ABCD1

Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Retrieved on: 
Sunday, February 4, 2024
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Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

Key Points: 


Orphan designation: 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride Treatment of adrenoleukodystrophy, 18/11/2016 Positive

SwanBio Advances to Higher-Dose Cohort in First-in-Human Study of Gene Therapy for Adrenomyeloneuropathy

Retrieved on: 
Wednesday, October 4, 2023
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, AMN, DSMB, Adrenoleukodystrophy, Clinical trial, Mutation, CTA, AAV, Trial of the century, Data monitoring committee, Degenerative disease, ABCD1, Patient, Central committee, Therapy, PROPEL, Safety, Pharmaceutical industry

SwanBio Therapeutics , a gene therapy company developing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced it has begun the second dose-escalation cohort of its first interventional clinical study, PROPEL.

Key Points: 
  • SwanBio Therapeutics , a gene therapy company developing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced it has begun the second dose-escalation cohort of its first interventional clinical study, PROPEL.
  • PROPEL is a Phase 1/2 first-in-human clinical trial assessing the safety and efficacy of SBT101, an investigational gene therapy designed to compensate for the ABCD1 mutation that causes adrenomyeloneuropathy (AMN).
  • An independent Data Safety Monitoring Board (DSMB) recommended progressing the study to the higher-dose cohort following the review of the initial safety data readout in August.
  • No serious adverse events related to the procedure, nor the gene therapy product, have been reported to date.

SwanBio Therapeutics Initiates First-in-Human Study of AAV Gene Therapy for Adrenomyeloneuropathy

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Thursday, December 15, 2022
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SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced the initiation of its PROPEL clinical trial.

Key Points: 
  • SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced the initiation of its PROPEL clinical trial.
  • The first-in-human trial will study the companys lead candidate, SBT101, an investigational AAV-based gene therapy intended to treat the progressive and debilitating neurodegenerative disease adrenomyeloneuropathy (AMN).
  • SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN).
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.

Viking Therapeutics Announces FDA Has Lifted Clinical Hold on Phase 1b Trial of VK0214 in Patients with X-ALD

Retrieved on: 
Tuesday, July 19, 2022
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SAN DIEGO, July 19, 2022 /PRNewswire/ -- Viking Therapeutics, Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced that the FDA has lifted the clinical hold placed on the clinical trial of VK0214 in patients with X-linked adrenoleukodystrophy (X-ALD). As a result, the company expects to resume study enrollment in the coming weeks.

Key Points: 
  • The FDA lifted the clinical hold following its review of results from an in vivo genotoxicity study that it had requested due to the study being conducted with multiple doses in patients.
  • Viking had planned to conduct this study prior to Phase 2 but accelerated its execution based on FDA's request.
  • The company does not believe that the long-term development timeline for VK0214 has been significantly impacted by the temporary clinical hold.
  • The Phase 1b study of VK0214 is designed to enroll patients with the adrenomyeloneuropathy (AMN) form of X-ALD.

GI Dynamics Announces the Presentation of Updated Data Sets at the 2022 Meeting of the American Diabetes Association

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Thursday, July 14, 2022
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of City Hospital, Birmingham, U.K. at the 82nd Annual Scientific Sessions of the American Diabetes Association (ADA) in New Orleans on June 6, 2022.

Key Points: 
  • of City Hospital, Birmingham, U.K. at the 82nd Annual Scientific Sessions of the American Diabetes Association (ADA) in New Orleans on June 6, 2022.
  • Both studies demonstrate the safety and efficacy profile that EndoBarrier provides patients diagnosed with type 2 diabetes and/or obesity.
  • The Worldwide EndoBarrier Registry continues to support a positive benefit:risk profile for patients with type 2 diabetes and/or obesity treated with EndoBarrier.
  • Many benefits are seen in patients including considerable improvements in weight, HbA1c, systolic blood pressure and cholesterol as shown below:

Poxel Announces the Publication of Two Preclinical Articles on X-Linked Adrenoleukodystrophy for PXL065 and PXL770

Retrieved on: 
Tuesday, July 5, 2022
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POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announces the publication of two preclinical articles on X-Linked Adrenoleukodystrophy (ALD) for PXL065 and PXL770.

Key Points: 
  • POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announces the publication of two preclinical articles on X-Linked Adrenoleukodystrophy (ALD) for PXL065 and PXL770.
  • An article on PXL065 has been published in The Journal of Inherited Metabolic Disease (JIMD) and is entitled Therapeutic potential of deuterium-stabilized (R)-pioglitazone - PXL065 - for X-linked adrenoleukodystrophy.
  • These publications describe similar beneficial preclinical profiles of PXL770 and PXL065 in models of ALD.
  • Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

SwanBio Presents Design of Innovative Natural History Study Aimed to Evolve Understanding of Adrenomyeloneuropathy and Inform Future Treatments

Retrieved on: 
Monday, June 27, 2022
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CYGNET is the first AMN clinical study to feature wearables, which may help us identify sensitive outcomes related to clinically relevant changes early in men with AMN.

Key Points: 
  • CYGNET is the first AMN clinical study to feature wearables, which may help us identify sensitive outcomes related to clinically relevant changes early in men with AMN.
  • SwanBio anticipates recruiting approximately 80 patients for the CYGNET natural history study; as of early June, the study was over 40% enrolled across five different global sites.
  • Clinicians or patients interested in learning more about this study can review SwanBios CYGNET flyer or contact SwanBio at [email protected] .
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.

FDA Advisory Committee Unanimously Endorses eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy

Retrieved on: 
Thursday, June 9, 2022
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On the question Do the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD)?

Key Points: 
  • On the question Do the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD)?
  • We are grateful to the families, clinicians and committee members who participated in todays advisory committee discussion and remain committed to working with the FDA as it completes its review of the eli-cel Biologics License Application.
  • In addition to granting eli-cel BLA priority review, the FDA previously granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation.
  • Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the worldsetting the standard for industry.

bluebird bio Announces Posting of Briefing Documents for Upcoming FDA Advisory Committee Meeting

Retrieved on: 
Tuesday, June 7, 2022
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The advisory committee meeting will take place June 9-10, 2022.

Key Points: 
  • The advisory committee meeting will take place June 9-10, 2022.
  • The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.
  • bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
  • bluebird bio is a trademark of bluebird bio, Inc.

SwanBio Therapeutics Announces $56 Million Series B Financing to Advance Novel Gene Therapies for Neurological Conditions

Retrieved on: 
Wednesday, May 18, 2022
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SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies for the treatment of devastating, inherited neurological conditions, today announced the completion of a $56 million Series B financing round, led by founding investors Syncona Limited and Mass General Brigham Ventures.

Key Points: 
  • SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies for the treatment of devastating, inherited neurological conditions, today announced the completion of a $56 million Series B financing round, led by founding investors Syncona Limited and Mass General Brigham Ventures.
  • Since our initial investment, SwanBio has made excellent progress, including rapidly advancing its lead program for AMN toward the clinic.
  • We are excited by the potential we see in this business to become a leading gene therapy company, delivering life-changing treatments to patients with debilitating neurological disorders.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.