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MIMM 387 (17)

feb 27 2012.docx

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McGill University
Microbiology and Immun (Sci)
MIMM 387
Robert Murgita

thMonday February 27 2012 Genzymes Mission Statement Missing an enzyme responsible for the breakdown of lipids One complication is a severely enlarged spleen and liver This was a success story but not all of them are The RD Path to a New Therapeutic Research scientists play the largest role in the preclinical research phase INDinvestigational new drug application permission to start a clinical trial NDAnew drug application Even after a drug is accepted there are still more tests to be doneregulation You must regulate the drug in the population because sometimes things come up that were not seen in trials 110 drugs that begin in the preclinical stage end up being approved 2 Basically 10 years to get a drug approved and a 13 billion dollar cost A lot of drugs do not make it past Phase III Sometimes when you decide to invest in Phase III the drug will fail Especially for small companies this is catastrophic Gene Therapy for Cystic Fibrosis Defect in transport of Na and Cl across epithelium This leads to thick viscous secretions and there is an impact in the lungs major pancreas liver and intestines These patients often get a lot of Pseudomonas infections ReplicationDefective Adenovirus Vector Ad2CFTR They made an adenovirus vector to infect the cells in the lung The E1 gene responsible for replication of the vector is taken out to prevent infection of patients and the CFTR gene was put in its place Effective Transduction of Mouse Airways with Ad Vector Betagalactosidase was used as a reporter gene blue colour From the results they knew that they had a good vector to transmit the gene to the airways They then used the actual CFTR vector A number of studies were done Phase I Trial of Recombinant Ad Vector in CF Patients It was safe enough in animals but at this point it is not known if it would be safe in humans They had to construct a plastic chamber for the patient to sit in and the chamber was in a negative pressure room so that the vector did not get out and doctors were all covered up This was because at the time there was a lot of worry about the virus getting out You give one dose if nothing happens you go up a log and stop when you see adverse effects
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