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August 01, 2017 • DNA

A biotechnology company designing treatments with gene edits and transfers using benign viruses is raising $83.5 million in its second venture funding round. With today’s announcement, Homology Medicines Inc. in Bedford, Massachusetts, a one year-old developer of genetic therapies for rare diseases, raised a total of $127 million since its founding.


(Nogas1974, Wikimedia Commons)

Homology Medicines creates treatments that perform gene editing or transfer healthy genes to replace mutations causing disease, delivered with engineered adeno-associated viruses, benign viruses designed to deliver genetic material into the cells of patients with defective genes. The viruses are naturally occurring microbes that can infect cells, but do not integrate with the cell's genome or cause disease in humans, other than at most mild reactions. The company’s technique injects healthy genes in viral shells called capsids, into the target cells, where they penetrate cell membranes and deliver the healthy gene into the cell nucleus holding the DNA.

In gene therapies, the Homology Medicines process delivers healthy genes to replace missing or mutated genes at the root of inherited diseases, where they result in expression of functioning proteins that were previously missing or faulty. The company says in preclinical studies, gene therapies were tested for diseases of the liver, eyes, central nervous system, skeletal muscles and cardiac muscles.

For gene editing, the adeno-associated viruses deliver pieces of DNA specific to precise regions of DNA. Upon reaching their targets in the DNA, the delivered DNA pieces use a natural process called homologous recombination to repair the suspected fragments. The company’s lead product addresses inherited metabolic disorders that result from genetic errors.

Homology Medicines licenses research on gene editing and therapies with adeno-associated viruses by Saswati Chatterjee, a professor of surgery at City of Hope medical center in Duarte, California. Chatterjee, a scientific founder of the company, pioneered gene transfers into blood-forming stem cells, and later identified and isolated naturally occurring adeno-associated viruses from those cells.

In September 2016, Homology Medicines licensed research from California Institute of Technology that enables adeno-associated viruses to cross the blood-brain barrier, often an obstacle in treating nervous system disorders. This technology is based on research by Viviana Gradinaru and Benjamin Deverman, published in the journal Nature Biotechnology earlier in 2016.

The $83.5 million financing round was led by venture investment company Deerfield Management, joined by current Homology Medicines investors 5AM Ventures, ARCH Venture Partners, and Temasek. New investors taking part in the funding are Fidelity Management and Research Company, HBM Healthcare Investments, Maverick Ventures, Novartis, Rock Springs Capital, Vida Ventures, Vivo Capital, and Alexandria Venture Investments.

“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,” says Arthur Tzianabos, CEO of Homology Medicines in a company statement.

Originally posted in Science & Enterprise

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