Evidence generation and reproducibility in cell and gene therapy research: A call to action

Regenerative medicine clinical readiness

(2021) Satsuki Yamada et al.   Regenerative Medicine

“Dangling” Accelerated Approvals in Oncology

(2021) Julia A. Beaver et al.   NEW ENGLAND JOURNAL OF MEDICINE

ISSCR Guidelines for Stem Cell Research and Clinical Translation: The 2021 update

(2021) Robin Lovell-Badge et al.   Stem Cell Reports

Enhancing Chimeric Antigen Receptor T cell Efficacy in Solid Tumors

(2020) Giovanni Fucá et al.   CLINICAL CANCER RESEARCH

CAR T-cell product performance in haematological malignancies before and after marketing authorisation

(2020) Magdi Elsallab et al.   LANCET ONCOLOGY

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Senolytic CAR T Cells in Solid Tumors and Age-Related Pathologies

(2020) Judith Feucht et al.   MOLECULAR THERAPY

Recalibrating Health Technology Assessment Methods for Cell and Gene Therapies

(2020) Aris Angelis et al.   PHARMACOECONOMICS

Single cell transcriptomics comes of age

(2020) Sarah Aldridge et al.   Nature Communications

Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals

(2020) Magdi Elsallab et al.   Molecular Therapy-Methods & Clinical Development

Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges

(2019) Marina Cavazzana et al.   NATURE REVIEWS DRUG DISCOVERY

Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

(2019) Erik Tambuyzer et al.   NATURE REVIEWS DRUG DISCOVERY

Enhancing patient-level clinical data access to promote evidence-based practice and incentivize therapeutic innovation

(2018) Alice Fortunato et al.   ADVANCED DRUG DELIVERY REVIEWS

Registry Contributions to Strengthen Cell and Gene Therapeutic Evidence

(2018) Mohamed Abou-El-Enein et al.   MOLECULAR THERAPY

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

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

Concise Review: A Comprehensive Analysis of Reported Adverse Events in Patients Receiving Unproven Stem Cell-Based Interventions

(2018) Gerhard Bauer et al.   Stem Cells Translational Medicine

How to design preclinical studies in nanomedicine and cell therapy to maximize the prospects of clinical translation

(2018) John P. A. Ioannidis et al.   Nature Biomedical Engineering

Human tissues in a dish: The research and ethical implications of organoid technology

(2017) Annelien L. Bredenoord et al.   SCIENCE

Modeling Development and Disease with Organoids

(2016) Hans Clevers   CELL

Overcoming Challenges Facing Advanced Therapies in the EU Market

(2016) Mohamed Abou-El-Enein et al.   Cell Stem Cell

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Assessing and reporting heterogeneity in treatment effects in clinical trials: a proposal

(2010) David M Kent et al.   Trials