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Biopharma

To develop potential treatments and cures for diseases, researchers need to draw on a variety of highly accurate sequencing data such as genomic, transcriptomic, DNA methylation and targeted sequencing data.

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Accelerate your research and development with highly accurate and comprehensive sequencing data

Whether you are conducting research on novel potential drug targets, biomarkers, or therapeutics such as gene therapies, PacBio offers a sequencing solution that can improve your chances of success and may reduce your time to market. Sequencing has become an integral part of the research and development process and helps to develop the critical understanding of determinants of health and disease on the molecular level.

HiFi sequencing allows you to overcome many limitations that short-read or less accurate long-read methods may pose and can play important roles in enhancing your research on

 

  • Target or biomarker identification
  • AAV gene therapies
  • CRISPR-Cas9 gene editing
  • Biologics development

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Gene therapy

Discover, verify, and improve AAV vector design with full-length, highly accurate AAV.

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Gene editing

Assess on- and off-target CRISPR-Cas9 gene editing outcomes and conduct insertion site analysis.

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Biologics R&D

Enhance your work on biologics, from plasmid sequencing, cell line verification, mRNA full length sequencing, to directed evolution.

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Webinar

HiFi 101 for biopharma: insights from an industry panel

Sequencing plays an increasingly important role in drug discovery and development. Whether it’s identifying biomarkers, engineering mRNA or oligonucleotide therapeutics, optimizing viral vectors, or characterizing immune repertoires, accurate and complete sequencing is essential.

Watch a foundational webinar focused on PacBio HiFi sequencing and its growing impact in the biopharma industry.

Watch the recording to:

  • Understand the unique advantages of HiFi sequencing compared to other sequencing technologies.
  • Discover how highly accurate long reads enable advancements in ASO design, antibody development, AAV vector characterization, and evaluation of gene editing outcomes.
  • Learn how you can access HiFi sequencing through service providers or owning your own instruments.
  • Hear from fellow industry panelists how they are incorporating HiFi sequencing into their areas of research & development.

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The benefits of HiFi sequencing for popular biopharma applications

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AAV gene therapy

  • HiFi can be widely applied in AAV development (e.g., genome heterogeneity, sequence integrity)
  • Outstanding performance on ITRs and complicated structures

Antibody development

  • High read quality
  • Improved throughput especially with Kinnex kits
  • Read length to span full-length scFv and Fab

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mRNA vaccine development

  • Fidelity for sequence identity
  • Ability to sequence poly-A tail
  • Throughput improvement with multiplexing, Kinnex kits, and automation

Application brief

HIFI SEQUENCING FOR ANTIBODY DEVELOPMENT

Antibodies play a central role in both therapeutic development and biomedical research, but conventional approaches using phage display and traditional sequencing methods often fall short when it comes to capturing the full complexity of antibody molecules.

Download this application brief to learn how HiFi sequencing is uniquely able to overcome shortcomings of other sequencing technologies for antibody development.

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Identify novel drug targets and biomarkers quickly and confidently

PacBio is an established company providing quality data and excellent customer service to facilitate your research process. HiFi sequencing enables you to see what you cannot see with other methods to allow you to discover novel biomarkers or potential drug targets first.

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WGS

HiFi sequencing allows you to identify more variants than other methods and assess challenging regions while providing phasing information.

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RNA sequencing

There is increasing evidence that isoforms are critical to understanding health and disease.

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DNA methylation

5mC information is critical to understanding gene regulation and is provided with every DNA sequencing run.

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Tech note

A PRACTICAL GUIDE TO AMPLIFICATION-FREE PURETARGET CUSTOM PANELS

Custom PureTarget panels give the highest accuracy look at on- and off-target gene edits. Deep coverage of native DNA, including methylation, lets you assess point mutations and structural variants. PureTarget is being used to perform amplification-free gene editing assessment in zebrafish embryos and humanized mouse lines detecting allele frequencies as low as 1%.

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Application brief

HIGHLY ACCURATE HIFI READS FOR AAV RESEARCH

Your moment of discovery in AAV research is waiting. PacBio long-read HiFi sequencing allows you to sequence the entire AAV genome with extraordinary accuracy and completeness so that you can quickly discover and optimize gene therapy vectors.

Download this application brief to discover the advantages of using PacBio highly accurate long-read sequencing for your AAV research.

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Blog

Isoform level information leads to the development of novel ASOs for Parkinson’s disease

HiFi long-read sequencing economically provides highly accurate multi-omic data for all areas of the genome. Increase what you can see to include all variant types and transcripts at isoform level. A recent study used PacBio to identify a promising region in SNCA transcripts relevant to Parkinson’s disease, and used this information to design and test a novel antisense oligo (ASO).

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Video

A biopharma company uses HiFi sequencing to help in the development of novel AAV gene therapies

The speakers from Homology medicines use AAV genome sequencing as well as RNA sequencing to enhance their research and development beyond what short reads do.

HiFi sequencing to stratify ALS patients for personalized treatments

A novel study will use long-read sequencing to stratify patients suffering from amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The sequencing data will provide novel information on disease associated variants that short-read sequencing may not detect, as well as methylation information, to help match patients with supplements or treatments. Further research may uncover relevant biomarkers or novel targets.

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Video

Using long-read sequencing for directed evolution of novel AAV capsids

Directed evolution approaches can be limited by the sequence length that short-read sequencing allows. Irvin Garza of UT Southwestern overcame this obstacle by using long-read sequencing, which allowed him to introduce mutations in the cap gene of the AAV capsid in a much longer region. This lead to the discovery of several interesting candidates for novel capsids with desired tissue specificity to the central nervous system.

Featured long-read sequencing systems

PacBio long-read sequencers unlock the tools needed for novel gene therapy approaches.

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