Form Bio and PacBio Announce Collaboration To Launch New Resources Set To Transform AAV Design and Analysis Best Practices

Form Bio, announced a range of new initiatives aimed at advancing and unifying the AAV industry. The initiatives, including an AAV data dictionary, open-source tools and more, are being driven by a new AAV Working Group, jointly assembled by Form Bio and PacBio and composed of leading AAV experts across industry and academia. The resulting materials are being made available on a new AAV Resource Hub at aav.formbio.com.

AAV is widely recognized as one of the most promising vectors for gene therapy due to its non-pathogenic nature. However, collaboration on AAV-based programs is often hindered by industry silos that lead to divergent terminology and methods, which can differ widely between various companies and institutions operating at the forefront of AAV innovation. In addition, the lack of standard definitions and practices works against uniformity of regulatory submissions, making it harder and more time consuming for regulatory agencies to evaluate potential new therapies.

In response, Form Bio and PacBio have convened leading experts to form an AAV Working Group dedicated to developing and promoting standardized nomenclature and reporting practices to foster consistency and reliability in AAV research and development. The charter of the newly formed AAV Working Group is to develop standardized nomenclature, establish standardized reporting, and promote wide-spread adoption of these initiatives.

Key members of the AAV Working Group:

• Nam Tonthat, PhD - Taysha Gene Therapies
• Pranam Chatterjee, PhD - Department of Biomedical Engineering, Duke University
• Douglas McCarty, PhD - Nationwide Children's Hospital
• Phillip W.L. Tai, PhD - Department of Microbiology and Physiological Systems, UMass Chan Medical School; Horae Gene Therapy Center, UMass Chan Medical School
• Elizabeth Tseng, PhD - PacBio
• Nadia Sellami, PhD - PacBio
• Eric Talevich, PhD - Scientific Solutions Architect, Form Bio
• Alpha Diallo, PhD - Head of Solutions Science, Form Bio
• Amicia Elliott, PhD - Product Director, Life Sciences, Form Bio
• Brandi Cantarel, PhD - VP of Bioinformatics, Form Bio
• Claire Aldridge, PhD - Chief Strategy Officer, Form Bio

“Getting AAV-based gene therapies to the clinic requires speed and collaboration among many different parties. But it’s become clear that speed and collaboration are being held back by the lack of a common language and practices in dealing with AAV vectors,” said Claire Aldridge, Ph.D, chief strategy officer for Form Bio. “We see working with our partners at PacBio, and bringing together leaders across academia and industry as a critical need in helping AAV-based gene therapies reach their fullest potential faster,” she added.

“PacBio is thrilled to be part of this effort and partnering with Form Bio and leading experts in the field. The value of highly accurate long-read sequencing for gene therapy is clear, and we are excited to see how these resources help researchers develop the next generation of vectors.” Nadia Sellami, Ph.D, Segment Lead, Cell and Gene Therapy for PacBio.

The AAV Working Group has generated several key, publicly available resources:

• Preprint Publication: Standardized Nomenclature and Reporting for PacBio HiFi Sequencing and Analysis of rAAV Gene Therapy Vectors
• Open Source Workflow: LAAVA (Long-read AAV Analysis) 
• Glossary of Terms: AAV Data Dictionary
• Regulatory Case Study: Characterizing Drug Products with NGS

All the above resources and more can be explored at the new AAV Resource Hub at aav.formbio.com.

These resources aim to streamline AAV development processes, reduce costs, and improve accessibility to these powerful therapies. By fostering consistency and clarity in AAV research and development, Form Bio and PacBio are paving the way for more efficient and effective gene therapy solutions, ultimately benefiting patients worldwide.