for fighting life-altering disease.
our focus
We are focused on developing and delivering new therapies for severe genetic diseases that currently have limited or no treatment options. Our goal is to provide hope for a better life – and more bluebird days – for patients and their families.
β-Thalassemia (Beta-thal)
People living with the most severe form of beta-thal cannot produce sufficient amounts of a key component of hemoglobin—a vital protein that helps red blood cells carry oxygen that the body needs to function. They require frequent transfusions of healthy red blood cells to survive, tethering them to the healthcare system for life and increasing their risk for severe complications.
Cerebral Adrenoleukodystrophy (CALD)
CALD is a rare, rapidly progressive neurodegenerative disease primarily affecting young children. The x-linked condition is caused by a mutation in the ABCD1 gene and results in rapid loss of neurological function after the initial onset of symptoms. Without treatment, nearly half of patients die within five years of symptom onset.
Sickle Cell Disease (SCD)
SCD is much more than pain. This inherited disease results in progressive organ damage, significantly reduced quality of life and shortened life expectancy. Patients often require hospitalizations to manage their symptoms, and despite advances, more than half of all people with SCD die before the age of 50. Change is long overdue for patients whose disease has long been overlooked.
National Organization for Rare Disorders (NORD). Beta thalassemia. ©2021 Available at https://rarediseases.org/rare-diseases/thalassemia-major/. Accessed April 2022.
Engelen M, Kemp S, de Visser M, et al. X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management. Orphanet J Rare Dis. 2012;7:51.
Moser HW, Mahmood A, Raymond GV. X-linked adrenoleukodystrophy. Nature Clin Pract Neurol. 2007;3(3):140-51.
Miller W. Stem cell-transplantation therapy for adrenoleukodystrophy: current perspectives. J Neurorestoratology. 2017;5:5-19.
Mahmood A, Dubey P, Moser HW, et al. X-linked adrenoleukodystrophy: therapeutic approaches to distinct phenotypes. Pediatr Transplant. 2005;9(Suppl7):55-62.
Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018;4:18010.
Payne AB, Mehal JM, Chapman C, Haberling DL, Richardson LC, Bean CJ, Hooper WC. Trends in sickle cell disease-related mortality in the United States, 1979-2017. Ann Emerg Med. 2020; 76 (3S):S28-S36.
Engelen M, Kemp S, de Visser M, et al. X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management. Orphanet J Rare Dis. 2012;7:51.
Moser HW, Mahmood A, Raymond GV. X-linked adrenoleukodystrophy. Nature Clin Pract Neurol. 2007;3(3):140-51.
Miller W. Stem cell-transplantation therapy for adrenoleukodystrophy: current perspectives. J Neurorestoratology. 2017;5:5-19.
Mahmood A, Dubey P, Moser HW, et al. X-linked adrenoleukodystrophy: therapeutic approaches to distinct phenotypes. Pediatr Transplant. 2005;9(Suppl7):55-62.
Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018;4:18010.
Payne AB, Mehal JM, Chapman C, Haberling DL, Richardson LC, Bean CJ, Hooper WC. Trends in sickle cell disease-related mortality in the United States, 1979-2017. Ann Emerg Med. 2020; 76 (3S):S28-S36.