Sanofi scientists and bioengineers are now investigating three new approaches to hemophilia treatment: an extended half-life factor replacement therapy, a rebalancing RNA Interference (RNAi) therapy, and a potentially curative gene therapy.
- Factor replacement therapy, the cornerstone of hemophilia care, replaces missing blood-clotting factors in the body and requires intravenous injections. Together with Sobi, Sanofi is taking a unique bioengineering approach to extended half-life factor replacement therapy, aiming to provide high, sustained protection against bleeds. With this uniquely designed molecule, the companies’ goal is to provide near normal factor levels for more than half the week.
- Rebalancing RNAi therapy, a new approach under investigation in the clinic, aims to rebalance pro-coagulant and anti-coagulant proteins in the blood that regulate clotting. RNA is responsible for translating DNA instructions into proteins in the body. Sanofi researchers are using small, “interfering” RNA molecules (RNAi) to prevent the body from manufacturing a protein that inhibits coagulation. Their ambition is to develop a once-monthly, subcutaneous, single-dose treatment that could be used for hemophilia A and B, with or without inhibitors (antibodies that attack replacement factors).2
- Gene therapies ultimately aim to cure patients by reprogramming their DNA to produce the missing factor. This requires delivering corrected DNA to a patient’s cells using a retrovirus.
Sanofi researchers are exploring how to deliver corrected DNA to liver cells using a type of retrovirus called a lentivirus. Their goal is to create a gene therapy that would be durable and effective in all patients, including children. This approach is still in the experimental phase, and it will be many years before patients can benefit from it, and until scientists know how long the effect will last.