Aldevron Presentation - Robust Characterization Assays Enable Efficient GMP Production of Ribonucleoprotein Complexes
Thursday, May 30, 2024 12:45 PM - 01:00 PM
Exhibit Hall - Theatre A
Global Showcase Presentation
Presenter(s)
• Max Sellman, Senior Product Manager, Gene Editing, Aldevron, USA
The CRISPR-associated gene editing system has emerged as a highly advantageous method to develop new gene and cell therapies. A proven technique for effective ex vivo genome editing in a variety of cell types is to deliver a CRISPR-ribonucleoprotein (RNP) complex consisting of single guide RNA (sgRNA) and Cas nuclease. Therapeutic developers may seek to co-deliver RNP and a homology-directed repair (HDR) DNA template to enable HDR and gene correction.
RNP delivery has become a widespread approach for gene-editing clinical trials, but drug developers face significant challenges when initiating their clinical programs, including the design of the target-specific guides, the production of reliable, high-quality nucleases and repair templates, and the ability to characterize the RNP complex for the efficient and safe delivery of a genomic therapy.
Aldevron cGMP nucleases and donor template delivery technology have been demonstrated to achieve high knock-in rates at key loci. We have developed a suite of novel characterization assays measuring specific activity of the RNP complex in vitro, free (unbound) guide RNA, and free Cas nuclease. These analytical data can support in the quality release process and stability studies for a GMP-manufactured RNP complex, enabling robust data generation in light of evolving regulatory requirements.
Session Objectives:
• Learn how Aldevron GMP gene editing reagents (CRISPR nucleases, Nanoplasmid) are being applied in support of clinical programs worldwide.
• Explore novel characterization assays developed by Aldevron in support of RNP platform The CRISPR-associated gene editing system has emerged as a highly advantageous method to develop new gene and cell therapies.
A proven technique for effective ex vivo genome editing in a variety of cell types is to deliver a CRISPR-ribonucleoprotein (RNP) complex consisting of single guide RNA (sgRNA) and Cas nuclease. Therapeutic developers may seek to co-deliver RNP and a homology-directed repair (HDR) DNA template to enable HDR and gene correction.
RNP delivery has become a widespread approach for gene-editing clinical trials, but drug developers face significant challenges when initiating their clinical programs, including the design of the target-specific guides, the production of reliable, high-quality nucleases and repair templates, and the ability to characterize the RNP complex for the efficient and safe delivery of a genomic therapy. Aldevron cGMP nucleases and donor template delivery technology have been demonstrated to achieve high knock-in rates at key loci.
We have developed a suite of novel characterization assays measuring specific activity of the RNP complex in vitro, free (unbound) guide RNA, and free Cas nuclease. These analytical data can support in the quality release process and stability studies for a GMP-manufactured RNP complex, enabling robust data generation in light of evolving regulatory requirements.
Learning Objectives:
• Learn how Aldevron GMP gene editing reagents (CRISPR nucleases, Nanoplasmid) are being applied in support of clinical programs worldwide.
• Explore novel characterization assays developed by Aldevron in support of RNP platform
• Max Sellman, Senior Product Manager, Gene Editing, Aldevron, USA
The CRISPR-associated gene editing system has emerged as a highly advantageous method to develop new gene and cell therapies. A proven technique for effective ex vivo genome editing in a variety of cell types is to deliver a CRISPR-ribonucleoprotein (RNP) complex consisting of single guide RNA (sgRNA) and Cas nuclease. Therapeutic developers may seek to co-deliver RNP and a homology-directed repair (HDR) DNA template to enable HDR and gene correction.
RNP delivery has become a widespread approach for gene-editing clinical trials, but drug developers face significant challenges when initiating their clinical programs, including the design of the target-specific guides, the production of reliable, high-quality nucleases and repair templates, and the ability to characterize the RNP complex for the efficient and safe delivery of a genomic therapy.
Aldevron cGMP nucleases and donor template delivery technology have been demonstrated to achieve high knock-in rates at key loci. We have developed a suite of novel characterization assays measuring specific activity of the RNP complex in vitro, free (unbound) guide RNA, and free Cas nuclease. These analytical data can support in the quality release process and stability studies for a GMP-manufactured RNP complex, enabling robust data generation in light of evolving regulatory requirements.
Session Objectives:
• Learn how Aldevron GMP gene editing reagents (CRISPR nucleases, Nanoplasmid) are being applied in support of clinical programs worldwide.
• Explore novel characterization assays developed by Aldevron in support of RNP platform The CRISPR-associated gene editing system has emerged as a highly advantageous method to develop new gene and cell therapies.
A proven technique for effective ex vivo genome editing in a variety of cell types is to deliver a CRISPR-ribonucleoprotein (RNP) complex consisting of single guide RNA (sgRNA) and Cas nuclease. Therapeutic developers may seek to co-deliver RNP and a homology-directed repair (HDR) DNA template to enable HDR and gene correction.
RNP delivery has become a widespread approach for gene-editing clinical trials, but drug developers face significant challenges when initiating their clinical programs, including the design of the target-specific guides, the production of reliable, high-quality nucleases and repair templates, and the ability to characterize the RNP complex for the efficient and safe delivery of a genomic therapy. Aldevron cGMP nucleases and donor template delivery technology have been demonstrated to achieve high knock-in rates at key loci.
We have developed a suite of novel characterization assays measuring specific activity of the RNP complex in vitro, free (unbound) guide RNA, and free Cas nuclease. These analytical data can support in the quality release process and stability studies for a GMP-manufactured RNP complex, enabling robust data generation in light of evolving regulatory requirements.
Learning Objectives:
• Learn how Aldevron GMP gene editing reagents (CRISPR nucleases, Nanoplasmid) are being applied in support of clinical programs worldwide.
• Explore novel characterization assays developed by Aldevron in support of RNP platform
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