Ebb & Flow

A number of smart investors are placing bets that gene therapy will finally work. Third Rock Ventures led a $35 million series B round for Genetix Pharmaceuticals Inc. last week. The round included one new investor, Genzyme Ventures, as well as existing investors TVM Capital, Forbion and Easton Capital.

The biotech is initially targeting single gene diseases that are treatable by allogeneic bone marrow transplantation. The company harvests somatic bone marrow cells from the patient, then inserts a normal version of the mutated gene into the cells using its Lentipak lentiviral vector, which is based on research done at Harvard and MIT. The marrow cells are then transplanted back into the patient...