BioInsights - Vol 4 Issue 7

CELL & GENE THERAPY INSIGHTS

Vol 4 Issue 7

Cell & Gene Therapy Vol 4 Issue 7

Featuring

Issue Spotlights: Rare Diseases – Clinical & Commercial Developments | Latest Advances in Bioprocessing: Overcoming Bottlenecks
Other articles

Latest Advances in Bioprocessing: Overcoming Bottlenecks

Ioannis Papantoniou

Guest Editor:

Ioannis Papantoniou, ATMP Bioprocess Research Coordinator at KU Leuven

Expert Insight

Development of purification steps for several AAV serotypes using POROS™ CaptureSelect™ AAVX affinity chromatography

Development of purification steps for several AAV serotypes using POROS™ CaptureSelect™ AAVX affinity chromatography

M Toueille, E ATTEBI, L L Dejoint et al.

Rare Diseases – Clinical & Commercial Developments

Guest Editor:

Jaap Boelens, Pediatrician-Oncologist, Immunologist at University Medical Centre Utrecht

Sven Kili

Guest Editor:

Sven Kili, CEO at Sven Kili Consulting

Editorial

Unique Role of Patient Advocacy Groups in Clinical Development of CGTx for Rare Diseases

Unique Role of Patient Advocacy Groups in Clinical Development of CGTx for Rare Diseases

Michelle Berg

16 October 2018

Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

Hemant Khanna

Expert Insight

Advances & challenges in developing gene therapies for rare kidney diseases

Advances & challenges in developing gene therapies for rare kidney diseases

Valeria Padovano, Michael J Caplan

Gene Therapy for Childhood Cerebral Adrenoleukodystrophy

Gene Therapy for Childhood Cerebral Adrenoleukodystrophy

Christine Duncan

17 October 2018

Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization

Gene replacement therapy for hemoglobinopathies: clinical benefit & challenges for widespread utilization

M Cavazzana, A Miccio, I Andre-Schmutz et al.

16 October 2018

Interview

SR-TIGET: Continuing to Drive Enabling Technology Development & Clinical Innovation in Rare Diseases

SR-TIGET: Continuing to Drive Enabling Technology Development & Clinical Innovation in Rare Diseases

Alessandro Aiuti

17 October 2018

The Critical Role of Patient Advocacy Groups in the Development of Rare Disease Gene Therapies

The Critical Role of Patient Advocacy Groups in the Development of Rare Disease Gene Therapies

Susan Wilson, Elisabeth Linton

16 October 2018

The Future of Gene Therapy for Rare Eye Diseases

The Future of Gene Therapy for Rare Eye Diseases

Aniz Girach

16 October 2018

Leukodystrophies: a Patient Advocacy Perspective

Leukodystrophies: a Patient Advocacy Perspective

Maria Kefalas

16 October 2018

Advancing the Development of AAV-Based Gene Therapy for Rare Diseases

Advancing the Development of AAV-Based Gene Therapy for Rare Diseases

Suyash Prasad

14 October 2018

Journal articles

Research Article

A Novel Low Cost High Yield Clinical Scale Cell Separator

A Novel Low Cost High Yield Clinical Scale Cell Separator

P Liberti, D Ritter, T Khristov