TRANSFORMING LIVES THROUGH CURATIVE THERAPIES
WATCH OUR STORYbluebird bio has rebranded as Genetix Biotherapeutics, returning to our foundational roots and renewing our commitment to delivering transformative genetic therapies for patients living with sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy. These first-in-class, one-time administered therapies treat the underlying cause of disease and can offer significant and lasting benefits to patients.
On June 2nd, 2025, we marked a pivotal moment: announcing a newly appointed leadership team and our transition to a privately held company. We are now stronger, both financially and operationally, and better positioned to harness our decades of scientific innovation and extensive clinical data from hundreds of treated patients to unlock the full potential of our life-changing genetic therapies.
READ THE PRESS RELEASEOUR MISSION
While our name has changed, our mission remains the same: to transform lives through curative therapies for patients with rare, genetic diseases—now with sharpened focus on commercial execution, scaling patient access and improving the treatment experience for both patients and providers.
Delivering
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Commercial
execution
Scaling
access
Improved treatment
experience
UNDERSTANDING GENETIC THERAPIES
For patients and their families, understanding the genetic disease that is impacting their lives is the first step toward finding answers—and hope.
Many genetic diseases are caused by one or more differences in a gene in the DNA that a person is born with. For many of these diseases, most treatments only manage and reduce symptoms. Genetic therapies are designed to treat the underlying cause of the disease and serve as a potential cure.
Genetix Biotherapeutics' therapies are treatments based on the person's own blood stem cells. The cells are collected, modified outside the body to add working copies of the affected gene, and then given back to the person, restoring the function of the affected gene.
After more than 30 years of research and clinical development, these therapies are approved under the FDA's rigorous safety standards to treat serious rare diseases.
Our Focus
Sickle Cell Disease
A lifelong, progressive blood disorder caused by a genetic defect in hemoglobin that affects red blood cell shape and function. Without curative treatment, patients suffer from painful crises, repeated hospitalizations, strokes and other organ damage, poor quality of life, and may have a lower life expectancy. Our goal is to enable people with sickle cell disease to live without the burden of unpredictable pain and health crises.
Beta-thalassemia
A severe, inherited blood condition that reduces the body's ability to produce hemoglobin, a protein found in red blood cells that carries oxygen throughout the body. Many patients require ongoing transfusions and treatments to reduce iron in the body, which can impact daily life. Despite these treatments, patients develop organ damage from anemia and iron-overload and may have a lower life expectancy. Our goal is to provide targeted, lasting treatment that reduces or eliminates the need for ongoing transfusions.
Cerebral Adrenoleukodystrophy (CALD)
CALD is a rare genetic disease affecting boys which is caused by a defective ABCD1 gene that leads to the destruction of the protective covering around nerve cells and causes progressive and permanent damage to the brain. Our goal is to provide transformational treatment to slow the progression of damage to the brain.
Our Therapies
Each of our approved therapies is built on decades of research and clinical experience—and focused on giving patients with rare diseases a new path forward.
Clinical trials
Genetix is committed to long-term safety and follow-up. Our clinical research includes extended studies that monitor participants for up to 15 years: both for those who joined Phase 1, 2, or 3 trials, and for patients receiving therapies after FDA approval. For more information on our clinical trials email us at clinicaltrials
OUR PATIENT SUPPORT
Our patient services program offers personalized assistance for patients and families navigating the treatment journey—from insurance questions to treatment center coordination to one-on-one support every step of the way.
* The patient support website is still bluebirdbio branded for the time being.
LEADERSHIP
United by purpose
Our leadership brings together decades of experience in biotech, commercialization, and patient advocacy. Together, we are laser-focused on getting patients the life-changing genetic therapies they need when they need them.
David Meek is a seasoned biopharma executive with over 30 years of global leadership experience. He has served as CEO of Mirati Therapeutics, FerGene, and Ipsen, and brings deep expertise in commercialization and corporate strategy.
David Meek
Chief Executive Officer
Brian Riley has more than 25 years of experience in manufacturing, quality, and operations across cell therapy and biologics. He has held leadership roles at Beam Therapeutics, Catalent Pharma Solutions, and Biogen Idec.
Brian Riley
President & Chief Technical Officer
Dr. Joanne Lager has over 20 years of experience leading drug development across all phases of research. She has held senior leadership roles at NiKang Therapeutics, iTeos Therapeutics, Sanofi, and GSK, and began her career in pediatric hematology/oncology at Duke University Medical Center.
Joanne Lager
MD, Chief Medical Officer
Tom Klima has led commercial strategy across oncology and rare disease, including launches at Gamida Cell, Atara Biotherapeutics, and Algeta U.S. He brings deep experience building high-performing teams and delivering impactful therapies to market.
Tom Klima
Chief Commercial & Operating Officer
Wendy DiCicco is a life sciences finance and operations executive with over 30 years of experience. She has served as CFO and COO across public and private companies and currently sits on the boards of EyePoint Pharmaceuticals and Imvax, Inc.
Wendy DiCicco
Chief Financial Officer
Ellen Forest is a senior HR leader with extensive experience across biotech, healthcare, and private equity. She has held key roles at Yumanity Therapeutics, Cogen Immune Medicine, Merrimack Pharmaceuticals, and Partners HealthCare.
Ellen Forest
Chief People Officer
Adrian Chaisson brings broad expertise in drug development and cross-functional leadership, from discovery through commercialization. He previously held senior roles at Be Biopharma, FerGene, and consulting firms, driving strategy and execution across first-in-class genetic therapies.
Adrian Chaisson
Chief of Staff
Join the mission!
Interested in working with a team of people passionate about transforming the lives of patients with innovative genetic therapies?
SEE CAREER OPPORTUNITIESCALD = cerebral adrenoleukodystrophy