Hereditary spastic paraplegia

Charles River and Genetic Cures for Kids Announce Gene Therapy Manufacturing Collaboration

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Tuesday, November 28, 2023
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Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) and Genetic Cures for Kids Inc (GC4K), an Australian non-profit foundation focused on research programs and clinical trials to find cures for rare diseases, today announced a plasmid DNA contract development and manufacturing organization (CDMO) collaboration.
  • In recent years, Charles River has significantly broadened its cell and gene therapy portfolio to simplify complex supply chains and meet growing demand for plasmid DNA, viral vector, and cell therapy services.
  • Combined with the Company’s legacy testing capabilities, Charles River offers a comprehensive “concept-to-cure” advanced therapies solution.
  • - Golden Whitrod, Tallulah Moon’s mother and Co-Founder and President, Genetic Cures for Kids, Inc.

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

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Thursday, December 15, 2022
Research, Genetics, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Science, Creativity, Quality of life, Patient, Clinical trial, Incontinence, FDA, Mass, Primate, Safety, PROPEL, ABCD1, Trial of the century, Pain, AMN, AAV, Adrenoleukodystrophy, Mutation, Disease, Therapy, Aircraft, Tissue, Hereditary spastic paraplegia, Pharmaceutical industry, Sales

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.

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

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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.

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

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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.

Additional Preclinical Data Supports Clinical Advancement of First AAV-Based Gene Therapy for Adrenomyeloneuropathy

Retrieved on: 
Tuesday, May 17, 2022
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These data support the ongoing clinical advancement of SBT101 as a potential treatment for adrenomyeloneuropathy (AMN), a progressive, inherited, and debilitating neurodegenerative disease.

Key Points: 
  • These data support the ongoing clinical advancement of SBT101 as a potential treatment for adrenomyeloneuropathy (AMN), a progressive, inherited, and debilitating neurodegenerative disease.
  • The SBT101 preclinical data, presented at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, DC, demonstrate dose-dependent functional improvement as measured by grip strength in a mouse model of AMN.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.
  • SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN).

SwanBio Therapeutics to Present Preclinical Data on Lead Candidate at American Society of Gene & Cell Therapy

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Tuesday, April 19, 2022
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SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, will present safety, efficacy, and biodistribution data from preclinical studies of its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN) at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, DC May 16-19.

Key Points: 
  • SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, will present safety, efficacy, and biodistribution data from preclinical studies of its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN) at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, DC May 16-19.
  • These data have formed the basis of SwanBios progress to advance SBT101 into clinical trials.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.
  • SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures.

Updated Preclinical Data Support Potential of First AAV-Based Gene Therapy as a Treatment for Adrenomyeloneuropathy

Retrieved on: 
Tuesday, April 5, 2022
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These data support the potential of SBT101 as a treatment for adrenomyeloneuropathy (AMN), a progressive, inherited, and debilitating neurodegenerative disease caused by a deficiency in the ABCD1 gene which primarily affects the spinal cord.

Key Points: 
  • These data support the potential of SBT101 as a treatment for adrenomyeloneuropathy (AMN), a progressive, inherited, and debilitating neurodegenerative disease caused by a deficiency in the ABCD1 gene which primarily affects the spinal cord.
  • SwanBio will present additional data during the AAN virtual meeting April 24-26, 2022.
  • These data represent the tremendous progress weve made to date on our pipeline and scientific platform.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.

FDA Grants Orphan Drug Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Adrenomyeloneuropathy (AMN)

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Tuesday, March 15, 2022
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This designation qualifies the sponsor of the drug for certain incentives, including most notably, seven years of marketing exclusivity upon drug approval from the FDA.

Key Points: 
  • This designation qualifies the sponsor of the drug for certain incentives, including most notably, seven years of marketing exclusivity upon drug approval from the FDA.
  • SBT101 is the first AAV-based gene therapy candidate cleared by the FDA for human studies, designed to compensate for the disease-causing ABCD1 mutation in people living with adrenomyeloneuropathy (AMN).
  • Earlier this year, the FDA cleared SwanBios Investigational New Drug application for SBT101 and granted SBT101 Fast Track Designation.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.

SwanBio Therapeutics to Present New Preclinical Data Showcasing AAV-Based Gene Therapy Candidate for Adrenomyeloneuropathy at American Academy of Neurology Annual Meeting

Retrieved on: 
Friday, March 4, 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 that the company will present data supporting the clinical advancement of its lead candidate, SBT101, at the American Academy of Neurology (AAN) 2022 Annual Meeting.

Key Points: 
  • SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced that the company will present data supporting the clinical advancement of its lead candidate, SBT101, at the American Academy of Neurology (AAN) 2022 Annual Meeting.
  • The meeting is being held in Seattle April 2-7, 2022, and virtually April 24-26, 2022.
  • In preclinical non-human primate models, weve observed good tolerability and dose-dependent biodistribution in the CNS with SBT101, which we believe will translate into patients in the clinic.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions.

FDA Grants Fast Track Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Patients With Adrenomyeloneuropathy (AMN)

Retrieved on: 
Wednesday, February 16, 2022
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AMN is an adult-onset progressive, degenerative spinal cord disease caused by mutations in the ABCD1 gene leading to loss of mobility, incontinence and pain.

Key Points: 
  • AMN is an adult-onset progressive, degenerative spinal cord disease caused by mutations in the ABCD1 gene leading to loss of mobility, incontinence and pain.
  • We look forward to continuing to advance SBT101 as we work toward our goal of bringing life-changing treatments to patients.
  • The Fast Track process is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.
  • SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions.