Delivering on siRNA

In June 2009, Ablitech filed for patent protection on:

• Method for protecting siRNA and other oligonucleotides for systemic and local delivery
• Successful incorporation of protected siRNA into cells
• Activity of these oligonucleotides in the target cells

Highlights

• Differentiated from current therapeutic delivery technologies
• NOT encapsulation technology
• No leakage
• Minimum 36 hours of protection in serum
• Clean process which fully restores active oligonucleotides in the cell
• Delivery designed to protect outside the cell and NO reduced efficacy in the cell

How It Works

A thin shell of protection is created by linking a hydrophilic polymer, such as polyethyleneoxide, to the oligonucleotide using very selective chemistry which reverses once inside the cell.

Based on digestion studies, siRNA was fully stable at 36h in 30% fetal calf serum. 

TLC under UV showing Fetal Calf Serum digestion after 36 hours. 

Samples include siRNA (control), siRNA P550 Protected (control),

siRNA (digest), and siRNA P550 Protected (digest).

Uptake into Mouse bladder cancer cells (MB49) has been demonstrated.  Shown below are cells exposed for 20h to

a fluorescent-labeled unprotected oligonucleotide and a fluorescent-labeled protected oligonucleotide.  

 Protected oligo                                Unprotected oligo