Genzyme Corp. has announced that it has acquired extensive gene therapy assets from Avigen, Inc., including rights to a broad patent estate focused on adeno-associated virus technology, a Phase 1/2 clinical development program in Parkinson's disease, and a clinical collaboration in hemophilia.
In exchange for these assets, Genzyme has made an up-front cash payment of $12 million to Avigen, and will make potential milestone and royalty payments based on the development, approval and sale of products developed under the intellectual property portfolio.
The acquired assets will support and diversify Genzyme's gene therapy research program, which already includes work in cardiovascular disease, lysosomal storage disorders, and other conditions.
Together with last month's acquisition of viral manufacturing facilities from Cell Genesys, Genzyme has significantly strengthened its ability to develop gene therapy products that utilize either adenovirus or adeno-associated virus vectors.
"This agreement provides Genzyme with a strong patent estate for our existing gene therapy platform, and reflects our commitment to achieve the medical advances that we believe are possible with this technology," said Rich Gregory, Genzyme's head of research.
"We look forward to advancing Avigen's ongoing work in Parkinson's disease and hemophilia, areas where we believe gene therapy could play a meaningful role in treatment."
The advanced program in Genzyme's gene therapy portfolio is a Phase 2 clinical trial examining the safety and effectiveness of locally delivered Ad2/HIF-1alpha, an engineered form of the HIF-1alpha gene.
This experimental therapy is designed to promote the growth of new blood vessels and improve circulation in the limbs of patients with peripheral arterial disease.
Genzyme's gene therapy portfolio also includes pre-clinical work related to lysosomal storage disorders and, in partnership with Excigen, Inc., atrial fibrillation.
Genzyme is also conducting pre-clinical gene therapy research through a joint effort with Applied Genetic Technologies.
The Phase 1/2 clinical trial in Parkinson's disease is designed to evaluate the safety of increasing doses of AV201 in individuals with mid- to late-stages of the disease.
AV201 is designed to restore the therapeutic effectiveness of levodopa, the primary treatment for Parkinson's disease, by enhancing the brain's ability to convert it into dopamine.