The state of gene therapies: The FDA perspective

Molecular Therapy

Volumn 20, Issue 5, 2012, Pages 877-878

  Takefman, Daniel ^a   Bryan, Wilson ^a  

^a CENTER FOR BIOLOGICS EVALUATION AND RESEARCH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE THERAPY AGENT;

CONFIDENTIALITY; DRUG EFFICACY; DRUG MANUFACTURE; DRUG SAFETY; EDITORIAL; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; GOOD MANUFACTURING PRACTICE; INTERNATIONAL COOPERATION;

EID: 84860509150     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.51     Document Type: Editorial

References (5)

1. Hacein-Bey-Abina, S, Von Kalle, C, Schmidt, M, McCormack, MP, Wulffraat, N, Leboulch, P et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419. (Pubitemid 37296260)
2. American Society of Gene & Cell Therapy. NIH Gene Therapy Symposium. Natcher Conference Facility, National Institutes of Health, Bethesda, MD (26-27 September 2011).
3. Sasinowski, F. Quantum of effectiveness evidence in FDA's approval of orphan drugs. Cataloguing FDA's flexibility in regulating therapies for persons with rare disorders (October 2011).
4. Kesselheim, AS, Myers, JA and Avorn, J (2011). Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA 305: 2320-2326.
5. Mitsumoto, J, Dorsey, ER, Beck, CA, Kieburtz, K and Griggs, RC (2009). Pivotal studies of orphan drugs approved for neurological diseases. Ann Neurol 66: 184-190.