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. Presently, over 250 companies are engaged in the gene therapy pipeline, the gene therapy market is estimated to be worth $2.6 billion in 2023.

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 Therapy Pipeline 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.

Nearly 45% of gene therapy pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies. 

Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

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