Rare Diseases

Trials evaluating the efficacy of CD20 CAR-T cell therapy in WM and CNS lymphoma 1:00
Trials evaluating the efficacy of CD20 CAR-T cell therapy in WM and CNS lymphoma
Mazyar Shadman • 9 Dec 2022
Treating otoferlin deficiency with gene therapy 2:51
Treating otoferlin deficiency with gene therapy
Nawal Ouzren • 26 Apr 2022
Novel gene therapy solutions for Usher syndrome type I 2:05
Novel gene therapy solutions for Usher syndrome type I
Nawal Ouzren • 26 Apr 2022
Developing gene therapies for GJB2 gene related hearing loss 1:59
Developing gene therapies for GJB2 gene related hearing loss
Nawal Ouzren • 26 Apr 2022
Overcoming manufacturing issues in gene therapies for rare diseases 1:38
Overcoming manufacturing issues in gene therapies for rare diseases
John Maslowski • 27 Jan 2022
Innovations in treating rare diseases with gene therapies 2:12
Innovations in treating rare diseases with gene therapies
John Maslowski • 27 Jan 2022
OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome 1:29
OTL-103: An autologous gene therapy for Wiskott Aldrich syndrome
Kent Christopherson • 27 Jan 2022
Novel gene therapies for metachromatic leukodystrophy 2:31
Novel gene therapies for metachromatic leukodystrophy
Kent Christopherson • 27 Jan 2022
Challenges in manufacturing viral vectors for the clinic 4:51
Challenges in manufacturing viral vectors for the clinic
Nathalie Clement • 27 Jan 2022
Treating Krabbe disease with FBX-101 1:58
Treating Krabbe disease with FBX-101
John Maslowski • 27 Jan 2022
Increasing patient recruitment to rare disease trials 1:06
Increasing patient recruitment to rare disease trials
Will Chou • 26 Jan 2022
MOMENTUM: ARU-1801 in sickle cell disease 1:57
MOMENTUM: ARU-1801 in sickle cell disease
Will Chou • 26 Jan 2022
ARU-1801, a novel lentiviral gene therapy for SCD 2:26
ARU-1801, a novel lentiviral gene therapy for SCD
Will Chou • 27 Jan 2022
Addressing issues in conducting clinical trials for rare diseases 1:10
Addressing issues in conducting clinical trials for rare diseases
Will Chou • 27 Jan 2022
Key developments in the future of advanced therapies 1:44
Key developments in the future of advanced therapies
Lavakumar Karyampudi • 27 Jan 2022
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris 2:23
DSG3-CAART: A novel CAAR T-cell therapy in pemphigus vulgaris
Samik Basu • 26 Jan 2022
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