Elixirgen Therapeutics’ treatment for a set of genetic conditions that can lead to bone marrow failure has been able to elongate the caps of chromosomes in two patients with telomere biology disorders (TBD).
Early results from the company’s ongoing Phase I/II trial examining EXG-34217 (NCT04211714) saw a successful elongation of the chemical that acts to protect parts of a person’s chromosomes in two patients.
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According to the Mayo Clinic, there are currently no curative treatments for the indication with care mostly limited to prevention or transplantation. Results from the single-arm open-label trial have been able to elicit an extension of CD34+ cells ex vivo in two out of the 12 patients enrolled on the trial.
The trial data, published in the New England Journal of Medicine (NEJM) Evidence, comes after the company’s gene therapy was previously granted the hat-trick of rare pediatric disease, regenerative medicine advanced therapy (RMAT), and orphan drug designations by the US Food and Drug Administration (FDA).
Kasiani Myers, principal investigator for the trial, said: “The data published today in NEJM Evidence demonstrates sustained telomere elongation in blood cells in patients with TBDs without toxicity, an outcome that has not been achieved by other treatments for those with this disorder.
“In addition, EXG-34217 administration did not require a preconditioning regimen or immunosuppression typically required for other treatment regimens, which is critical in this radiation- and chemotherapy-sensitive population. There is a significant unmet need in this space since the only option for patients with TBDs who develop bone marrow failure is hematopoietic stem cell transplantation, which can result in life-threatening complications.”
EXG-34217 is described as a dose of autologous CD34+ haematopoietic stem cells containing a protein responsible for the elongation of stability of telomeres. Typically, as a person gets older, the gapping of the end of the telomere chromosomes gets shorter, allowing DNA strands to separate. One of the telomere disorders frequently resulting from the condition is known as dyskeratosis congenita, which can lead to bone marrow failure and affect the skin and other organs.
Research by GlobalData found that there are currently only two marketed therapies for dyskeratosis congenita. The company is now enrolling participants aged 12 years and older, in the Phase I/II study (NCT04211714) evaluating EXG-34217 in patients with TBDs with bone marrow failure.
Elixirgen Therapeutics CEO Aki Ko commented: “This unique gene plays a critical role in several key genomic functions, including telomere extension, to restore the integrity and potency of stem cells. EXG-34217 harnesses the power of this protein and allows us to provide a potential new treatment option for TBDs. This therapy is genotype and mutation-independent since elongation of telomeres by ZSCAN4 is telomerase-independent.”
Elsewhere in the field of gene therapy for rare disorders, An investigator-initiated trial examining YolTech Therapeutics’ gene-edited treatment for patients living with primary hyperoxaluria type 1. Meanwhile, Entrada Therapeutics can proceed with the development of its Duchenne muscular dystrophy therapy, ENTR-601-44 after the FDA lifted a two-year clinical hold.
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