Gene Therapy, RNA and Pens at European Hemophilia Congress in Paris

02/02/2017 - 4 minutes

The hottest biotechs in the field of hemophilia are stealing the show at this year’s edition of the EAHAD hemophilia congress in Paris.

A disorder for which no cure is available, hemophilia is caused by absent or defective genes coding for blood clotting factors, turning simple injuries into health risks and causing spontaneous bleeding. Researchers and companies worldwide working to improve hemophilia therapy are meeting this week in Paris for the 10th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

We recently reviewed the latest advances in hemophilia, a field teeming with innovative solutions and technology. Among the most interesting presented at the congress are new results on gene therapy and RNAi, a pen to treat hemophilia and many companies fighting to reduce the dosing frequency of prophylactic therapy. On top of that, Shire has reported that current estimates of people suffering from the disease could be completely wrong…

Novo Nordisk hemophilia

Novo Nordisk is making plans to use its famous insulin pens to deliver hemophilia drugs. According to a study evaluating user experience with the pens presented at the congress, participants liked the device as it is easy to use, well designed, more portable and involving fewer steps than their current kits for hemophilia.

The ultimate goal of the Danish company is to use its FlexTouch pen to deliver concizumab, an antibody against tissue factor pathway inhibitor (TFPI) currently in Phase I for both hemophilia A and B.

Shire hemophilia

Shire has presented a study revealing that the incidence of hemophilia could be more than three times higher than current estimates. It also showed that only 25% of hemophiliacs receive adequate treatment. These findings might push efforts to put an end to this situation and stimulate market growth.

Shire presented positive results from a Phase II/III trial for Adynovate (BAX 855) in children with hemophilia A. Interestingly, the company also showed early stage in vitro results for combination therapies with a biosimilar of Roche’s emicizumab (ACE910). It looks like the antibody, not yet in the market, already has strong competitors getting ready for when its patent expires.

sobi bioverativ hemophilia

Sobi, in Sweden, has co-developed recombinant clotting factors with an extended half-life in partnership with Biogen’s spin-off Bioverativ. To do so, they fuse the clotting factor to the Fc portion immunoglobulin G1 proteins.

The team has presented positive long-term safety and efficacy results for Eloctate in hemophilia A and Alprolix in hemophilia B. Both are already in the market and reduce dosing frequency to weekly injections.

Opko biologics hemophilia

OPKO Biologics, in Israel, follows a strategy similar to Sobi’s. Its CTP technology extends the half-life of proteins by fusing them with the C-terminal peptide of human chorionic gonadotropin (hCG).

The company has presented data for MOD-5014 (FVIIa-CTP) supporting the advance into Phase II/III trials. The drug is intended for delivery twice a week, which is double of that from Sobi’s products.

spark therapeutics hemophilia

Spark Therapeutics is one of the leaders in the development of gene therapy for hemophilia and uniQure‘s main competitor. The American company will report results from its Phase I/II trial for SPK-9001 in hemophilia B showing sustained activity of the therapy after 12 weeks, with only one reported bleeding.

Despite good results, Spark is facing strong competition from the Dutch uniQure. Its gene therapy AMT-060 has already shown sustained effects for at least 52 weeks in a patient subpopulation. Both companies now have breakthrough designation from the FDA and the race to reach the market is tight.

alnylam hemophilia

Sanofi‘s partner, Alnylam, is conducting clinical trials across the UK, Switzerland and Bulgaria to test its unique RNAi technology for hemophilia. Its candidate fitusiran, which blocks antithrombin to improve clotting, is proving safe and effective in Phase I/II trials.

This unique treatment has the potential to reduce dosing to a monthly basis and is suitable for patients with both hemophilia A and B, also including those that have developed resistance to standard treatments.

Among the companies presenting are many others including Genentech, Bayer and Catalyst Biosciences. The sheer number of innovative approaches under development is great news. Such a wide arrange of solutions could provide a better quality of life for hemophilia patients, each treatment suited for the particular needs of different patients. Especially now that, thanks to Shire, scientists know the number of patients suffering from the condition could actually be much higher.

Images from Sashkin, nobeastsofierce, Roberta Canu, Mond Duang, Art tools, LeonP, Pakpoom Nunjui /


Do you want to remove this advert? Become a member!
Do you want to remove this advert? Become a member!

Support Us

Become a Member