Celiac Disease

  • Celiac disease is an autoimmune disease that is triggered by consuming gluten and results in damage to the small intestine
  • Symptoms include diarrhea, fatigue, headaches, anemia, nausea, dermatitis herpetiformis (itchy skin rash)
  • Significant patient population does not respond to gluten free diets
  • Health consequences for not treating include malnourishment, cancer and other autoimmune conditions
  • Estimated prevalence is 1:133 (2.5 million people) with celiac disease in the US (Fasano, Arch Intern Med. 2003 PMID: 12578508)
  • 0.3% to 0.5% of celiac disease patients are non-responsive (Malamut Gastroenterology. 2024 38556189)
  • There are no approved treatment options for celiac disease

Vitiligo

  • Vitiligo is an autoimmune disease of the skin mediated primarily by NK and CD8+ T cells that attack melanocytes leading to patchy depigmentation of the skin
  • It is estimated that vitiligo affects 2 million people in the U.S (NIH)
  • The global vitiligo treatment market size was estimated at $1.2 billion in 2018 and is projected to reach $1.9 billion by 2026 (Fortune Business Insights)

Type 1 Diabetes

  • Type 1 diabetes (T1D) is classified by three stages of progression
  • Stage 1: At least one diabetes-related autoantibody but has normal blood sugar and no symptoms
  • Stage 2: At least two diabetes-related autoantibodies and abnormal blood sugar levels but otherwise symptom free
  • Stage 3: Significant beta cell loss has occurred; abnormal blood sugar levels; hemoglobin A1C >6.4%; excessive thirst and urination; blurry vision; fatigue; requires insulin for disease management
  • 64,000 people diagnosed with Type 1 diabetes annually (https://beyondtype1.org/type-1-diabetes-statistics/)
  • 2 approved treatment options to delay the onset of Type 1 diabetes

Acute Graft-vs-Host-Disease (aGvHD)

  • US Prevalence is estimated at approximately 5,000 cases and occurs in up to 50% of allogeneic stem cell transplant recipients(1)
  • Onset of aGvHD typically occurs within 3 months of transplant
  • Symptoms occur in the skin (rash), GI tract (vomiting, diarrhea) and liver (jaundice)

(1) https://doi.org/10.1186/1750-1172-2-35

Chronic Graft-vs-Host-Disease (cGvHD)

  • US Prevalence is estimated at approximately 14,000 cases and occurs in up to 40% of recipients(2)
  • In addition to symptoms in the skin, GI tract and liver, cGvHD may also manifest itself in the lungs, mucosal surfaces (eyes, mouth, GU tract), muscle and joints (connective tissue)

(2) https://pubmed.ncbi.nlm.nih.gov/34158154/

Alopecia Areata (AA)

  • AA is an autoimmune disease in which immune cells attack and damage hair follicles and is mediated primarily by CD8+ T cells and NK cells
  • The global alopecia treatment market was valued at $2.7 billion in 2018, and is projected to reach $3.9 billion by 2026, registering a CAGR of 4.6% from 2019 to 2026 (Allied Mkt Research)