A snapshot of biologic drug development

Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in the toxicological evaluation of potential therapeutics

(2015) James M. Murray et al.   MOLECULAR GENETICS AND METABOLISM

Oligonucleotide delivery: a patent review (2010 – 2013)

(2014) Santiago Grijalvo et al.   EXPERT OPINION ON THERAPEUTIC PATENTS

Gene Therapies for Cancer: Strategies, Challenges and Successes

(2014) Swadesh K. Das et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

(2014) Stéphane Palfi et al.   LANCET

Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis

(2014) Maria Chiriaco et al.   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antisense Oligonucleotide Therapies

(2014) Kendall S. Frazier   TOXICOLOGIC PATHOLOGY

Clinical Development of Gene Therapy Needs a Tailored Approach: A Regulatory Perspective from the European Union

(2014) Gopalan Narayanan et al.   Human Gene Therapy Clinical Development

Gene Therapy for Blindness

(2013) José-Alain Sahel et al.   Annual Review of Neuroscience

Long-Term Effects of AAV1/SERCA2a Gene Transfer in Patients With Severe Heart Failure

(2013) Krisztina Zsebo et al.   CIRCULATION RESEARCH

History of gene therapy

(2013) Thomas Wirth et al.   GENE

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia

(2013) R. J. Brentjens et al.   Science Translational Medicine

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

(2013) Josep Tabernero et al.   Cancer Discovery

Antisense oligonucleotides on neurobehavior, respiratory, and cardiovascular function, and hERG channel current studies

(2013) Tae-Won Kim et al.   JOURNAL OF PHARMACOLOGICAL AND TOXICOLOGICAL METHODS

Antisense inhibition of coagulation factor XI prolongs APTT without increased bleeding risk in cynomolgus monkeys

(2012) H. S. Younis et al.   BLOOD

Gene therapy for primary immunodeficiencies: Part 2

(2012) Alessandro Aiuti et al.   CURRENT OPINION IN IMMUNOLOGY

Monoclonal antibody therapeutics: history and future

(2012) Nicholas APS Buss et al.   CURRENT OPINION IN PHARMACOLOGY

Immunogenicity of different stressed IgG monoclonal antibody formulations in immune tolerant transgenic mice

(2012) Vasco Filipe et al.   mAbs

Use of Animal Models of Human Disease for Nonclinical Safety Assessment of Novel Pharmaceuticals

(2012) Sherry J. Morgan et al.   TOXICOLOGIC PATHOLOGY

Current progress of siRNA/shRNA therapeutics in clinical trials

(2011) John C. Burnett et al.   Biotechnology Journal

Therapeutic Oligonucleotides: The Road Not Taken

(2011) C. A. Stein et al.   CLINICAL CANCER RESEARCH

Making sense of therapeutics using antisense technology

(2011) Ravinder Malik et al.   Expert Opinion on Drug Discovery

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Current prospects for RNA interference-based therapies

(2011) Beverly L. Davidson et al.   NATURE REVIEWS GENETICS

Monoclonal antibody TGN1412 trial failure explained by species differences in CD28 expression on CD4+ effector memory T-cells

(2010) D Eastwood et al.   BRITISH JOURNAL OF PHARMACOLOGY

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

(2010) A. Joseph et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Preclinical development of monoclonal antibodies

(2010) Kathryn Chapman et al.   mAbs

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

siRNA vs. shRNA: Similarities and differences

(2009) Donald D. Rao et al.   ADVANCED DRUG DELIVERY REVIEWS

Juvenile animal toxicity study designs to support pediatric drug development

(2009) Gregg D. Cappon et al.   BIRTH DEFECTS RESEARCH PART B-DEVELOPMENTAL AND REPRODUCTIVE TOXICOLOGY

Alternative Strategies for Toxicity Testing of Species-Specific Biopharmaceuticals

(2009) Jeanine L. Bussiere et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Immunogenicity of biologically-derived therapeutics: Assessment and interpretation of nonclinical safety studies

(2009) Rafael Ponce et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Nonviral Methods for siRNA Delivery

(2008) Kun Gao et al.   MOLECULAR PHARMACEUTICS