Osteoarthritis right now is the most common musculoskeletal disorder affecting 300 million people
Based on the Global Osteoarthritis Patient Perception Survey patients search for pharmacological treatments to replace symptomatologic drugs which in many cases have adverse effects. At the same time, despite any advancements no disease- modifying drug is currently available.
The proposed genetically engineered drug is intended for both types of OA:
• Primary osteoarthritis, presenting with mild, yet disruptive, symptoms, at least for the first years of the disease.
• Secondary osteoarthritis, caused by a pre-existing joint abnormality and characterized by severe symptoms.
Real Word Implementation
After the end of the iGEM phase of the project we envisage the following course of actions in order
for the solution to reach the market:
Finalize the design and discovery – Duration: 2 years
The wetlab experiments will be completed and most likely complemented and repeated to achieve at least 2 more engineering cycles before viable success. More in silico experiments throughout this process.
Design and discovery of delivery methods – Duration: 2 years
Laboratory and in silico research to design delivery method, considering first injectable form and researching the possibility for transdermal diffusion
Preclinical research – Duration : 2 years
Laboratory and animal testing to answer basic questions about efficacy, toxicity and pharmacokinetics (dosage undefined)
Phase 0 – Duration : 1 year
Clinical study on 10 healthy human volunteers with subtherapeutic dose to study pharmacokinetics and half-life of the drug
Phase I – Duration: 1 year
Clinical study on 20-100 healthy human volunteers to assess safety and dosage, using ascending quantities of sub-therapeutic doses
Phase II – Duration: 2 years
Clinical study on 100-300 patient volunteers to assess efficacy and side effects with therapeutic doses.
Phase III – Duration: 3 years
Clinical study on 2,000 patient volunteers to assess efficacy and monitor adverse reactions with therapeutic doses. Successful completion would lead to a pre-approval.
Phase IV – Duration: long term
Large scale manufacturing. Post market long term monitoring on several thousand patient volunteers to assess long term safety and efficacy.
Administration of Final Drug
The final drug would be incorporated in a therapeutic procedure as shown in the graphic.
Major safety risks
• Safety of the drug will be assessed via rigorous preclinical and clinical trials as described above.
• Additional safety assessment will consider potential risks due to the delivery method, either an injectable hydrogel or patch for transdermal delivery.
• Regarding the genetic circuit itself, further studies are needed to elucidate whether the increase in local expression of the genes selected for the effect leads to a malfunction or abnormality.
• Fully apply and comply to GMO regulations.
• Increase efficiency. The insertion of the present genetic circuit into mesenchymal stem cells seems to be inadequately efficient. This, combined with the cell survival through transportation from the patient to the company and back could reflect in reduced efficiency.
• Keep costs low so as to ensure the final product is affordable.