Molecular PharmingB.C. MuthubharathiIII B.Sc., Biotechnology

Animal Pharming

• Transgenic animals for human drugsIt is not..

To satisfy our meat requirements

Global demand

• Human proteins and vaccines• Monoclonal antibodies – Antibodies Specifically targeted towards disease proteins

Cystic fibrosis

Hemophilia

Osteoporosis Arthritis

Animal as a bioreactor

• Genetically transformed by splicing and inserting foreign gene ( Human gene) into their chromosome

• Inserted gene express protein through Milk, Urine, Blood, sperm and egg

• 600 transgenic animals Supply worldwide demand of some

pharmaceuticals

(1) A human gene responsible for producing a desired protein is isolated in a laboratory.(2) An animal is given hormonal treatment to produce a large number of embryos, and the embryos are collected from the oviduct.(3) The human gene is inserted into thefertilized egg via micro injection, DNA of the pronucleus is injected into the fertilized embryo.(4) The transgenic embryo is placed in asurrogate host which gives birth to thetransgenic animal.(5) The offspring is tested for the new gene.

Cloning

• Replicating an identical gene, cell or organism from a single ancestor

Disadvantage of traditional method

• Cell and bacterial cultures require constant monitoring and sampling

• Expansion is more costly, because substantial machinery must be purchased and maintained

• Isolating and purifying proteins is more difficult than purifying proteins from an animal’s milk or bodily fluid

Advantages of Molecular pharming

• Naturally carry the cellular mechanism needed to produce complex protein

• Purifing process may become more simple if harvesting proteins from poultry egg and urine

• Unit cost per protein is significantly less

Pharming products currently in development

Animal Drug UseSheep alpha1 anti trypsin deficiency leads to

emphysemasheep factor VIII, IX treatment of hemophiliapig, tissue plasminogen

activatortreatment of thrombosis

goat human protein C treatment of thrombosisgoat glutamic acid

decarboxylasetreatment of type 1 diabetes

goat Pro542 treatment of HIVchicken, cow, goat monoclonal antibodies other vaccine production

Cow

factor VIII

fibrinogen

collagen I, II

lactoferrin

human serum albumin

alpha-lactalbumin

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