Biotech Product Proposal

Jenna Beales, Sasha Uban, Nick Sullivan, Tyler Abell

Problem (2-3 paragraphs)
Hunter syndrome currently has no cure. It is usually fatal, and very hard to catch
symptoms at a young age. It is hereditary, passed down from the mothers side, but only affects
males, due to the son only inheriting the damaged X chromosome from his mother. Girls escape
the effects of the disease because they inherit a healthy X chromosome as well as the damaged
one, and that healthy X chromosome is able to function properly.
In this disease, the enzyme iduronate sulfatase is not produced properly, or is not
produced at all. Because of the malfunctioning or missing enzyme, complicated sugars called
mucopolysaccharides, more specifically the sugars dermatan and heparan sulphate, are not
broken down and instead build up and affect many of the bodys systems.
The respiratory system can be blocked due to build up of tissues and sleep apnea is a
common issue. In the heart, cardiovascular complications such as high blood pressure, heart
murmur, and leaky heart valves are symptoms. Most people with Hunter syndrome dont heal
well and often have complications after surgery. This makes treatment for skeletal and
connective tissue problems very difficult. Joint flexibility and motion is often impacted and
impaired. Problems associated with the buildup of fluid and tissue around the brain and spinal
cord are difficult to address because of the risks of treating these parts of the body that already
exist. Seizures may be an eventual symptom of Hunter syndrome.
Previous attempted solutions (2-4 paragraphs)
Previous attempts have included long-term enzyme replacement therapy with
Idursulfase medications delivered weekly through an IV. Unfortunately, once the treatment can
be termed long-term, anti-idursulfase serum antibodies build up in the immune system and the
treatment becomes much less effective. Because this is such a new area of study and
medication, observations are still being made pertaining to the results of this drug and
treatment.
Some of the side effects already observed in reaction to introducing idursulfase into
patients bodies include serious allergic reactions, headache, fever, skin reactions and high
blood pressure. Side effects have been seen to lesson over time, but the positive results of the
treatment in regards to the enzyme replacement have also been seen to become less effective.

Current limitations/present need (2-3 paragraphs)

Biotechnology and its applications in medication is still relatively new, so scientists are
still in the beginning stages of attempting to find cures with this new technology. Because of this,
most of the developments in this field are still being studied for negative side effects or longterm results.
Hunter syndrome has not been cured yet, and treatments for it only limit the side effects.
Some side effects that could arise would be a bad reaction to the product's introduction to the
patients immune systems. It is also not clear whether the enzyme iduronate sulfatase can be
produced in a similar way to insulin.
Your proposed/product (2-5 paragraphs)
We would use Chinese hamster ovary cells (abbreviated as CHO cells) in cell
production lines, frozen in a cell bank or storage facility prior to use. These would provide the
starter material for the genetic engineering process. To produce a the iduronate sulfatase
enzyme, the gene for that enzyme would be obtained from human DNA and inserted into the
CHO cells, causing them to express or "manufacture" the enzyme. Next we would purify the
enzyme, so that unwanted substances are removed.The next step would be to prove that our
product contains the correct enzyme. To do this, we would use the techniques of
chromatography to check that our product is made of the correct substances. Then we would
conduct tests to make sure that the iduronate sulfatase enzyme was functioning properly. To do
this, we would use test tubes containing the mucopolysaccharides and add our product
consisting of the iduronate sulfatase enzyme. If our product breaks down the sugar we would
then know that our product functioned as intended. Afterwards, the product would be ready for
development into a final product and would then be transported to a sterile area, where it would
be put into its final packaging. In the last stage of the production process, we would fill the
manufactured enzyme into vials using a sterile manufacturing process to prevent product
contamination. At this point, the vials would be ready to be sealed, labeled, and inspected. Only
after Quality Assurance (QA) approval would the iduronate sulfatase enzyme be shipped from
the manufacturing facility.
Once our product has been made, the next stage is to introduce it to patients. Our
product would be given once weekly by intravenous (IV) infusion, meaning that the medication
would be delivered directly into your bloodstream, and from there to the bodys cells and organs
to break down the mucopolysaccharide buildup. Treatments would typically be expected to last
about three to four hours. The patient would receive an infusion once a week. A healthcare

professional could possibly prescribe pretreatment medications, such as antihistamines or antifever therapies, to reduce the symptoms of any reactions that could occur during an infusion.
These medications would be taken around 60 minutes before the start of the infusion. The IV
would then be set up and the healthcare professional would typically prescribe the infusion to
drip slowly into the vein for about three to four hours. During this time the healthcare
professional would check vital signs and again when the IV needle has been removed and the
process has ended.
Citations (at least 5 credible, valuable sources of information)
"How Enzymes Are Made." Aldurazyme. Ed. NA. Genzyme, 2016. Web. 17 May 2016.
Hunter Syndrome. Treatment. Ed. NA. Mayo Foundation for Medical Education and Research
10 Dec. 2015. Web. 05 May 2016.
Li, Shuang, Xiaofeng Yang, Shuai Yang, Muzi Zhu, and Xiaoning Wang. Technology
Prospecting
on Enzymes: Application, Marketing and Engineering. Computational and Structural
Biotechnology Journal. Research Network of Computational and Structural Biology
(RNCSB) organization, 9 Nov. 2012. Web. 05 May 2016.
Miller-Keane Encyclopedia and Dictionary of Medicine, Nursing, and Allied Health, Seventh
Edition. 2003 by Saunders, an imprint of Elsevier, Inc. All rights reserved.
MPS ll. MPS Society. Ed. NA. National MPS Society. 04 May 2011. Web. 05 May 2016.
Neufeld, Elizabeth F. Enzyme Replacement Therapy - A Brief History. Enzyme Replacement
Therapy - A Brief History. U.S. National Library of Medicine, 2006. Web. 05 May 2016.
Virtual mentor and credentials
We sent an email to a genetic consultant and are waiting for a reply.