FROM INNOVATION
TO IMPACT

Built to deliver
genetic medicine
to patients
at scale

With decades of scientific and clinical experience, Genetix is focused on delivering commercially viable, one-time treatments, so more patients can access life-changing care.

Our Mission

Transforming lives through curative therapies

Our Focus

Genetix is focused on the commercial execution of our three FDA-approved treatments and scaling our capabilities to ensure they reach the tens of thousands of patients who still need life-changing care.

Delivering for patients

Patients are at the center of everything we do, with programs and services designed to help them navigate treatment with clarity and confidence.

Scaling access

The promise of genetic medicine is now a reality, and we are focused on expanding access so more patients can receive treatment.

Partnering with healthcare providers

Strong partnerships with Qualified Treatment Centers are essential to our mission, and close collaboration is required to ensure patients receive the care they deserve.

Enhancing the 

treatment journey

Guided by the urgency that patients face, we are deeply committed to delivering our treatments with speed, precision, and an unwavering commitment to safety.

Who We Are

Genetix is the market leader in genetic medicine for rare diseases, with three FDA-approved treatments and one of the most mature clinical datasets in hematology.

Built on more than 30 years of scientific and clinical experience, we are focused on expanding access and advancing a sustainable, commercially viable model to deliver these therapies to the patients who need them most.

Our Story

Founded in 1992, Genetix Pharmaceuticals emerged from academic research with a focus on developing treatments that provide patients with a cure rather than managing symptoms. The company established its core gene addition technology, enabling functional gene correction using a patient’s own cells.

Early scientific breakthroughs led to initial clinical proof of concept, laying the foundation for future clinical development and commercialization.

In 2010, following the release of initial clinical data from Genetix Pharmaceuticals, the company secured investment, transitioned leadership, and was renamed bluebird bio. During this period, three programs advanced through late-stage development, leading to FDA approval of three first-in-class gene addition cell therapies.

This work moved genetic medicine from early clinical validation to full approval, establishing a new curative treatment paradigm.

In 2025, the company was acquired to accelerate its transition to a commercially focused organization. Rebranded as Genetix Biotherapeutics under strengthened leadership, we entered a new phase focused on execution, delivery, and expanding access—all while remaining grounded in the company’s foundational mission.

Today, we are focused on scaling access to genetic medicines and expanding patient access to transformative care.

Our Commitments

Delivering on our mission requires close collaboration with patients, healthcare providers, and our Qualified Treatment Centers (QTCs). As such, we are committed to showing up as strong, reliable partners across the healthcare ecosystem to ensure our treatments are delivered safely, effectively, and at scale.

How we serve our patients

We provide transformative, one-time treatments for progressive rare diseases with urgency, unlocking new possibilities in patients’ lives. We approach every patient interaction with respect and provide clear information, individualized support, and opportunities for connection through the journey to a cure.

Our partnership with healthcare providers

We work in partnership with the medical community to provide safe, effective, and sustainable one-time treatments. We are committed to transparency, reliability, and meaningful support that enables confident navigation of the patient treatment journey.

How we show up with
our partners

We collaborate to deliver our treatments to patients expeditiously. Our relationships are grounded in shared values of innovation, exceptional service, transparency, quality, reliability, trust, and respect, enabling sustainable and scalable partnerships.

Continue Exploring

Our Treatments

Discover our FDA-approved treatments for rare genetic diseases

Genetic Medicine

Learn how our approach is designed to address disease at its source