Aki Ko, Chief Executive Officer and co-founder of Elixirgen Therapeutics, discusses treatment advances in telomere biology disorders.
Telomere biology disorders are a group of conditions that occur when patients have premature or faster shortening of telomeres due to genetic mutations. This can cause impaired function of many organ systems over time, most commonly affecting bone marrow. Symptoms of telomere biology disorders vary but commonly include:
- Slow creation of blood cells
- Abnormal pigmentation
- Eye and/or dental abnormalities
- Esophageal and/or urethral stenosis
- Avascular necrosis of femur and/or humerus
- Osteopenia
- Pulmonary fibrosis
- Liver disease
As Mr. Ko explains, currently, these disorders are mainly treated with hematopoietic stem cell transplants. However, this treatment poses challenges such as donor matching, preconditioning, and immunosuppression.
EXG-34217 is an autologous cell therapy that addresses the root cause in the telomere biology disorders.
In a recent phase 1/2 study, EXG-34217 administration led to the successful ex vivo elongation in the telomeres by 1.24-fold. This observation means that telomeres have been brought into a healthy range. Additionally, no long-term adverse effects occurred up to 9 months following treatment.
As this trial is still in progress, Mr. Ko states that additional results are hopeful for the near future.
For more information on rare congenital and genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/