Gene Therapy Pipeline Update: AGTC Presents Promising Data of its AAV Vectors for Ocular Gene Therapy

Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company, developing transformational genetic therapies for patients suffering from rare and debilitating diseases, announced new data from non-clinical studies evaluating the effect of pre-existing anti-AAV antibodies on the transduction and expression efficiency of AAV vectors.

According to the company’s CSO, Preexisting immunity to AAV remains a challenge for many AAV gene therapies due to the high prevalence of AAV in the general population. While the eye has the partial immune privilege and appears less affected by NAbs, fully understanding the degree of vector neutralization that occurs following ocular administration of AAV-based gene therapies is important for the approval of our clinical-stage product candidates and for their safe and effective use in patients. The data to be presented support the safety and efficiency of our AAV vectors independent of Nab levels, which should allow the use of these vectors in larger patient populations.”

The company will be presenting the data in a poster at the European Society of Gene and Cell Therapy (ESGCT) taking place in Barcelona.

Check out the complete news here.

Gene Therapies for Ophthalmic Diseases

The announced results show positive signs for the future development of gene therapies in patients with ophthalmic diseases. The pipeline already has several candidates in development for eye disorders. Here is a snapshot of the gene therapies pipeline.

Infographic Gene Therapy Pipeline Review

The results are exciting for the overall gene therapies market, which is expected to grow to close to $12 billion by 2030. Check out the free insights about the market here.