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 initiateda gene therapy trial in two ALD patients for whom there wereno matched donors. Autologous CD34+ cells were removed fromthe patients, genetically corrected ex vivo with a lentiviralvector encoding wild-typeABCD1, and then re-infused into thepatients after they had received myeloablative treatment. Overa span of 24 to 30 months of follow-up, we detected polyclonalreconstitution, with 9 to 14% of granulocytes, monocytes, andT and B lymphocytes expressing the ALD protein. These resultsstrongly suggest that hematopoietic stem cells were transducedin the patients.Right now most of the focus in the genetics business is on whole-genome sequencing and the race to make the price more affordable, as well as the applications that will make the interpretation of genetic data easier for doctors and patients. But the whole point of knowing our genes - and the possible mutations that we carry - is the idea that we may someday be able to challenge nature and actually fix the problems. Investments in the companies are that creating these medicines are the key to this future.
Photo Source: A Hermida