Targeted Genetics expands AAV patent portfolio, has exclusive rights to newly issued AAV1 patent.
to University of Pennsylvania (Philadelphia, PA) and is exclusively licensed to Targeted Genetics. Targeted Genetics utilizes AAV vectors to deliver genes of interest into targeted cells to treat or prevent disease.
"The patent on AAV1 is an important addition to our portfolio of AAV-related assets, and helps strengthen Targeted Genetics' leadership role in the development of AAV-based therapies," said Barrie J. Carter, Ph.D., chief scientific officer of Targeted Genetics. "Studies by a number of groups have demonstrated that AAV vectors containing AAV1 serotype capsids are extremely efficient for gene expression when delivered to muscle. Issuance of this patent provides Targeted Genetics with expanded disease targets for which delivery to the muscle is considered most optimal for successful treatment or prevention of disease."
Dr. Carter added, "Due to their enhanced expression in muscle, genes delivered using AAV1 appear to express secreted proteins that have greater bioavailability in circulation than genes delivered with AAV2 to muscle. This could potentially result in an opportunity for lower cost of goods, less frequent dosing and improved therapeutic utility."
"AAV vectors have become one of the most promising gene delivery systems, due to many characteristics that give it particular utility for in vivo gene delivery," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "Having access to intellectual property that covers a broad range of AAV assets places Targeted Genetics in a stronger position to capitalize on the product development opportunities that utilize AAV. We believe that AAV1 assets and technology have the potential to become a major component of our future product development and partnering activities."
AAV naturally exists as several different serotypes, which differ physically in the composition of their capsid proteins and genome structures. AAV vectors containing different AAV capsid serotypes have been reported to transduce certain tissues more efficiently and with different specificity. The patent covers compositions of AAV vectors with AAV1 serotype capsids. Vectors containing AAV1 capsid proteins have been reported to have higher levels of expression of the gene of interest when delivered to muscle compared to other AAV capsid serotypes such as AAV2 or AAV5.
Targeted Genetics was the first to bring AAV vectors into clinical trials. AAV is a naturally occurring virus that is not associated with any disease in humans. AAV vectors can efficiently deliver genetic information to numerous cell types and can be engineered to carry a variety of DNA sequences. These vectors are highly stable, persist in cells for extended periods of time, and carry a very low frequency of integration into host-cell DNA.
Targeted Genetics develops gene-based products for preventing and treating acquired and inherited diseases. The company has three clinical product development programs, targeting cystic fibrosis, AIDS prophylaxis and rheumatoid arthritis. The company also has a promising pipeline of product candidates focused on hemophilia and cancer and a broad platform of gene delivery technologies.
Targeted Genetics Corporation,
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|Title Annotation:||Targeted Genetics Corp.; adeno-associated viral|
|Publication:||BIOTECH Patent News|
|Date:||Jul 1, 2004|
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