FDA Approval Precedent (for gene therapy)
**What it means:** The fact that seven AAV-based gene therapies have received FDA approval provides a regulatory pathway that the MEF2C gene therapy team can follow. FDA has extensive experience revie...
Explainers
Plain-English explanations of MEF2C research terms, concepts, and scientific mechanisms.
Drug Pipeline (in pharma context)
**What it means:** A "drug pipeline" is the entire journey a drug takes from the lab bench to the pharmacy shelf. Think of it as a factory assembly line: 1. **Discovery** → finding compounds that affe...
First-in-Human (FIH) Trial
**What it means:** This is the **very first time a drug is given to people** — never humans before, only animals (and cell cultures). It's essentially Phase 1a. The goals are: 1. **Safety** — does the...
Phase 1a/b Clinical Trial
**What the phases mean:** - **Phase 1a:** First-in-human testing. The primary goal is **safety** — "Is this drug safe in humans at all?" A small group of patients receives escalating doses to find the...
AAV (Adeno-Associated Virus)
**What it means:** AAV is a tiny, harmless virus used as a **delivery vehicle** (vector) to carry therapeutic genes into cells. Think of it like a Trojan horse — the virus looks normal from the outsid...