Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Having -c- – wherein x is chalcogen – bonded directly to...
Reexamination Certificate
2004-06-16
2008-12-16
Wilson, James (Department: 1624)
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Having -c-, wherein x is chalcogen, bonded directly to...
C514S266400, C544S284000, C544S291000
Reexamination Certificate
active
07465738
ABSTRACT:
The present invention relates to compounds useful as promoters of the SMN2 gene. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of Spinal Muscular Atrophy.
REFERENCES:
patent: 5304121 (1994-04-01), Sahatjian
patent: 5534518 (1996-07-01), Henrie et al.
patent: 5886026 (1999-03-01), Hunter et al.
patent: 6051605 (2000-04-01), Capiris et al.
patent: 6099562 (2000-08-01), Ding et al.
patent: 6204267 (2001-03-01), Tang et al.
patent: 2002/0137747 (2002-09-01), Moriarty et al.
patent: 2306374 (1974-08-01), None
patent: 204534 (1986-12-01), None
patent: 322390 (1989-06-01), None
patent: 393574 (1990-10-01), None
patent: WO 98/50370 (1998-11-01), None
patent: WO 01/42216 (2001-06-01), None
patent: WO 03/02544 (2003-01-01), None
Brichta et. al., “Valproic acid increases the SMN2 protein level: . . . ”, Human Mol. Gen., 2003, vol. 12, No.19, pp. 2481-2489.
Jablonka, S. et al., “Axonal Defects in Mouse Models of Motoneuron Disease”, J. of Neurobiology, 2004, vol. 58, No. 2, pp. 272-286.
Berge, S.M. et al., “Review Article: Pharmaceutical Salts”,Journal of Pharmaceutical Sciences, vol. 66, No. 1 (Jan. 1977), pp. 1-19.
Brichta, L. et al., “Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy”,Human Molecular Genetics, vol. 12, No. 19 (2003), pp. 2481-2489.
Chan, J.H. et al., “Selective Inhibitors ofCandida albicansDihydrofolate Reductase: Activity and Selectivity of 5-Arylthio-2,4-diaminoquinazolines”,Journal of Medicinal Chemistry, vol. 18, No. 38 (1995), pp. 3608-3616.
Gavrilov, D.K. et al., “Differential SMN2 expression associated with SMA severity”,Nature Genetics, vol. 20 (Nov. 1998), pp. 230-231.
Harris, N.V., “Antifolate and antibacterial activities of 5-substituted 2,4-diaminiquinazolines”,Journal of Medicinal Chemistry, vol. 33, No. 1 (1990), pp. 434-444.
Hsieh-Li, H. et al., “A mouse model for spinal muscular atrophy”,Nature Genetics, vol. 24 (Jan. 2000), pp. 66-70.
Kugelberg, E. et al., “Heredofamilial Juvenile Muscular Atrophy Simulating Muscular Dystrophy”,Arch. Neurol Psychiat., vol. 75 (1956), pp. 500-509.
Lefebvre, S. et al., “The role of the SMN gene in proximal spinal muscular atrophy”,Human Molecular Genetics, vol. 7, No. 10 (1998), pp. 1531-1536.
March's Advanced Organic Chemistry, 5th Ed. Eds: Smith, MB and March, J, John Wiley & Sons, New York 2001, 4 pages.
Monani, U.R. et al., “The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn-/-mice and results in a mouse with spinal muscular atrophy”,Human Molecular Genetics, vol. 9, No. 3 (2000), pp. 333-339.
Nicole, S. et al., “Spinal muscular atrophy: recent advances and future prospects”,Muscle&Nerve, (Jul. 2002), pp. 4-13.
Parsons et al., “Intragenic telSMN Mutations: Frequency, Distribution, Evidence of a Founder Effect, and Modification of the Spinal Muscular Atrophy Phenotype by cenSMN Copy Number”,American Journal of Human Genetics, vol. 63 (1998), pp. 1712-1723.
Periodic Table of the Elements, CAS version, Handbook of Chemistry and Physics, 75 ed.
Petersen, S. et al., “Über Isatin-N-carbonsäureamide und ihre Reaktionen”, Justus Liebigs Annalen Der Chemie, vol. 12 (1974), pp. 2003-2014 (English abstract on p. 2003).
Pharmaceutical Sciences, 16thEd., Ed. W. Martin, Mack Publishing Co., Easton, PA 1980, 3 pages.
Rosowsky, A. et al., “Structure-activity and structure-selectivity studies on diaminoquinazolines and other inhibitors ofPneumocystis cariniiandToxoplasma gondiidihydrolate reductase”,Antimicrobial Agent and Chemotherapy, American Society for Microbiology, Washington, DC, US, vol. 39, No. 1 (Jan. 1995), pp. 79-86.
Sorrell, T.N.,Organic Chemistry, University Science Books, Sausalito CA (1999), 4 pages.
Unangst, P.C. et al., “(Aryloxy)alkylamines as Selective Human Dopamine D4Receptor Antagonists: Potential Antipsychotic Agents”,Journal of Medicinal Chemistry, vol. 40, No. 25 (1997), pp. 4026-4029.
Whitlow, M. et al., “X-ray Crystal Structures of Candida albicans Dihydrofolate Reductase: High Resolution Ternary Complexes in which the Dihydronicotinamide Moiety of NADPH is Displaced by an Inhibitor”,Journal of Medicinal Chemistry, vol. 44, No. 18 (2001), pp. 2928-2932.
Spinal Muscular Atrophy—Families of SMA Home Page [online], 2007 [retrieved on Jan. 3, 2008], 1 page. Retrieved from the Internet: <URL: www.fsma.org>.
Chen Xiaocun
Hurley Dennis James
Jarecki Jill
Makings Lewis R.
Miller Mark T.
Fish & Richardson P.C.
Truong Tamthom N
Vertex Pharmaceuticals Incorporated
Wilson James
LandOfFree
Compounds useful as promoters of SMN2 does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Compounds useful as promoters of SMN2, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Compounds useful as promoters of SMN2 will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-4051929