#Spain #San Sebastián #Viralgen #Axovia #BBS

A Innovative Partnership in Gene Therapy

In a remarkable step forward for patients suffering from Bardet-Biedl Syndrome (BBS), Viralgen and Axovia Therapeutics have announced a collaboration aimed at advancing the development and manufacturing of an AAV9-based investigational gene therapy. This partnership represents a significant milestone in the medical community's fight against retinal dystrophy associated with the genetic condition BBS.

Understanding Bardet-Biedl Syndrome

Bardet-Biedl Syndrome is a genetic disorder that presents a myriad of complications, particularly affecting retinal health, leading to progressive vision loss and, in some cases, complete blindness. This condition is primarily caused by mutations in the BBS1 gene, significantly impairing photoreceptor function. As a result, individuals with BBS experience not only declining vision but also potential obesity and other multisystem disorders.

Recognizing the urgent need for effective treatment options, Viralgen and Axovia have combined their expertise to develop a gene therapy designed to target the underlying cause of vision loss. The therapy utilizes an AAV9 vector optimized for the BBS1 gene, which focuses on halting photoreceptor cell death and thereby preserving vision in affected individuals.

The Collaboration

Viralgen, a fully integrated Contract Development and Manufacturing Organization (CDMO), is known for its specialized capabilities in AAV viral vector manufacturing. Leveraging its state-of-the-art facilities in San Sebastián, Spain, Viralgen will play a crucial role in the commercial production of the investigational therapy. The company's advanced Pro10™ manufacturing platform ensures high titers for all AAV serotypes, crucial for effectively delivering the therapy to patients.

Dr. Victor Hernandez, Co-Founder and Chief Scientific Officer of Axovia, expressed enthusiasm about this partnership, stating, "As Axovia advances its pipeline of potential therapies addressing the genetic causes of blindness, we are pleased to partner with Viralgen to ensure we have appropriately scaled AAV manufacturing for our lead program, AXV-101, targeting retinal dystrophy associated with BBS."

Accelerating Development

The partnership aims to expedite the clinical development of AXV-101, which is expected to enter clinical trials by mid-2025. Both companies are committed to facilitating rapid progress toward making this groundbreaking treatment available to patients in need. Jimmy Vanhove, CEO of Viralgen, emphasized their mission, saying, "We are dedicated to supplying quality vectors and timely production to provide disease-modifying treatments for patients suffering from BBS1-related retinal dystrophy."

The collaboration between Viralgen and Axovia not only highlights the importance of combining strengths from both companies but also demonstrates their dedication to addressing the pressing medical needs of individuals affected by Bardet-Biedl Syndrome. With few options available to this patient population, this innovative gene therapy offers new hope.

Promising Future for Gene Therapy

The commitment to research and development in the field of gene therapy showcases how innovative partnerships can deliver crucial advancements in healthcare. By harnessing the latest technology and expertise, Viralgen and Axovia are not only working to bring their investigational therapy to market but are also setting a standard in the gene therapy landscape.

As this collaboration unfolds, it emphasizes the potential of gene therapy to modify disease courses and improve the quality of life for patients suffering from previously untreatable conditions. The merging of know-how from both Viralgen and Axovia stands as a testament to the power of collaboration in the ever-evolving realm of medical science.

In conclusion, this partnership marks a significant step forward in the quest for effective treatments for Bardet-Biedl Syndrome, potentially transforming the way we approach genetic disorders and paving the way for future gene therapy innovations.