Companies Form Expert Working Group to Introduce Essential Tools for AAV Development and Research Professionals

SAN FRANCISCO / ACCESSWIRE / June 27, 2024 / Form Bio, the provider of advanced computational life sciences technology and PacBio (NASDAQ:PACB), a leading developer of high-quality, highly accurate sequencing solutions, today 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 technologies 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 widespread adoption of these initiatives.

Members of the AAV Working Group include:

• Nam Tonthat, Ph.D - Asklepios BioPharmaceutical
• Pranam Chatterjee, Ph.D - Department of Biomedical Engineering, Duke University
• Douglas McCarty, Ph.D - Nationwide Children's Hospital
• Phillip W.L. Tai, Ph.D - Department of Microbiology and Physiological Systems, UMass Chan Medical School; Horae Gene Therapy Center, UMass Chan Medical School
• Adam Cockrell, Ph.D - Solid Biosciences
• Elizabeth Tseng, Ph.D - PacBio
• Nadia Sellami, Ph.D - PacBio
• Eric Talevich, Ph.D - Form Bio
• Alpha Diallo, Ph.D - Form Bio
• Amicia Elliott, Ph.D - Form Bio
• Brandi Cantarel, Ph.D - Form Bio
• Claire Aldridge, Ph.D - 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 vectorsl," said 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 of the above resources and more can be explored on the new AAV Resource Hub at aav.formbio.com.

These resources aim to streamline development processes and collaboration across the AAV industry. 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.

To access these and other resources or to learn more about getting involved with the AAV Working Group, please visit aav.formbio.com.

About Form Bio

Form Bio provides award-winning software and AI solutions for cell and gene therapy leaders. By combining data, technology and expertise, Form's solutions accelerate timelines from discovery to clinic by providing drug developers with rapid in silico characterization, prediction, simulation and optimization of their therapeutics - enabling higher yields, enhanced safety and shorter, less-expensive development cycles. With cross-disciplinary expertise spanning software engineering, biology, bioinformatics and data science, the Form Bio team collaborates closely with customers on their most pressing and strategic challenges and opportunities. For more information, visit or follow the company at

About PacBio

PacBio (NASDAQ: PACB) is a premier life science technology company that designs, develops, and manufactures advanced sequencing solutions to help scientists and clinical researchers resolve genetically complex problems. Our products and technologies stem from two highly differentiated core technologies focused on accuracy, quality and completeness which include our HiFi long-read sequencing and our SBB short-read sequencing technologies. Our products address solutions across a broad set of research applications including human germline sequencing, plant and animal sciences, infectious disease and microbiology, oncology, and other emerging applications. For more information, please visit and follow @PacBio.

PacBio products are provided for Research Use Only. Not for use in diagnostic procedures.

Forward-Looking Statements

This press release may contain "forward-looking statements" within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the U.S. Private Securities Litigation Reform Act of 1995. All statements other than statements of historical fact are forward-looking statements, including statements relating to PacBio's collaboration with Form Bio and the AAV Working Group; the potential benefits and impacts of the AAV Working Group; the benefits or potential benefits of AAV-based programs; and other future events. You should not place undue reliance on forward-looking statements because they are subject to assumptions, risks, and uncertainties and could cause actual outcomes and results to differ materially from currently anticipated results, including the risk that PacBio's collaboration with Form Bio and the AAV Working Group may not be successful or deliver the benefits that PacBio currently anticipates. Additional factors that could materially affect actual results can be found in PacBio's most recent filings with the Securities and Exchange Commission, including PacBio's most recent reports on Forms 8-K, 10-K, and 10-Q, and include those listed under the caption "Risk Factors." These forward-looking statements are based on current expectations and speak only as of the date hereof; except as required by law, PacBio disclaims any obligation to revise or update these forward-looking statements to reflect events or circumstances in the future, even if new information becomes available.

Contact Information

Angela Anderson
Form Bio
angela@formbio.com

Jake Robison
Inizio Evoke Comms
jake.robison@inizioevoke.com

Todd Friedman
PacBio (Investor Contact)
ir@pacificbiosciences.com

PacBio Media Contact
pr@pacificbiosciences.com

SOURCE: Form Bio