scholarly journals PBI4 A LANDSCAPE ASSESSMENT OF CELL AND GENE THERAPY REIMBURSEMENT IN THE UNITED STATES

2019 ◽  
Vol 22 ◽  
pp. S418
Author(s):  
J. Hodgson ◽  
H. Zec ◽  
W. Bedell ◽  
A. Burrell
Keyword(s):  
United States ◽  
Gene Therapy ◽  
The United States ◽  
Landscape Assessment ◽  
Cell And Gene Therapy

scholarly journals Ethical and Practical Considerations for Cell and Gene Therapy Toward an HIV Cure: Findings from a Qualitative In-Depth Interview Study in the United States

2021 ◽  
Author(s):  
Karine Dubé ◽  
John Kanazawa ◽  
Hursch Patel ◽  
Michael Louella ◽  
Laurie Sylla ◽  
...  
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Inductive Reasoning ◽  
The United States ◽  
Global Scale ◽  
Interview Study ◽  
Hiv Cure ◽  
Depth Interview ◽  
Cell And Gene Therapy ◽  
Hiv Cure Research

Abstract Background: HIV cure research involving cell and gene therapy has intensified in recent years. There is a growing need to identify standards, safeguards, and protections to ensure cell and gene therapy HIV cure research remains ethical and acceptable to as many stakeholders as possible as it advances on a global scale.Methods: To elicit ethical and practical considerations to guide cell and gene therapy HIV cure research, we implemented a qualitative, in-depth interview study with three key stakeholder groups in the United States: 1) biomedical HIV cure researchers, 2) bioethicists, and 3) community stakeholders. Interviews were transcribed verbatim. We applied conventional content analysis focused on inductive reasoning to analyze the rich qualitative data and derive key ethical and practical considerations related to cell and gene therapy towards an HIV cure.Results: We interviewed 13 biomedical researchers, 5 community members, and 1 bioethicist. Informants generated considerations related to: perceived benefits of cell and gene therapy towards an HIV cure, perceived risks, considerations necessary to ensure an acceptable benefit/risk balance, cell and gene therapy strategies considered unacceptable, additional ethical considerations, considerations for first-in-human cell and gene therapy HIV cure trials. Informants also proposed important safeguards to developing cell and gene therapy approaches towards an HIV cure, such as the importance of mitigating off-target effects, mitigating risks associated with long-term duration of cell and gene therapy interventions, and mitigating risks of immune overreactions.Conclusion: Rapidly evolving cell and gene therapy towards an HIV cure is accompanied by a host of ethical and practical challenges. To minimize risks to potential participants and facilitate the translation of scientific advancements from the bench to the clinic, cell and gene therapy HIV cure research must be thoughtfully developed and implemented. To protect the public trust in cell and gene therapy HIV cure research, ethical and practical considerations should be periodically revisited and updated as the science continues to evolve.

scholarly journals The United States’ Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research

2019 ◽  
Vol 24 (3) ◽  
pp. 147-152 ◽  
Author(s):  
Daniel Eisenman
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Clinical Trials ◽  
The United States ◽  
Regulatory Environment ◽  
Evidence Based ◽  
Regulatory Structure ◽  
Regulatory Changes ◽  
Current State ◽  
Therapy Field

Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth. Discussion: This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained. Conclusion: The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.

Introduction to Genetics and the Law

2021 ◽  
Author(s):  
Maxwell Mehlman ◽  
Mette Hartlev ◽  
Sonia Suter
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Genetic Testing ◽  
Informed Consent ◽  
Genomic Analysis ◽  
The United States ◽  
Legal Issues ◽  
Legal Doctrine ◽  
The Law ◽  
Material Information

This chapter provides an overview of the relationship between genetics and the law. Advances in genetics and genomics have created both hopes and concerns and raise a number of legal issues in both the United States and Europe. To understand the legal issues in genetics, it is helpful to have some understanding of the different types of genetic analysis that can be used and for which purposes. In virtually all these contexts, the doctrine of informed consent is important. While the law in the United States and Europe does not generally require non-directiveness, the legal doctrine of informed consent applies in all jurisdictions, imposing on physicians and genetic counselors the legal obligation to disclose material information that could influence a patient's decisions about genetic testing. The other ethical and legal issues that arise with genetic or genomic analysis differ depending on the purpose, context, and technology used. These legal issues concern the methods of regulating genetic tests offered in the clinical setting and through the “direct-to-consumer” online genetic testing industry; genetic discrimination; the scope of privacy protections of genetic information; and the regulation of human gene therapy, gene therapy research, and germline modifications.

