Adrenoleukodystrophy (ALD) is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, an insulating membrane that surrounds nerve cells in the brain. People with ALD accumulate high levels of saturated, very long chain fatty acids (VLCFA) in the brain and adrenal cortex because they do not produce the enzyme that breaks down these fatty acids in the normal manner. The loss of myelin and the progressive dysfunction of the adrenal gland are the primary characteristics of ALD. ALD has two subtypes. The most common is the X-linked form (X-ALD), which involves an abnormal gene located on the X-chromosome.
Bluebird bio, Inc. and Gritstone Oncology, Inc. today announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy.
"Lorenzo's Oil" was to help a seriously ill boy suffering from a peroxisomal disorder (adrenoleukodystrophy/ALD). The true story was turned into a film which made the rare disease well known. Scientists from the University of Bonn, the German Center for Neurodegenerative Diseases and the German Cancer Research Center investigated such peroxisomal diseases on fruit flies.
Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.
In a recent clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy (CALD) -- a neurodegenerative disease that typically claims young boys' lives within 10 years of diagnosis -- effectively stabilized the disease's progression in 88 percent of patients, researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Massachusetts General Hospital report today.
Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced that it has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration to conduct a Phase 2 study of VK2809 in patients with hypercholesterolemia and fatty liver disease.
Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced the initiation of dosing in the company's Phase 2 clinical trial of VK5211 in patients who recently suffered a hip fracture.
Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced the successful completion of a short-term safety, tolerability, and pharmacokinetic study of VK5211 in healthy elderly subjects. VK5211, the company's lead program for muscle and bone disorders, is an orally available, non-steroidal selective androgen receptor modulator (SARM) being developed for the treatment of patients recovering from non-elective hip fracture surgery.
A drug used to control Type II diabetes can help repair the spinal cords of mice suffering from the inherited disease adrenoleukodystrophy which, untreated, leads eventually to a paralysis, a vegetative state and death.
The Bellvittge Biomedical Research Institute has signed a licensing agreement with the Spanish biotechnology company Minoryx of a patent for the treatment of X-linked adrenoleukodystrophy, a rare serious neurodegenerative disease which has no effective treatment.
bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.
bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD).
genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents.
bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that the company has secured an additional $30 million in financing.
A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe immunodeficiency disorder.
bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.
Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."
Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, announced today that it has completed a $35 million Series B financing with new investors Third Rock Ventures and Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. Proceeds from the financing will be used to advance current clinical programs, strengthen platform capabilities and further expand the team.
Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today the company’s clinical program to treat Adrenoleukodystrophy (ALD). The company is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM).
The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.
Scientists have used human stem cells to dramatically improve the condition of mice with a neurological condition similar to a set of diseases in children that are invariably fatal, according to an article in the June issue of the journal Cell Stem Cell.