top of page

Gene therapies represent an exciting new way to treat disease. Naturally, stakeholders have a lot of questions about these transformative therapies – from how they work and how they’re made to what diseases they’ll treat and whether they’ll be covered by insurance. 

​

Here we have gathered some of the most informative resources on gene therapies from our advisory council members and beyond. We hope this one-stop-shop is a helpful guide to learning about gene therapies.

What are Gene Therapies? 

Asset 2.png

Background on the science behind gene therapies and how they’re made.

The American Society of Gene and Cell Therapy

Sarepta Therapeutics

The American Society of Gene and Cell Therapy

What Is Gene Therapy? How Does It Work?

arrow-01.png

U.S. Food & Drug Administration

The American Society of Gene and Cell Therapy

genehome

arrow-01.png

bluebird bio

The Value of Gene Therapies

Asset 4.png

Value for Whom? Incorporating Patient Perspectives into Value
Assessment for Novel Cell and Gene Therapies

arrow-01.png

EveryLife Foundation Position Paper developed by Health Advances in collaboration with the Partnership to Improve Patient Care

Solutions to Access Barriers

Asset 3.png

Improving Access to
Newborn Screening

arrow-01.png

Institute for Gene Therapies

Ensuring Patient Access to Gene Therapies

arrow-01.png

Institute for Gene Therapies

IGT Comments on Policy Proposals

comment-01.png
policy-briefs
bottom of page