WRD is interested in establishing alliances to develop therapeutics, expand disease biology understanding, and identify biomarkers that impact:
- Hematology
- Hemophilia
- Extended half-life of coagulation factors
- Oral anti-hemophilic agents
- Immune tolerance
- Other rare hematologic (non-malignant) indications
- Sickle cell anemia & beta-Thalassemia with focus on disease modifying and/or therapies that significantly change disease pathology
- Hemostasis (systemic and topical)
- Orphan Neuromuscular Diseases
- Duchenne/Becker muscular dystrophy and myotonic dystrophy: complement internal muscle-building approaches with disease-modifying therapies
- Friedreich's ataxia: upregulate frataxin expression
- Amyotrophic lateral sclerosis (ALS): protein misfolding approaches
- Disease modifying approaches for other neuromuscular diseases, such as spinal muscular atrophy (SMA)
- Other Proteinopathies
- Lysosomal storage diseases
- Novel enzyme replacement therapies (e.g., extended half-life or blood-brain barrier penetration)
- Small molecule approaches
- Alpha-1 antitrypsin deficiency
- General Mechanisms of Interest
- Chaperones and other modifiers of protein trafficking, misfolding, or degradation that could span across multiple diseases
- Targeting technologies/platforms (e.g., muscle and CNS targeting)
- Modifiers of gene transcription via epigenetic approaches
- Novel or differentiated protein replacement therapies
- Gene therapy approaches
- Antibody-drug conjugates
Not actively seeking partnering opportunities in:
- Undifferentiated approaches towards enzyme replacement in well-served markets
- Intrathecal enzyme replacement therapy
