OUR APPROACH TO GENETIC MEDICINE

One-time
treatments
designed to treat the underlying cause of disease

Using a patient's own cells, our approach focuses on one-time treatments that are designed to target the underlying cause of genetic disease and deliver durable clinical benefit.

What is genetic disease?

Genetic diseases are typically inherited from birth and are caused by mutations, or changes, in a person's genes, or DNA.

DNA contains instructions for making proteins. Mutations can disrupt these instructions, leading to the body producing absent, deficient, or defective proteins.

Because proteins are essential for how cells function, these changes can disrupt normal biological processes which can result in serious health complications.

In sickle cell disease, a mutation in the beta-globin gene leads to the production of abnormal hemoglobin (Hb), causing red blood cells to deform and restrict blood flow

Icon of a full IV bag, representing genetic medicine

In beta-thalassemia, a mutation in the beta-globin gene leads to the reduction or elimination of functional hemoglobin (Hb), causing chronic anemia and transfusion dependence

What is genetic medicine?

Ex vivo autologous cell therapy is a form of genetic medicine that uses a patient's own cells to deliver the therapeutic benefit. Cells are collected, modified outside the body, and then returned to the patient, where they then produce the protein the body needs to treat a genetic disease.

The Genetix Approach

Genetix pioneered the field of ex vivo autologous cell therapy with our gene addition technology.

This approach begins and ends with the patient's own cells, using a precise process to introduce functional genes and restore the protein the body needs to treat a genetic disease.

Collecting a patient's cells

The Genetix treatment process begins with a patient's own cells, which are collected through a process called apheresis. Before treatment, these cells contain a genetic mutation that prevents production of functional protein, which in this case is hemoglobin (Hb).

Our approved gene addition cell therapies

No off-target editing

Does not require DNA
cutting, avoiding the risk of
off-target editing associated
with gene editing

Optimized cell health

Avoids electroporation during manufacturing, supporting cell health, enabling the majority of patients to receive treatment from a single cell collection

Long-term durability

Backed by 30 years of research and clinical experience, with hundreds of patients treated and data extending 10+ years

Our approved genetic therapies

Built on our gene addition technology, these therapies are designed to address the underlying cause of disease.

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Our Treatments

Discover our FDA-approved treatments for rare genetic diseases

Pipeline

Explore our clinical programs and ongoing research shaping what’s next