Vol 7 Issue 7

CELL AND GENE THERAPY INSIGHTS

Vol 7 Issue 7

Cell and Gene Therapy Insights Vol 7 Issue 7

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Featuring

Issue Spotlight: Clinical trial design, supply chain and operations
Other articles
Webinars

Clinical trial design, supply chain and operations

Editorial

CRISPR gene editing for sickle cell disease: one disease, differentiated approaches

CRISPR gene editing for sickle cell disease: one disease, differentiated approaches

Lisa A Michaels, MD, Bruce E Eaton, PhD

Innovator Insight

Preparing for pivotal: solving challenges in scale for cell and gene therapy clinical trials

Preparing for pivotal: solving challenges in scale for cell and gene therapy clinical trials

S Viswanathan, E Goodhue Meyer, C Greenberg et al.

Interview

Strengthening the clinical supply chain for individualized therapies

Strengthening the clinical supply chain for individualized therapies

James Andrew Case

Driving clinical progress of gene therapy in cystic fibrosis

Driving clinical progress of gene therapy in cystic fibrosis

Uta Griesenbach

Challenges in the gene therapy of bone marrow failure syndromes

Challenges in the gene therapy of bone marrow failure syndromes

Learning lessons from the long, troubled history of stem cell therapy for future clinical success

Learning lessons from the long, troubled history of stem cell therapy for future clinical success

Addressing issues in clinical development of AAV-driven gene therapy

Addressing issues in clinical development of AAV-driven gene therapy

Journal articles

Expert Insight

Solving the problem of financing one-time treatments with evidence uncertainty: which types of outcomes-based payment models could work best for novel CAR-T therapies in multiple myeloma? A systematic review of the published literature

Solving the problem of financing one-time treatments with evidence uncertainty: which types of outcomes-based payment models could work best for novel CAR-T therapies in multiple myeloma? A systematic review of the published literature

Cassidy-Candice Dietrich, Clare Hague, Stefan Boes

Innovator Insight

When using a closed and automated manufacturing platform, is there an option to maintain flexibility?

When using a closed and automated manufacturing platform, is there an option to maintain flexibility?

Kaman Kim, Carlos Yuraszeck, Joseph O’Connor

Commentary

Critical need for establishing value that justifies the current rising costs of cell and gene therapy

Critical need for establishing value that justifies the current rising costs of cell and gene therapy

Richard T Maziarz, MD

Webinars on Demand: Expert Commentary

Case study: Optimization of lentiviral vector production from early stages to GMP

Case study: Optimization of lentiviral vector production from early stages to GMP

Claire Wartel-Weill, Natalia Elizalde, Rakel López de Maturana

Delivering an end-to-end industrialization roadmap for autologous cell therapy

Delivering an end-to-end industrialization roadmap for autologous cell therapy

M Hewitt PhD, J Garrity, W Bagni

Assuring manufacturing and data integrity for cell and gene therapy regulatory compliance

Assuring manufacturing and data integrity for cell and gene therapy regulatory compliance

Jelena Jurlin, Dmitry Fridman

Expression systems for viral vector production: advantages of the Sf9 Baculovirus system and simple solutions to address its specific analytical challenges

Expression systems for viral vector production: advantages of the Sf9 Baculovirus system and simple solutions to address its specific analytical challenges

Yi Fang Lee, Srinath Kashi Ranganath