ABCD1

bluebird Provides Update on FDA Review Timelines for Betibeglogene Autotemcel (beti-cel) for Beta-Thalassemia and Elivaldogene Autotemcel (eli-cel) for Cerebral Adrenoleukodystrophy (CALD)

Retrieved on: 
Tuesday, January 18, 2022
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(NASDAQ: BLUE) today announced that the US Food and Drug Administration (FDA) has extended the review period for the biologics licensing applications (BLA) for its lentiviral vector gene therapies betibeglogene autotemcel (beti-cel) for -thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).

Key Points: 
  • (NASDAQ: BLUE) today announced that the US Food and Drug Administration (FDA) has extended the review period for the biologics licensing applications (BLA) for its lentiviral vector gene therapies betibeglogene autotemcel (beti-cel) for -thalassemia and elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD).
  • The extension of the FDA review timeline does not relate to new safety events for either beti-cel or eli-cel.
  • The FDA accepted the BLA for eli-cel for patients with cerebral adrenoleukodystrophy (CALD) under the age of 18 for priority review in December 2021.
  • The FDA previously granted both beti-cel and eli-cel Orphan Drug status, Breakthrough Therapy designation and Rare Pediatric Disease designation.

bluebird bio Announces FDA Priority Review of Biologics License Application for eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) in Patients Without a Matched Sibling Donor

Retrieved on: 
Friday, December 17, 2021
Stem Cells, Biotechnology, FDA, Health, General Health, Pharmaceutical, Oncology, Research, Genetics, Science, Clinical Trials, White matter, MDS, Spinal cord, Time, Survival, Breakthrough therapy, Risk, Health, CALD, Disease, Graft-versus-host disease, Vomiting, Disorder, Communication, Biologics license application, Forward-looking statement, FDA, Myelodysplastic syndrome, Diagnosis, U.S. Securities and Exchange Commission, Sickle cell disease, Lentivirus, SUSAR, Safety, Security (finance), Pancytopenia, ALD, Neurodegeneration, Research, Bluebird, Male, View, Food, BLA, Death, National Medical Products Administration, Thought, Brain, Private Securities Litigation Reform Act, Industry, Lists of diseases, Bird, Bluebird bio, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Adverse effect, Goal, Patient, Mortality, PDUFA, Priority review, Sumathi Best Television Current Reporting Award, LVV, Stem cell, MFD, Pharmaceutical industry, ABCD1

It is estimated that more than 70% of patients diagnosed with CALD do not have a matched sibling donor.

Key Points: 
  • It is estimated that more than 70% of patients diagnosed with CALD do not have a matched sibling donor.
  • The BLA for eli-cel is supported by efficacy and safety data from the completed Phase 2/3 Starbeam study (ALD-102) (N=32).
  • All patients who completed ALD-102, as well as those who will complete ALD-104, are invited to participate in a long-term follow-up study (LTF-304).
  • bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.

Poxel Announces its Participation at Upcoming Scientific Conferences Related to Adrenoleukodystrophy (ALD)

Retrieved on: 
Monday, September 27, 2021
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POXEL SA (Euronext POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced that the Poxel team will participate at several upcoming scientific conferences related to X-linked adrenoleukodystrophy (ALD), a severe orphan neurometabolic disease with no approved therapies.

Key Points: 
  • POXEL SA (Euronext POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders, today announced that the Poxel team will participate at several upcoming scientific conferences related to X-linked adrenoleukodystrophy (ALD), a severe orphan neurometabolic disease with no approved therapies.
  • Poxel's scientific team will present data and plans pertaining to ALD that align with the recently announced new strategic direction of increasing Poxels focus on rare metabolic diseases.
  • Poxel is committed to focus its pipeline on high value, rare metabolic indications and NASH, with the goal of creating pipeline synergies, maximizing resources, and driving shareholder value.
  • Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

bluebird bio Receives EC Approval for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age With Early Cerebral Adrenoleukodystrophy (CALD) Without Matched Sibling Donor

Retrieved on: 
Wednesday, July 21, 2021
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bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. SKYSONA is the first one-time gene therapy approved in the European Union (EU) to treat CALD, a rare neurodegenerative disease that occurs in childhood and can rapidly lead to progressive, irreversible loss of neurologic function, and death.

Key Points: 
  • SKYSONA is a one-time gene therapy custom-designed to treat the underlying cause of the neurologic condition CALD.
  • SKYSONA uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patients own HSCs.
  • The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of VLCFAs.
  • The marketing authorization of SKYSONA is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102).

bluebird bio Receives Positive CHMP Opinion for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age with Early Cerebral Adrenoleukodystrophy (CALD)

Retrieved on: 
Friday, May 21, 2021
Research, Medical Supplies, Genetics, Clinical trials, Stem cells, Biotechnology, General Health, Pharmaceutical, Health, Science, Branches of biology, Life sciences, Biology, ATP-binding cassette transporters, Bioethics, Gene therapy, ABCD1, Therapy, Adrenoleukodystrophy

A CHMP positive opinion is one of the final steps before the EC decides whether to authorize a new medicine.

Key Points: 
  • A CHMP positive opinion is one of the final steps before the EC decides whether to authorize a new medicine.
  • We support all initiatives that, like gene therapy, could change the lives of patients.
  • The addition of the functional ABCD1 gene allows patients to produce the ALD protein, which is thought to facilitate the breakdown of VLCFAs.
  • To date, 32 patients have been treated with SKYSONA in ALD-102, and 30/32 patients were evaluable for follow-up at Month 24.

bluebird bio Presents Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)

Retrieved on: 
Monday, March 15, 2021
Oncology, Health, FDA, Genetics, Clinical trials, Biotechnology, Branches of biology, Neurological disorders, Biology, Rare diseases, Peroxisomal disorders, Bluebird bio, Adrenoleukodystrophy, ATP-binding cassette transporters, Gene therapy, Leukodystrophy, ABCD1, Very long chain fatty acid

The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs.

Key Points: 
  • The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs.
  • All reported data below from ALD-102 are as of October 2020 and all reported data below from LTF-304 are as of November 2020.
  • In October 2020, the European Medicines Agency (EMA) accepted bluebird bios marketing authorization application (MAA) for its investigational eli-cel gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD).
  • eli-cel, Lenti-D and bluebird bio are trademarks of bluebird bio, Inc.

bluebird bio to Present Data from Clinical Development Program of Lenti-DTM Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress

Retrieved on: 
Tuesday, September 3, 2019
Stem cells, Biotechnology, Pharmaceutical, Oncology, Health, Genetics, Branches of biology, Biology, Genetics, Rare diseases, Gene delivery, Peroxisomal disorders, Fatty acids, Very long chain fatty acid, Adrenoleukodystrophy, Leukodystrophy, Gene therapy, ABCD1

bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) will be presented at the 13th European Pediatric Neurology Society (EPNS) Congress, to be held September 17 21, 2019 in Athens, Greece.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) announced that updated data from the clinical development program for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy (CALD) will be presented at the 13th European Pediatric Neurology Society (EPNS) Congress, to be held September 17 21, 2019 in Athens, Greece.
  • Gene therapy for CALD consists of the patients own stem cells transduced with Lenti-D lentiviral vector that encodes ABCD1 cDNA for human adrenoleukodystrophy protein (ALDP).
  • This allows for the production of functional ALDP that enables the local degradation of very long chain fatty acids (VLCFAs) in the brain.
  • Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD.