Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders

Current Treatment Options in Neurology—SMA Therapeutics

(2019) Megan A. Waldrop et al.   Current Treatment Options in Neurology

Oncorine, the World First Oncolytic Virus Medicine and its Update in China

(2018) Min Liang   CURRENT CANCER DRUG TARGETS

A guide to manufacturing CAR T cell therapies

(2018) Philipp Vormittag et al.   CURRENT OPINION IN BIOTECHNOLOGY

Pegaptanib: choroidal neovascularization in patients with age-related macular degeneration and previous arterial thromboembolic events

(2018) Maurizio Battaglia Parodi et al.   EUROPEAN JOURNAL OF OPHTHALMOLOGY

CAR-T: trailblazing the path from clinical development to the clinic

(2018) Samuele Butera   GENE THERAPY

The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic

(2018) Wei-Wei Zhang et al.   HUMAN GENE THERAPY

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

(2018) Maria Pia Cicalese et al.   MOLECULAR THERAPY

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

(2018) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

INVOSSA, a first-in-class of cell and gene therapy for osteoarthritis treatment: the phase III trial

(2018) B. Lee   OSTEOARTHRITIS AND CARTILAGE

INVOSSA-K induces an anti-inflammatory environment in a rat mia model via macrophage polarization

(2018) H. Lee et al.   OSTEOARTHRITIS AND CARTILAGE

Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

(2018) Marina França Dias et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Gene therapy: evidence, value and affordability in the US health care system

(2018) Grace Hampson et al.   Journal of Comparative Effectiveness Research

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

The Natural History of Inherited Retinal Dystrophy due to Biallelic Mutations in the RPE65 Gene

(2018) Daniel C. Chung et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

FDA approves patisiran to treat hereditary transthyretin amyloidosis

(2018) Heather Wood   Nature Reviews Neurology

Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy

(2018) Stephen Russell et al.   Expert Opinion on Orphan Drugs

Gene therapy and the adeno-associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations

(2018) Charmaine A. Ramlogan-Steel et al.   CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Gene therapy for RPE65-related retinal disease

(2018) Virginia Miraldi Utz et al.   OPHTHALMIC GENETICS

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

(2018) Samiah Al‐Zaidy et al.   PEDIATRIC PULMONOLOGY

Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease

(2018) Vamshi K. Rao et al.   Journal of Managed Care & Specialty Pharmacy

Efficacy and safety of nusinersen in infants with presymptomatic spinal muscular atrophy (SMA): Interim results from the NURTURE study

(2017) E. Bertini et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones

(2017) Jerry R. Mendell et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

Spinraza—a rare disease success story

(2017) V Prakash   GENE THERAPY

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug

(2017) Francesca Ferrua et al.   HUMAN GENE THERAPY

Transthyretin familial amyloid polyneuropathy: an update

(2017) Violaine Plante-Bordeneuve   JOURNAL OF NEUROLOGY

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Axicabtagene ciloleucel, a first-in-class CAR T cell therapy for aggressive NHL

(2017) Zachary J. Roberts et al.   LEUKEMIA & LYMPHOMA

Viral vanguard: Designing cancer-killing viruses to chase metastatic tumors

(2017) Shraddha Chakradhar   NATURE MEDICINE

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

2016 EMA drug approval recommendations

(2017) Asher Mullard   NATURE REVIEWS DRUG DISCOVERY

Efficacy and safety of nusinersen in children with later-onset spinal muscular atrophy (SMA): end of study results from the phase 3 CHERISH study

(2017) E. Mercuri et al.   NEUROMUSCULAR DISORDERS

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

(2017) Sattva S. Neelapu et al.   NEW ENGLAND JOURNAL OF MEDICINE

Invossa™ (TISSUEGENE-C) induces an anti-inflammatory environment in the arthritic knee joints via macrophage polarization

(2017) K. Choi et al.   OSTEOARTHRITIS AND CARTILAGE

Chimeric antigen receptor T-cell therapy for glioblastoma

(2017) Analiz Rodriguez et al.   Translational Research

Acute lymphoblastic leukemia: a comprehensive review and 2017 update

(2017) T Terwilliger et al.   Blood Cancer Journal

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Nusinersen for the treatment of spinal muscular atrophy

(2017) Claudia A. Chiriboga   Expert Review of Neurotherapeutics

Transthyretin familial amyloid polyneuropathy (TTR-FAP): Parameters for early diagnosis

(2017) Fabiola Escolano-Lozano et al.   Brain and Behavior

The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant

(2017) David L. Veenstra et al.   JOURNAL OF MEDICAL ECONOMICS

A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolgus monkeys

(2017) Michael St. Andre et al.   Skeletal Muscle

CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes

(2017) Eric Ehrke-Schulz et al.   Scientific Reports

Talimogene Laherparepvec (T-VEC) and Other Oncolytic Viruses for the Treatment of Melanoma

(2016) Praveen K. Bommareddy et al.   AMERICAN JOURNAL OF CLINICAL DERMATOLOGY

Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

(2016) James S. Butler et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research

(2016) Christopher Strouse et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure

(2016) P. G. Richardson et al.   BLOOD

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

(2016) Maria Pia Cicalese et al.   BLOOD

FDA approves new treatment of metastatic melanoma

(2016) Carrie Printz   CANCER

Oncolytic virus therapy: A new era of cancer treatment at dawn

(2016) Hiroshi Fukuhara et al.   CANCER SCIENCE

Current and Future Treatment Approaches in Transthyretin Familial Amyloid Polyneuropathy

(2016) Philippe Kerschen et al.   Current Treatment Options in Neurology

TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies

(2016) David Adams et al.   EXPERT OPINION ON PHARMACOTHERAPY

How to select the best available related or unrelated donor of hematopoietic stem cells?

