Vote of Confidence for Gene Therapy: $35 Million for Genetix Pharmaceuticals:

Genetix Pharmaceuticals, Inc. one of the leading companies in gene therapy, has secured $35 million in series B funding from investors Third Rock Ventures and Genezyme Joint Ventures. This is a sign that investors are starting to take gene therapy, which has been labeled as overly hyped in recent years, much more seriously.

Gene therapy is the other great promise of genetics (next to gene sequencing). The idea is that functional genes can be inserted into a patient's cells in order to actually fix a gene mutation that causes a hereditary disease. There are two major kinds of gene therapy: somantic gene therapy and germ line gene therapy.

The Genetix investment came as a result of a new study offering promise results in Adrenoleukodystrophy (ALD), a deadly neurodegenerative disorder, and beta-Thalassemia, a blood disorder that reduces the production of hemoglobin, which is responsible for carrying oxygen to the cells through out the body. A study published in the November 2009 issue of Science showed promising results:

We initiated a gene therapy trial in two ALD patients for whom there were no matched donors. Autologous CD34⁺ cells were removed from the patients, genetically corrected ex vivo with a lentiviral vector encoding wild-typeABCD1, and then re-infused into the patients after they had received myeloablative treatment. Over a span of 24 to 30 months of follow-up, we detected polyclonalreconstitution, with 9 to 14% of granulocytes, monocytes, and T and B lymphocytes expressing the ALD protein. These results strongly suggest that hematopoietic stem cells were transduced in the patients.

Photo Source: A Hermida