Expanded Access to Investigational Drugs Policy
Purpose and Philosophy Behind the Policy
Throne Biotechnologies (Throne) is a clinical-stage therapeutic company developing a disruptive stem cell technology aimed at fundamentally reversing Type 1 Diabetes (T1D), Alopecia Areata (AA), and other autoimmune diseases. This is achieved through immune education via Stem Cell Educator Therapy (GleukocellTM), which addresses the root causes of these conditions.
Over the past 15 years, international multicenter clinical trials conducted in the United States, China, and Spain have demonstrated the clinical safety and efficacy of Stem Cell Educator Therapy. Throne is committed to providing early access to this innovative therapy for patients with serious and life-threatening medical conditions prior to full FDA approval for prescription use.
Approach to Requests for Access to Stem Cell Educator Therapy
The primary and most preferred pathway for patients to access Stem Cell Educator Therapy is through participation in Throne’s ongoing clinical trials for T1D and AA.
For patients who do not meet the eligibility criteria for clinical trial participation, their treating physician—if able to comply with the conditions outlined in this policy—may request information on how to apply for access to Throne’s investigational product by contacting the company directly.
Access Policy
1. Patient Eligibility Criteria
To qualify for access to the investigational product, patients must meet the following criteria:
- Have a serious or life-threatening condition for which there is no satisfactory alternative treatment.
- Be evaluated by the treating physician as having potential benefits from the therapy that outweigh the potential risks.
- Have adequate venous access to undergo blood cell separation (apheresis).
- Be able to provide informed consent.
- Be willing and able to comply with all study requirements, including attending all study visits.
2. Investigational Product Criteria
- The product must be under clinical investigation in one or more clinical trials and must have a strong safety profile.
- The company must have an adequate supply of the investigational product.
- Granting expanded access must not interfere with the timely completion of clinical trials or delay marketing approval and broader patient availability.
3. Treating Physician Criteria and Responsibilities
The treating physician must agree in writing to the following conditions:
- Be properly licensed and authorized to practice medicine in their respective country or region.
- Meet all country-specific legal and regulatory requirements related to the provision of investigational products under Expanded Access programs.
- Comply with all of Throne’s safety reporting requirements and intellectual property protection protocols.
How to Request Expanded Access
Treating physicians may submit questions or requests related to Expanded Access by contacting:
📧 Email: connect@thronebio.com
Throne Biotechnologies will make every effort to acknowledge submitted requests within five (5) business days.