Making Hope A Reality

Orphan (or rare) diseases

While the term “orphan disease” implies it is rare in occurrence, the impact on the patients and families are long-term and in some cases, fatal. Much of bluebird’s core work focuses on three rare disease states: Adrenoleukodystrophy, β-thalassemia and sickle cell disease.

 

Additional Information

National Organization for Rare Disorders Rare Disease Fact Sheet

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β-thalassemia

bluebird bio on β-thalassemia

Watch and Learn: What is β-thalassemia?

 

Additional Information

St. Jude Children’s Research Hospital Beta Thalassemia Trait Fact Sheet

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Sickle Cell Disease

bluebird bio on SCD

Watch and Learn: What is SCD?

 

Additional Information

Sickle Cell Disease Association of America’s (SCDAA) 5 Facts You Should Know About Sickle Cell Disease Fact Sheet

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Product Candidate

At bluebird, we’re making real and exciting progress, especially with two separate product candidates – Lenti-D, a potential treatment for CALD, and LentiGlobin, a potential treatment therapy for TDT and SCD.

Our Clinical Trials

Our Product Pipeline

 

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The Next Five Years

We believe gene therapy is the beginning of a new era in healthcare and know the next five years will be revolutionary, with innovation at the heart of all we do.

A Collaborative Rare Disease Community

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Reimbursement Policies

As gene therapy advances, we must also work to ensure the right policies are in place to support this treatment.

 

Additional Information

Tapestry Networks ViewPoints Building a Sustainable Health System for Curative Therapies
The Alliance for Regenerative Medicine Curative Regenerative Medicines Preparing Health Care Systems For The Coming Wave

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