The Future of Adenovector-Based Therapies with Precigen's Helen Sabzevari, Ph.D.
Jan 30, 2025
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Helen Sabzevari, Ph.D., President and CEO of Precigen, shares insights on innovative adenovirus-based therapies. She discusses the promising use of gorilla adenovectors, which enhance the delivery of large genetic payloads for effective gene therapies. Sabzevari reveals exciting results from Precigen's recent BLA submission, with over 50% of patients achieving Complete Response in pivotal studies. She also highlights the company's groundbreaking work in treating recurrent respiratory papillomatosis and the potential for broad applications in immuno-oncology and beyond.
Precigen's use of gorilla adenovirus vectors enables the delivery of larger genetic payloads, enhancing the development of effective gene therapies.
The recent BLA submission for PRGN-2012 marks a pivotal shift towards commercialization, promising FDA approval for a groundbreaking treatment for RRP.
Deep dives
Innovative Developments in Gene Therapy
Presogen is focused on advancing gene therapy through its unique platforms: the Gorilla Adenoprovector platform and the UltraCarty platform, with special emphasis on the PRGN 2012 program targeting recurrent respiratory papillomatosis (RRP). This represents a significant step as it seeks to obtain the first FDA-approved treatment for adults with RRP. The regulatory submission is a crucial milestone, marking a transition from research and development to commercialization, which holds great promise for patients suffering from this condition. By leveraging innovative approaches in gene therapy, Presogen aims to address unmet medical needs in immuno-oncology and other areas.
Advantages of Gorilla Adenovectors
The Gorilla Adenovectors distinguish themselves by enabling multiple administrations without a decline in efficacy, unlike traditional adenoviral vectors that lose effectiveness after a few uses due to neutralizing antibodies. These vectors can accommodate larger genetic payloads, facilitating the delivery of numerous epitopes, thus enhancing the immune response against infections or cancers. Specifically, they can hold up to 12 kilobases of genetic material, allowing more comprehensive targeting of diseases like HPV-driven cancers. This approach leads to the potential for generating broader responses in immune cells, thereby preparing a more effective 'army' to combat diverse tumor cells.
Broad Applications and Positive Impact
The capabilities of Gorilla Adenovectors may extend beyond RRP to treat other HPV-related conditions, such as genital warts and various cancers, including cervical and throat cancers. Clinical trials utilizing these vectors have shown promising results, with patients experiencing partial and complete responses even after failing conventional treatments like checkpoint inhibitors. The successful phase one data has propelled Presogen to move forward with phase two trials, substantiating the therapeutic potential of their delivery system in overcoming significant healthcare challenges. This could revolutionize treatment options and provide a much-needed solution for patients with limited existing therapies.
Helen Sabzevari, Ph.D., President and CEO of Precigen, joins Host, Erin Harris to discuss how the company utilizes gorilla adenovirus vectors. These vectors offer significant advantages in delivering large genetic payloads, a crucial factor in developing effective gene therapies. Sabzevari also discusses Precigen's recent BLA submission for PRGN-2012, which includes Phase 1/2 pivotal study results where over 50% of patients achieved Complete Response and more than 85% experienced decreased surgical interventions.