Tackling Challenging Indications & Unmet Medical Need.

We are not afraid to go boldly into diseases and conditions for which few or no effective treatments have been developed. We love the challenge and accept the responsibility.

Our Programs

Our focus is on diseases with no effective treatment or cure.

Graft versus Host Disease (GvHD)

Graft versus Host Disease (GvHD) occurs due to pathogenic immune responses after hematopoetic stem cell transplants, frequently the only treatment for patients with aggressive hematological malignancies, such as acute myeloid leukemia.

~15%

Rate of severe acute Grade III-IV GvHD

For patients receiving peripheral blood stem cell transplants from mismatched donors.

M. M. Al Malki et al. BloodAdvances 5, 2650-2659 (2021).

>40%

Mortality rate for Grade III-IV aGvHD

Despite prophylactic regimens aimed at reducing incidence and severity, over one third of patients with aGvHD die as a result.

S. G. Holtan et al. Blood 140,LBA-4-LBA-4 (2022).

Inflammatory Bowel Disease

Severe refractory Inflammatory Bowel Disease (IBD) is a persistent, acute, symptomatic gut disease that continues to significantly impact the quality of life of patients despite continued use of anti-inflammatory or disease modifying drugs.

30%

Of IBD patients fail all lines of therapy

A significant portion of steroid refractory IDB patients never achieve disease control, significantly lowering their quality of life.

D. Wintjens et al. J of Crohn's and Colitis 15, 391-400(2020).

25%

Percent of Crohn's patients who will require
major surgery

Many Crohn's patients will ultimately require colectomies to alleviate symptoms, though this does not address the root cause of disease.

Tsai et al. Clin Gastroenterology andHepatology 19.10 (2021): 2031-2045.

Autoimmune Liver Diseases

The three most common autoimmune liver diseases are autoimmune hepatitis, primary biliary cholangitis and primary sclerosing cholangitis. These conditions may occur individually or as part of “overlap” syndromes.

2x

Elevated Mortality Risk

Autoimmune liver disease is associated with a 2 fold increased risk of death compared to controls.

R. Sharma et al. ClinGastroenterology andHepatology 19.12 (2021):2636-2647.

~15%

Percent of patients who require liver transplant

Patients frequently are or become refractory to multiple lines of therapy, leading to cirrhosis and liver failure.

D. Gleeson et al. Gut 60.12(2011): 1611-1629.

B and T Cell Mediated Autoimmune Diseases

Systemic autoimmune diseases are characterized by aberrant adaptive immune responses to autoantigens. Autoreactive B and T cells play a central role in the pathogenesis of autoimmunity and have been implicated in a large number of autoimmune disorders.

>80

Number of defined B and T cell driven autoimmune disorders

This figure continues to rise as new diseases are uncovered and diagnosed.

Source: Johns Hopkins School of Medicine

>100Bn

Annual Cost Burden of AID in the US

This figure only includes 7 of the over 100 known B and T cell mediated autoimmune diseases, which continue to increase in prevalence.

Source: American Autoimmune Related Diseases Association (AARDA)

Relentlessly dedicated to improving the lives of patients.

We are pursuing breakthrough innovation in the spaces of autoimmunity and inflammation with a world-class team aiming to change the way devastating diseases are treated.

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