Bioprocess solutions to assess clinical biodistribution in cell/gene therapies
Sign Up to like & getrecommendations! Published in 2020 at "Cytotherapy"
DOI: 10.1016/j.jcyt.2020.03.204
Abstract: Background & Aim Lack of comprehensive product knowledge in clinical applications may result in toxicity or reduced drug efficacy. The distributive fate and retention of cell/gene therapies is rarely evaluated, with very few studies assessing… read more here.
Keywords: biodistribution; product; cell gene; bioprocess solutions ... See more keywords
PRO103 The IMPACT of COVID-19 on the Future Pricing and Market Access of RARE Diseases and CELL-/GENE-Therapies
Sign Up to like & getrecommendations! Published in 2020 at "Value in Health"
DOI: 10.1016/j.jval.2020.08.1839
Abstract: Objectives: To explore the long-term impact of Covid-19 on the pricing, market access, and payer evidence requirements in rare diseases and cell/gene-therapies Methods: A survey-based Covid-19 payer impact tracker covering US and EU (n= 80… read more here.
Keywords: impact; gene therapies; cell gene; pricing market ... See more keywords
Implications of hematopoietic stem cells heterogeneity for gene therapies
Sign Up to like & getrecommendations! Published in 2021 at "Gene Therapy"
DOI: 10.1038/s41434-021-00229-x
Abstract: Hematopoietic stem cell transplantation (HSCT) is the therapeutic concept to cure the blood/immune system of patients suffering from malignancies, immunodeficiencies, red blood cell disorders, and inherited bone marrow failure syndromes. Yet, allogeneic HSCT bear considerable… read more here.
Keywords: cell; heterogeneity; hematopoietic stem; gene ... See more keywords
Patient and public perspectives on cell and gene therapies: a systematic review
Sign Up to like & getrecommendations! Published in 2020 at "Nature Communications"
DOI: 10.1038/s41467-020-20096-1
Abstract: Cell and gene therapies offer opportunities for treating disease with potential to restore function, and cure disease. However, they are not without risk and pose complex logistical, economic, ethical and social challenges for health systems.… read more here.
Keywords: patient public; systematic review; cell gene; gene ... See more keywords
Benefit Corporation: a path to affordable gene therapies?
Sign Up to like & getrecommendations! Published in 2019 at "Nature Medicine"
DOI: 10.1038/s41591-019-0676-z
Abstract: With the growing number of efficient gene therapies on the market, now is the time to take actions to ensure reasonable pricing of gene therapy products. Among these, we propose to incentivize gene therapy companies… read more here.
Keywords: corporation path; benefit corporation; affordable gene; gene ... See more keywords
A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies
Sign Up to like & getrecommendations! Published in 2017 at "Human Gene Therapy Methods"
DOI: 10.1089/hgtb.2017.096
Abstract: Lentiviral vectors are increasingly the gene transfer tool of choice for gene or cell therapies, with multiple clinical investigations showing promise for this viral vector in terms of both safety and efficacy. The third-generation vector… read more here.
Keywords: gene therapy; guide approaching; approaching regulatory; gene ... See more keywords
A Retinal Research Nonprofit Paves the Way for Commercializing Gene Therapies
Sign Up to like & getrecommendations! Published in 2017 at "Human Gene Therapy"
DOI: 10.1089/hum.2017.29058.bsh
Abstract: AN EMERGING, vision-restoring gene therapy for a devastating retinal disease is poised for Food and Drug Administration (FDA) approval. If it gets the regulatory nod, it will be the first gene therapy to receive FDA… read more here.
Keywords: research; treatment; gene therapy; gene ... See more keywords
Regulatory Consideration for the Nonclinical Safety Assessment of Gene Therapies.
Sign Up to like & getrecommendations! Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2022.090
Abstract: The nonclinical safety assessments for gene therapies are evolving, leveraging over 20 years of experimental and clinical experience. Despite the growing experience with these therapeutics, there are no approved harmonized global regulatory documents for developing… read more here.
Keywords: safety; gene therapies; nonclinical safety; gene therapy ... See more keywords
Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency.
Sign Up to like & getrecommendations! Published in 2023 at "Medical care"
DOI: 10.1097/mlr.0000000000001840
Abstract: Background: Gene therapy, altering the genes inside human cells, has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies.… read more here.
Keywords: european medicines; food drug; gene therapies; drug administration ... See more keywords
Health technology assessment for gene therapies in haemophilia
Sign Up to like & getrecommendations! Published in 2022 at "Haemophilia"
DOI: 10.1111/hae.14413
Abstract: Gene therapies are poised to become a new therapeutic option for persons with haemophilia (PwH), having begun to show durable efficacy and safety in clinical trials to date. However, value assessment of gene therapies faces… read more here.
Keywords: health technology; technology assessment; gene; haemophilia health ... See more keywords
Emerging gene therapies for enhancing the hemostatic potential of platelets
Sign Up to like & getrecommendations! Published in 2021 at "Transfusion"
DOI: 10.1111/trf.16519
Abstract: Platelet transfusions are an integral component of balanced hemostatic resuscitation protocols used to manage severe hemorrhage following trauma. Enhancing the hemostatic potential of platelets could lead to further increases in the efficacy of transfusions, particularly… read more here.
Keywords: enhancing hemostatic; hemostatic potential; hemorrhage; potential platelets ... See more keywords