Project portfolio

Idogen´s technology enables development of tolerogenic cell therapy in a number of areas. Idogen has chosen to develop, as a first application, a treatment method for patients with severe haemophilia Type A who currently lack any treatment option, after developing antibodies to their vital factor VIII therapy.

When the body’s immune system attacks factor VIII, a critical medication

IDO 8 is Idogen’s most advanced project, aimed at developing a tolerogenic cell therapy for patients with severe haemophilia A who have developed inhibitory antibodies against their standard treatment. Idogen´s treatment has the potential to restore the original efficacy of factor VIII treatment. The company has chosen haemophilia A as the first indication due to the major unmet medical need of these patients and because the disease has a well-defined antigen, presenting an opportunity to develop an effective treatment for this patient group. Haemophilia A is a rare disease and Idogen has been granted Orphan Drug Designation in Europe for the treatment – a key value-adding step for the company since orphan drug designation provides several advantages, such as less extensive requirements for clinical trials, scientific guidance from the regulator during development and ten-year market exclusivity after launch.

The company expects the first clinical trial to commence in H2 2021.

About 50,000 boys and men in the US and Europe are living with haemophilia A, a congenital disease that primarily affects boys and men. The treatment for patients with the severe form of haemophilia A is factor VIII replacement therapy. About 30 percent of the patients treated with factor VIII develop inhibitory antibodies, making the treatment ineffective. This complication can often be managed with regimens to induce immune tolerance, involving frequent injections of higher doses of factor VIII. The treatment is costly and may last for one or two years. The antibodies remain in about 30 percent of these “inhibitor patients”, leaving them unable to prevent bleeding. Inhibitor patients are being treated with agents with only a short duration of effect, at a cost of as much as SEK 3-8 million per patient a year, to stop acute bleeds.

A new and effective drug for this patient group will soon be launched in the US. One-year treatment costs are believed to be almost SEK 4 million, and analysts estimate that the preparation can reach annual sales of more than SEK 17 billion[1]. However, this drug is associated with the risk of severe side effects, and treatment is lifelong. There are still, therefore, major unmet needs for safer, more cost-effective therapies.

When the body’s immune system attacks a transplanted organ

The same method that is currently under development for the treatment of haemophilia A can also be used for other indications with only minor adjustments of the production process. The company has therefore made a strategic decision to commence parallel development of a product candidate for kidney transplantation, IDO T. The basic principle is to “teach” the patient’s immune system to recognise and accept the transplanted organ rather than attack it. This could eventually reduce the need for current methods of often lifelong treatment with drugs that inhibit immune system functionality. There is a major unmet need for long-acting, cost-effective and safe treatment to avoid the risk of transplant rejection.

Preclinical development for IDO T is ongoing with the aim of commencing a Phase I/IIa clinical trial during 2022.

Kidney transplants are the most common type of organ transplant and almost 80,000 kidney transplants are performed globally per year, including about 20,000 in Europe[2]. The largest and most serious complication is when the transplant recipient’s immune system attacks, destroys and rejects the donated organ. To prevent this, the transplanted patients – with few exceptions – are given lifelong treatment with a combination of drugs to suppress their immune system. While the proportion of patients who retain a functional transplanted kidney during the first year of transplantation has increased in recent decades, there has not been any improvement in the long-term survival of transplant recipients[3].

Immunosuppressive therapy also carries a risk of serious infections and cancer. Transplantation is therefore an indication with a major unmet medical need. Idogen’s tolerogenic cell therapy has the opportunity to reduce the need for immunosuppressive drugs and improve transplant survival.

When the body’s immune system attacks the body’s own proteins

Patients with autoimmune diseases are often treated for long periods of time with drugs that suppress the immune system. However, the effect on the underlying disease is rarely optimal and the treatment can lead to undesirable side effects. The unmet medical need for improved therapies is therefore high. The goal of Idogen’s tolerogenic cell therapy is to dramatically reduce the need for immunosuppressive drugs by shortening the treatment, thereby improving patient outcomes.

Idogen’s research unit is currently evaluating the potential of the company’s technology in a group of autoimmune diseases with a major unmet medical need, and where a treatment could be granted orphan drug designation.

[2] Global Observatory on Donation & Transplantation in collaboration with WHO.
[3] Afzali B, Taylor AL, Goldsmith DJA. What we CAN do about chronic allograft nephropathy: Role of immunosuppressive modulations. Kidney International, 2005, 68, 2429-2443.
Wang JH, Skeans MA, Israni AK. Current status of kidney transplant outcomes: dying to survive. Adv Chronic Kidney Dis, 2016, 23, 5, 281-286.

For further information or interest regarding collaborations,
please contact CEO Anders Karlsson, anders.karlsson[at]