By Fineline Cube 
Bioheng Therapeutics (China) announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational allogeneic CAR‑T cell product, CTD402, for the treatment of relapsed/refractory T‑cell acute lymphoblastic leukemia (R/R T‑ALL) and lymphoblastic lymphoma (R/R LBL).
What the RPDD Means
- Accelerated Development Pathway – RPDD can facilitate faster regulatory review and potential priority review status for future approvals.
- Market Differentiation – The designation signals the FDA’s recognition of CTD402’s potential to address a severe unmet need in pediatric oncology.
About CTD402
| Feature | Detail |
|---|---|
| Platform | Off‑the‑Shelf, CD7‑targeted, universal CAR‑T |
| Source | Healthy donor T‑cells |
| Engineering | Gene edits to prevent fratricide, GvHD, and HvG; enhanced anti‑tumor activity |
| Manufacturing | Single‑batch production for multiple patients |
| Clinical Milestone | FDA approved IND in March 2025 for both pediatric and adult R/R T‑ALL/LBL |
Bioheng Therapeutics – Company Snapshot
- Founded – 2017, clinical‑stage biopharma focused on universal CAR‑T platforms.
- Portfolio – Emphasis on hematologic malignancies and autoimmune diseases.
- Strategic Vision – Develop world‑leading off‑the‑shelf cell therapies to fill critical unmet needs.
Next Steps
- Clinical Development – Phase I/II trials in pediatric and adult cohorts will commence in Q1 2026.
- Regulatory Strategy – Leverage RPDD to negotiate expedited review pathways and potential orphan drug status.
- Commercial Planning – Prepare supply chain and distribution frameworks for a scalable off‑the‑shelf product.-Fineline Info & Tech