(2016) J.-M. Tiercy   HAEMATOLOGICA

Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis

(2016) Daniel Gaudet et al.   HUMAN GENE THERAPY

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

Considerations and challenges for patients with refractory and relapsed acute myeloid leukaemia

(2016) Jonathan Kell   LEUKEMIA RESEARCH

Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice

(2016) Cy A Stein   MOLECULAR THERAPY

Big win possible for Ionis/Biogen antisense drug in muscular atrophy

(2016) Ken Garber   NATURE BIOTECHNOLOGY

Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials

(2016) Eugenio Mercuri et al.   NEUROMUSCULAR DISORDERS

How to select the best available related or unrelated donor of hematopoietic stem cells?

(2016) J.-M. Tiercy   HAEMATOLOGICA

Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency

(2016) Kathryn V. Whitmore et al.   Frontiers in Immunology

Clinical development of gene therapy: results and lessons from recent successes

(2016) Sandeep RP Kumar et al.   Molecular Therapy-Methods & Clinical Development

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Exon skipping therapy for Duchenne muscular dystrophy

(2015) Ryszard Kole et al.   ADVANCED DRUG DELIVERY REVIEWS

Mipomersen, an Antisense Oligonucleotide to Apolipoprotein B-100, Reduces Lipoprotein(a) in Various Populations With HypercholesterolemiaSignificance

(2015) Raul D. Santos et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Haploidentical HSCT: a 15-year experience at San Raffaele

(2015) C Bonini et al.   BONE MARROW TRANSPLANTATION

Sinusoidal obstruction syndrome/veno-occlusive disease: current situation and perspectives—a position statement from the European Society for Blood and Marrow Transplantation (EBMT)

(2015) M Mohty et al.   BONE MARROW TRANSPLANTATION

Molecular Pathways: Mechanism of Action for Talimogene Laherparepvec, a New Oncolytic Virus Immunotherapy

(2015) F. J. Kohlhapp et al.   CLINICAL CANCER RESEARCH

Intracellular delivery of VEGF165 encoding gene therapeutic using trifunctional copolymers of ethylene oxide and propylene oxide

(2015) Oksana Bondar et al.   EUROPEAN POLYMER JOURNAL

TRUCKs: the fourth generation of CARs

(2015) Markus Chmielewski et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Adoptive therapy with CAR redirected T cells: the challenges in targeting solid tumors

(2015) Hinrich Abken   Immunotherapy

Talimogene laherparepvec (T-VEC) for the treatment of advanced melanoma

(2015) Douglas B Johnson et al.   Immunotherapy

pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia

(2015) Roman V. Deev et al.   JOURNAL OF CARDIOVASCULAR PHARMACOLOGY AND THERAPEUTICS

Talimogene Laherparepvec Improves Durable Response Rate in Patients With Advanced Melanoma

(2015) Robert H.I. Andtbacka et al.   JOURNAL OF CLINICAL ONCOLOGY

Genome Editing Technologies: Defining a Path to Clinic

(2015) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

Spinal Muscular Atrophy Therapeutics: Where do we Stand?

(2015) Constantin d’Ydewalle et al.   Neurotherapeutics

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

(2015) Ole B Suhr et al.   Orphanet Journal of Rare Diseases

Tissuegene-C (INVOSSA™) in patients with osteoarthritis: A phase II trials

(2015) J. Cho et al.   OSTEOARTHRITIS AND CARTILAGE

Preliminary results of a phase II randomized study to determine the efficacy and safety of genetically engineered allogeneic human chondrocytes expressing TGF-β1 in patients with grade 3 chronic degenerative joint disease of the knee

(2015) J.J. Cherian et al.   OSTEOARTHRITIS AND CARTILAGE

Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides

(2015) Umar Burki et al.   Nucleic Acid Therapeutics

Spinal Muscular Atrophy: Therapeutic Strategies

(2014) Diana Castro et al.   Current Treatment Options in Neurology

Key points of basic theories and clinical practice in rAd-p53 (Gendicine™) gene therapy for solid malignant tumors

(2014) Yi Li et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Clinical utility of recombinant adenoviral human p53 gene therapy: current perspectives

(2014) Guang-Xia Chen et al.   OncoTargets and Therapy

Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis

(2014) Wan Ling Wong et al.   Lancet Global Health

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

(2014) Valerie Ferreira et al.   Frontiers in Immunology

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Molecular Therapeutic Strategies for Spinal Muscular Atrophies: Current and Future Clinical Trials

(2013) Chiara Zanetta et al.   CLINICAL THERAPEUTICS

Diagnosis and treatment of familial hypercholesterolaemia

(2013) G. Kees Hovingh et al.   EUROPEAN HEART JOURNAL

Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)

(2013) Stuart J Moat et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Of CARs and TRUCKs: chimeric antigen receptor (CAR) T cells engineered with an inducible cytokine to modulate the tumor stroma

(2013) Markus Chmielewski et al.   IMMUNOLOGICAL REVIEWS

Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?

