3 Examples of FDA approved AAVs in gene therapy

Gene therapy stands at the forefront of medical innovation that offers powerful solutions for complex genetic disorders. A critical aspect of this field is the use of Adeno-Associated Viruses (AAVs) as vectors.

In this blog post, we'll show examples of AAVs as gene therapy vectors and examine the tangible results of FDA-approved AAV gene therapies, which offer actual solutions and hope for patients affected by genetic disorders.

FDA approved AAVs in gene therapy

Based on this overview of FDA-approved AAVs for gene therapy, we have selected three examples on which we will take a more detailed look.^1 2 

AAV example 1: Luxturna – Illuminating vision

Luxturna is a game-changer for individuals with Leber Congenital Amaurosis (LCA). LCA is a rare condition that results in severe vision impairment or blindness from birth. Luxturna is making history as the first FDA-approved AAV gene therapy for a genetic disease. The therapy employs a harmless AAV vector to transmit a functional RPE65 gene, crucial for vision, directly to the retina.

Clinical trials showed remarkable improvements in vision for LCA patients. Luxturna's FDA approval marked a milestone in gene therapy, paving the way for AAV-based treatments for genetic disorders.³ 

AAV example 2: Zolgensma – Transforming lives in SMA

Zolgensma is a treatment for Spinal Muscular Atrophy (SMA). SMA is a severe genetic disorder that affects the motor neurons of the spinal cord, causing muscle weakness and, in many cases, premature mortality.

Zolgensma is a monumental advancement in SMA treatment as it uses an AAV vector to deliver a functional copy of the SMN1 gene, which is crucial for motor neuron survival and addresses the genetic root of the disease.

Zolgensma's impact is nothing short of miraculous. Patients who previously had a grim prognosis now are more likely to enjoy enhanced motor function and quality of life.⁴ 

AAV example 3: Hemophilia B treatment with AAV-based therapies

Hemophilia B is a genetic bleeding disorder resulting from a deficiency of clotting factor IX. Individuals afflicted with Hemophilia B experience prolonged bleeding following injuries or surgeries that can be fatal.

Researchers have utilized AAVs to manage Hemophilia B. FDA-approved AAV gene therapies have been developed that can supply the missing clotting factor IX gene, thereby effectively targeting the underlying cause of the disorder.⁵ 

Conclusion and Outlook

It is evident that the confluence of science and compassion has resulted in groundbreaking advancements in the lives of patients battling genetic disorders. As solution provider for vital vector manufacturers, Single Use Support leads the way in state-of-the-art single-use technologies, with a mission to improve efficiency and safety in AAV production. Our dedication to progressing the field of gene therapy coincides with a collective vision - a vision that ultimately affects the health of patients.

The ongoing research and innovations in AAV gene therapies show great potential. On the one hand, researchers are making progress in advancing therapies to reduce immune responses and treat more gene diseases. On the other hand, there is ongoing development in the area of manufacturing AAVs for increased efficiency and safety. Single Use Support’s commitment lies in contributing to these advancements and making the promise of gene therapy a reality for a greater number of patients.