scholarly journals Prevalence of Neutralizing Antibodies to Adenoviral Serotypes 5 and 35 in the Adult Populations of The Gambia, South Africa, and the United States

2004 ◽  
Vol 11 (2) ◽  
pp. 351-357 ◽  
Author(s):  
Edward Nwanegbo ◽  
Eftyhia Vardas ◽  
Wentao Gao ◽  
Hilton Whittle ◽  
Huijie Sun ◽  
...  
Keyword(s):  
United States ◽  
South Africa ◽  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Fluorescent Proteins ◽  
The United States ◽  
Neutralization Assay ◽  
The Gambia ◽  
Serum Samples ◽  
Lung Carcinoma Cells

ABSTRACT One of the major limitations of the use of adenoviruses as gene therapy vectors is the existence of preformed immunity in various populations. Recent studies have linked failure of adenoviral gene therapy trials to the presence of antiadenoviral neutralizing antibodies (NAb). Understanding the distribution and specificity of such antibodies will assist in the design of successful recombinant adenoviral gene therapies and vaccines. To assess the prevalence of NAb to adenovirus serotypes 5 and 35 (Ad5 and Ad35), we analyzed serum samples from adult immunocompetent individuals living in The Gambia, South Africa, and the United States by using a neutralization assay. Serum samples were incubated with A549 lung carcinoma cells and adenoviruses encoding enhanced green or yellow fluorescent proteins; results were analyzed by fluorescence microscopy and flow cytometry. Using this technique, we found a high prevalence of NAb against Ad5 in Gambian, South African, and U.S. subjects at both low and high titers. Conversely, all subjects displayed a low prevalence of NAb to Ad35; when present, anti-Ad35 NAb were seen at low titers. Because of the ability of adenoviruses to elicit systemic and mucosal immune responses, Ad35 with its low NAb prevalence appears to be an attractive candidate vector for gene therapy applications.

scholarly journals Sponsorship and Funding for Gene Therapy Trials in the United States

JAMA ◽  
2020 ◽  
Vol 323 (9) ◽  
pp. 890
Author(s):  
Zachary Kassir ◽  
Ameet Sarpatwari ◽  
Brian Kocak ◽  
Courtney C. Kuza ◽  
Walid F. Gellad
Keyword(s):  
United States ◽  
Gene Therapy ◽  
The United States

Medicine, Animal Experimentation, and the Moral Problem of Unfortunate Humans

1996 ◽  
Vol 13 (2) ◽  
pp. 181-211 ◽  
Author(s):  
R. G. Frey
Keyword(s):  
United States ◽  
Health Care ◽  
Gene Therapy ◽  
Genetic Engineering ◽  
Technological Innovation ◽  
Research Work ◽  
The United States ◽  
Animal Experimentation ◽  
Moral Problem ◽  
Distant Future

We live in an age of great scientific and technological innovation, and what seemed out of the question or at least very doubtful only a few years ago, today lies almost within our grasp. In no area is this more true than that of human health care, where lifesaving and life-enhancing technologies have given, or have the enormous potential in the not so distant future to give, relief from some of the most terrible human illnesses. On two fronts in particular, xenograft or cross-species transplantation and genetic engineering of animals on behalf of gene therapy in humans, such relief appears very promising, if not actually on the horizon. Certainly, extensive research work on both fronts is underway both in the United States and abroad.

scholarly journals Onasemnogene Abeparvovec-xioi: World’s most expensive and approved therapy for spinal muscular atrophy

2021 ◽  
pp. 145-149
Author(s):  
Vinod Bairagi
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Spinal Muscular Atrophy ◽  
Healthcare Professionals ◽  
Muscular Atrophy ◽  
The United States ◽  
Severe Form ◽  
Expensive Drug ◽  
The U.S ◽  
Experience Difficulty

On May 24, 2019, the U.S. Food and Drug Administration approved Onasemnogene Abeparvovec-xioi (Zolgensma), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. It is priced in the United States at $2.1m (£1.6m; €1.9m) the world’s most expensive drug. Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure. Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. This review summarized the clinical guidelines for the Onasemnogene Abeparvovec-xioi use and may be useful to healthcare professionals.

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