Hung Trinh: Key Insights From a 2024 Scientific Exchange hosted by ARM, ISCT, and Daânher
Hung Trinh, CMC/MFG Consultant and Managing Director at Vertex Biopharm Consulting, shared a post on LinkedIn:
“Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Daânher
Rare-disease therapeutics face viability challenges due to small patient populations and drug-development and regulatory frameworks that were not developed to address rapidly progressive or quickly fatal conditions.
Because the majority of rare diseases are genetic in nature, gene-editing modalities offer substantial promise. This Scientific Exchange, co-hosted by the Alliance for Regenerative Medicine, the International Society for Cell and Gene Therapy, and Danaher Corporation in November 2024, set out to address the challenge of realizing the full promise of gene editing for rare-disease therapies by advancing platforms that leverage stable and reusable processes or components to develop multiple therapies.
Through multi-stakeholder engagement and discussions of case studies in CRISPR/Cas nuclease, base, and prime editing, 4 key opportunities emerged that deliver value by holding platform elements constant and/or streamlining development steps:
- Consistent delivery vehicle;
- Consistent manufacturing;
- Benefit-risk appropriate quality requirements;
- Expansive clinical trial designs.
Together, these opportunities could yield up to 5-fold efficiency gains and result in substantial value creation for patients, regulators, and developers, potentially decreasing the time required to dose patients with a new gene-editing therapy from years down to 6 months.”
Title: Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher
Authors: Fyodor Urnov, Sadik Kassim, Kiran Musunuru, David Liu, Ann Lee, Luis Barrera, Pam Stetkiewicz, Julianne Bruno, Matthew Hewitt, Troy Lister, Harry Malech, Lindsay Gasch, Matt Diver, Nicholas Gertler, Felix Grignon, Audrey Le, Michael Lehmicke, Vanessa Almendro-Navarro, Josephine Lembong
Read the Full Article on Cytotherapy.
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