Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
B7-H3-CAR-T exosomes + si-HLX
- Development stage
- Preclinical
- Lead developer
- Johns Hopkins All Children’s Hospital
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
B7-H3-CAR-T exosomes + si-HLX is a preclinical-stage exosome-based therapy developed by Johns Hopkins All Children's Hospital and Johns Hopkins University for the treatment of subgroup 3 medulloblastoma (G3-MB). The therapeutic candidate consists of exosomes derived from B7-H3-targeting chimeric antigen receptor (CAR) T-cells, which are loaded with lipid nanoparticle-formulated small interfering RNA targeting the H2.0-like homeobox (HLX) transcription factor (LNP-si-HLX). The B7-H3-CAR-T-derived exosomes are designed to cross the blood-brain barrier (BBB) and selectively target tumor cells expressing B7-H3. Upon internalization, the exosomes deliver their cytotoxic cargo (including perforin and granzyme B) and the therapeutic si-HLX, which inhibits HLX expression, suppresses tumor growth, and modulates the tumor immune microenvironment without inducing systemic immunotoxicity such as cytokine release syndrome (CRS).
- Other names
- B7-H3 (mAb) CAR-T exosomes carrying LNP-si-HLXB7-H3-CAR-T-Exos loaded with si-HLXB-7-H3-CAR-T-Exos loaded with si-HLXB 7-H3-CAR-T-Exos loaded with si-HLXB7-H3-CAR-T-Exosomes + si-HLXanti-B7-H3-CAR-Exosomes loaded with si-HLXanti-B-7-H3-CAR-Exosomes loaded with si-HLXanti-B 7-H3-CAR-Exosomes loaded with si-HLXanti-B7-H3/LNP-si-HLX-CAR-T exosomes
02
Targets
B7-H3
Beyond the preview
Go deeper on B7-H3-CAR-T exosomes + si-HLX.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on B7-H3-CAR-T exosomes + si-HLX.