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Recent popular reports from Yimaike★Focus on stem cell therapy! The potential and challenges of stem cellsYimai Meng broke the news ★ Gu Yuchun, Chief Scientist of Chengnuo Medical: Continue to lead, China's first "spot type" allogeneic source CAR-NK is about to enter the clinical stageFengke Transcend May 24, 2021 / Medical EMedClub News/--Today, Bluebird bio (Bluebird bio) treatment of cerebral adrenal leukodystrophy (CALD) gene therapy Skysona (elivaldogene autotemcel, eli-cel) marketing authorization has been granted by the European Medicines Agency for human drugs The committee’s positive recommendation.
CALD is the most serious form of adrenal leukodystrophy (ALD).
It is caused by mutations in the ABCD1 gene and is a rare X chromosome metabolic disorder.
Mutations in the ABCD1 gene lead to the lack of adrenal leukodystrophin (ALDP) and the toxic accumulation of very long chain fatty acids (VLCFA), thereby destroying the protective layer of nerve cells-myelin.
CALD is manifested as a functional disability caused by rapid neurodegeneration and eventually death.
CALD usually occurs in early childhood and progresses rapidly if left untreated.
Currently, the only treatment option for CALD patients is allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Although allo-HSCT is effective for patients with early CALD.
However, allo-HSCT has potential complications, including transplantation failure, graft-versus-host disease (GvHD), and opportunistic infections, especially in patients receiving non-sibling-matched donor cell transplants.
▲ Image source: Bluebird eli-cel uses Bluebird’s proprietary lentiviral vector to introduce a functional copy of the ABCD1 gene into the patient’s own hematopoietic stem cells, and then the patient will undergo myeloid clearing and transplantation.
Adding a functional copy of the ABCD1 gene can promote the production of functional ALDP enzymes, reduce the accumulation of VLCFA, and thereby inhibit CALD neuropathy.
Bluebird Bio’s eli-cel is a gene therapy prepared using patients’ autologous cells.
This therapy may reduce the risk of fatal immune complications when allogeneic cells are used for transplantation.
In April 2021, Bluebird Bio announced the latest data of eli-cel, showing that one-time gene therapy has positive durability and effectiveness.
Recommended reading: Bluebird Gene Therapy has achieved results in the treatment of childhood encephalopathyYimai Meng broke the news Reference materials: 1.
BioValley "Bluebird CALD Gene Therapy Marketing Application Receives Active Recommendation from CHMP" Yimai Ke has always been committed to the forefront of bio-innovative drugs Original news reports on technology, industry trends, industry insights, etc.
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