BioInsights - Vol 8 Issue 04

CELL & GENE THERAPY INSIGHTS

Vol 8 Issue 04

Cell & Gene Therapy Vol 8 Issue 04

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Featuring

Issue Spotlight: Gene delivery/gene editing platform evolution
Focus: Vector characterization
Other articles

Gene delivery/gene editing platform evolution

Louise Rodino-Klapac

Guest Editor:

Louise Rodino-Klapac, EVP, Head of R&D, Chief Scientific Officer at Sarepta Therapeutics

Foreword

Gene delivery & gene editing platform evolution

Gene delivery & gene editing platform evolution

Louise Rodino-Klapac

Expert Insight

Engineered exosomes: a transformative therapeutic modality

Engineered exosomes: a transformative therapeutic modality

P Jones, D Carter, D Lowe et al.

Unleashing the full potential of cell therapy through RNA engineering

Unleashing the full potential of cell therapy through RNA engineering

Metin Kurtoglu

Innovator Insight

Process & analytical insights for GMP manufacturing of mRNA lipid nanoparticles

Process & analytical insights for GMP manufacturing of mRNA lipid nanoparticles

E Cameau, P Zhang, S Ip et al.

Commentary

Therapeutic mRNA delivery with targeted lipid nanoparticles: next-generation transformative medicines

Therapeutic mRNA delivery with targeted lipid nanoparticles: next-generation transformative medicines

Umar Iqbal, Jagdeep K Sandhu

Interview

Encouraging signs for a new generation of AAV-driven gene therapy

Encouraging signs for a new generation of AAV-driven gene therapy

Dr Katherine A High

Breaking new ground in the clinical application of CRISPR/Cas9

Breaking new ground in the clinical application of CRISPR/Cas9

Laura Sepp-Lorenzino

Evolution in gene therapy for the treatment of hemophilia

Evolution in gene therapy for the treatment of hemophilia

Roland W Herzog, PhD

FastFacts

Accelerating adherent cell production for diabetes gene therapy using a scalable fixed-bed bioreactor platform

Accelerating adherent cell production for diabetes gene therapy using a scalable fixed-bed bioreactor platform

Tom Bongiorno

Vector characterization

Interview

AAV vector characterization best practices

AAV vector characterization best practices

Gaël Stephant

Viewpoint

The present hurdles and opportunities in our knowledge of AAV basic biology

The present hurdles and opportunities in our knowledge of AAV basic biology

Bijay Dhungel, John Rasko

Innovator Insight

Simplifying analytical development of viral vector production: robust and sensitive methods for common expression systems

Simplifying analytical development of viral vector production: robust and sensitive methods for common expression systems

Srinath Kashi Ranganath

FastFacts

Characterization of advanced therapies: leveraging advanced analytics to avoid FDA holds

Characterization of advanced therapies: leveraging advanced analytics to avoid FDA holds

Dana Cipriano, Alexei Saveliev

Journal articles

Interview

Trends and considerations in cell and gene therapy M&A, partnering and IP

Trends and considerations in cell and gene therapy M&A, partnering and IP

Ewan Townsend

Podcast

The quest for the Holy Grail in AAV chromatography: empty–full separation

The quest for the Holy Grail in AAV chromatography: empty–full separation

Ratish Krishnan, Oliver Rammo

Viewpoint

Overcoming the immune-related shortfalls of AAV

Overcoming the immune-related shortfalls of AAV

Genine Winslow

A new wave of allogeneic CAR-T cell therapies in oncology treatment

A new wave of allogeneic CAR-T cell therapies in oncology treatment

Silvan Tuerkcan, PhD

Poster

Applications and benefits of the CTS Rotea Counterflow Centrifugation System in cell therapy workflows

Applications and benefits of the CTS Rotea Counterflow Centrifugation System in cell therapy workflows

Carl Dargitz