneering research of Homology Medicine’s co-founder, virologist Saswati Chatterjee, of the City of Hope National Medical Center outside Los Angeles. Chatterjee and her team discovered a novel class of virus vectors that can perform these genetic corrections in vivo with a single administration of this form of gene editing.
Chatterjee also chairs the company’s newly announced scientific advisory board.
“Our technology is taking these virus vectors and directly injecting them and inducing repair and corrections,” Tzianabos said, pointing out that the virus vectors have been shown to be safe in tests on hundreds of humans and the new technology has performed well in tests on mice and rats.
Photo: Flickr user Lisa Williams
