Jean Tang, MD ’99, PhD ’03, Resident ’07 | Personalized Gene Therapy to Treat Rare Disease

Why It Matters

Tang’s work proves that personalized gene‑therapy can move from lab to market for ultra‑rare diseases, setting a template for future, more affordable CRISPR‑based treatments that could reshape rare‑disease care and broader gene‑editing applications.

Key Takeaways

• Gene‑edited skin grafts restore collagen VII in epidermolysis bullosa.
• Phase‑III trial showed >50% wound closure and pain reduction.
• NIH‑funded academic GMP facility enabled first patient treatments in 2013.
• Commercial partner Abiona scaled production, securing FDA approval in 2025.
• Future CRISPR spray aims to lower costs and treat multiple organs.

Summary

Dr. Jean Tang, a Stanford dermatologist, detailed her two‑decade journey developing a personalized gene‑therapy for epidermolysis bullosa (EB), a rare disorder affecting one in 100,000 where patients lack functional collagen VII. Using a retroviral vector to deliver the 9 kb COL7A1 gene into autologous skin cells, her team grew corrected grafts in a small GMP facility and surgically applied them to patients, beginning with the first treated in 2013. The phase‑I/II trials demonstrated safety—no viral spread or immune reaction—and early efficacy, with healed wounds in multiple patients. Phase‑III data from 11 patients (86 wounds) showed more than 50 % wound closure, three‑point pain reduction, and itch relief compared with untreated controls, culminating in FDA approval in 2025 after commercial partner Abiona assumed development and scaled manufacturing to a 10,000‑sq‑ft plant. Tang highlighted the logistical hurdles of transporting living grafts, the volatility of biotech financing, and the human impact—one teenager could finally attend prom. She also outlined the next frontier: CRISPR‑based topical editing to cut costs and extend therapy to ocular, gastrointestinal, and other organ manifestations of EB. The story underscores how academic innovation, when paired with strategic industry partnership, can translate a rare‑disease concept into an approved, life‑changing therapy, while raising critical questions about manufacturing scalability and long‑term affordability of cell‑based gene treatments.

Original Description

Jean Tang, MD ’99, PhD ’03, Resident ’07

Professor of Dermatology

Personalized Gene Therapy to Treat Rare Disease

For patients with epidermolysis bullosa, a rare genetic condition that causes skin as fragile as butterfly wings, even the slightest touch can lead to painful blistering and wounds that never heal. Dr. Tang will share her team’s 20-year groundbreaking journey from laboratory to FDA approval of personalized skin grafts to treat patients’ large, open wounds. Genetically engineered from a patient’s own cells, these grafts bring immense relief to patients, primarily young children. Learn how early intervention with gene therapies may transform the disease trajectory for future patients.

Part of Stanford Medicine Alumni Day 2026.