Rare Diseases – Clinical & Commercial Developments

CELL AND GENE THERAPY INSIGHTS

July 2018

Rare Diseases – Clinical & Commercial Developments

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

16 October 2018

Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

Developing Gene Therapy for CEP290-Associated Retinal Ciliopathies

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

Leukodystrophies: a Patient Advocacy Perspective

Leukodystrophies: a Patient Advocacy Perspective

16 October 2018

The Future of Gene Therapy for Rare Eye Diseases

The Future of Gene Therapy for Rare Eye Diseases

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

14 October 2018