From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases

Explore gene-based treatments for rare diseases

Explore gene and cell therapies transforming care for 300 million people with rare diseases worldwide.

On this three-week course from the Foundation for Rare Diseases you’ll study diverse gene therapy technologies, and clinical successes that have transformed lives.

Discover the science behind breakthrough therapies

95% of rare diseases lack treatments, which remain mostly symptomatic. In this course, you’ll explore the characteristics of rare diseases and understand why gene therapies offer an important opportunity to treat the disease directly at its root.

Master the technologies revolutionizing treatment

Dive into key technologies while being guided by international experts. You’ll examine: non-viral delivery systems (lipid nanoparticles, transposons), viral vectors (adenoviral, AAV, lentiviral, retroviral), gene editing (CRISPR-Cas9, base editing, prime editing), RNA therapeutics (mRNA, antisense oligonucleotides, siRNAs), and gene-modified hematopoietic stem cell therapy for blood disorders.

Understand safety and regulatory frameworks

Develop a strong understanding of critical safety considerations including genotoxicity and immunotoxicity. You’ll also learn how orphan drug designation programs incentivize rare disease drug development through market exclusivity and regulatory support.

Witness therapies changing lives

Gain a fundamental understanding of how revolutionary therapies move from concept to life-changing reality. You’ll expand your knowledge through clinical case studies in hemophilia, sickle cell disease, Fabry disease, and metabolic disorders, you’ll see how these revolutionary therapies move from concept to life-changing reality.

The course is ideal for university students and early-career researchers in the fields of biology, life sciences, and medicine. It is also open to anyone seeking to learn more about gene and cell therapies for rare diseases.

This course has been developed through funding from the ASGCT & Pfizer Medical Education Grant 2023.

The course is ideal for university students and early-career researchers in the fields of biology, life sciences, and medicine. It is also open to anyone seeking to learn more about gene and cell therapies for rare diseases.

This course has been developed through funding from the ASGCT & Pfizer Medical Education Grant 2023.

• Describe the landscape of rare diseases, their genetic basis, and current therapeutic gaps
• Explain viral vector technologies and their mechanisms of gene delivery
• Compare gene editing approaches including CRISPR, base editing, and prime editing
• Identify mRNA therapeutics and antisense oligonucleotides as emerging treatment strategies
• Explore clinical case studies demonstrating successful gene therapy applications
• Evaluate orphan drug designation programs and regulatory frameworks
• Discuss implementation barriers and future directions in gene therapy for rare diseases