(2013) Monir Shababi et al.   JOURNAL OF ANATOMY

Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Reduces Atherogenic Lipoproteins in Patients With Severe Hypercholesterolemia at High Cardiovascular Risk

(2013) Gregory S. Thomas et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   MUSCLE & NERVE

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

Homozygous familial hypercholesterolemia: Current perspectives on diagnosis and treatment

(2012) Frederick J. Raal et al.   ATHEROSCLEROSIS

T-cell suicide gene therapy prompts thymic renewal in adults after hematopoietic stem cell transplantation

(2012) L. Vago et al.   BLOOD

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency

(2012) A. Hassan et al.   BLOOD

Clinical pharmacological properties of mipomersen (Kynamro), a second generation antisense inhibitor of apolipoprotein B

(2012) Stanley T. Crooke et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Mipomersen

(2012) Daniel N. Ricotta et al.   Cardiology in Review

Apolipoprotein B Synthesis Inhibition With Mipomersen in Heterozygous Familial Hypercholesterolemia

(2012) Evan A. Stein et al.   CIRCULATION

Gene therapy for lipoprotein lipase deficiency

(2012) Daniel Gaudet et al.   CURRENT OPINION IN LIPIDOLOGY

Drug safety evaluation of defibrotide

(2012) Paul G Richardson et al.   Expert Opinion On Drug Safety

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Defibrotide for prophylaxis of hepatic veno-occlusive disease in paediatric haemopoietic stem-cell transplantation: an open-label, phase 3, randomised controlled trial

(2012) Selim Corbacioglu et al.   LANCET

Stem cells in severe infantile spinal muscular atrophy

(2012) Eugenio Mercuri et al.   NEUROMUSCULAR DISORDERS

Randomized, Placebo-Controlled Trial of Mipomersen in Patients with Severe Hypercholesterolemia Receiving Maximally Tolerated Lipid-Lowering Therapy

(2012) Mary P. McGowan et al.   PLoS One

Defibrotide Interferes With Several Steps of the Coagulation-Inflammation Cycle and Exhibits Therapeutic Potential to Treat Severe Malaria

(2011) Ivo M.B. Francischetti et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Familial Hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients

(2011) Anne C. Goldberg et al.   Journal of Clinical Lipidology

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease and Multiorgan Failure after Stem Cell Transplantation: A Multicenter, Randomized, Dose-Finding Trial

(2010) Paul G. Richardson et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Rexin-G, a targeted genetic medicine for cancer

(2010) Erlinda M Gordon et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

(2010) Frederick J Raal et al.   LANCET

A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

(2010) Natalia N. Singh et al.   RNA Biology

Local and Distant Immunity Induced by Intralesional Vaccination with an Oncolytic Herpes Virus Encoding GM-CSF in Patients with Stage IIIc and IV Melanoma

(2009) Howard L. Kaufman et al.   ANNALS OF SURGICAL ONCOLOGY

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA

(2009) Virginia B Mattis et al.   BMC NEUROSCIENCE

Oral Administration of the Thyrotropin-Releasing Hormone (TRH) Analogue, Taltireline Hydrate, in Spinal Muscular Atrophy

(2009) Zenichiro Kato et al.   JOURNAL OF CHILD NEUROLOGY

Phase II Clinical Trial of a Granulocyte-Macrophage Colony-Stimulating Factor–Encoding, Second-Generation Oncolytic Herpesvirus in Patients With Unresectable Metastatic Melanoma

(2009) Neil N. Senzer et al.   JOURNAL OF CLINICAL ONCOLOGY

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I–II study

(2009) Fabio Ciceri et al.   LANCET ONCOLOGY

Advanced Phase I/II Studies of Targeted Gene Delivery In Vivo: Intravenous Rexin-G for Gemcitabine-resistant Metastatic Pancreatic Cancer

(2009) Sant P Chawla et al.   MOLECULAR THERAPY

Introduction: Understanding the Role of Angiogenesis and Antiangiogenic Agents in Age-Related Macular Degeneration

(2009) Susan B. Bressler   OPHTHALMOLOGY

Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100

(2008) Rosie Z. Yu et al.   BIOCHEMICAL PHARMACOLOGY

Ranibizumab versus Verteporfin Photodynamic Therapy for Neovascular Age-Related Macular Degeneration: Two-Year Results of the ANCHOR Study

(2008) David M. Brown et al.   OPHTHALMOLOGY

The Natural History and Prognosis of Neovascular Age-Related Macular Degeneration

(2007) Tien Wong et al.   OPHTHALMOLOGY