Crigler–Najjar syndrome

Genethon Pursues Different Strategies for Ensuring Patient Access to Gene Therapies for Rare Diseases

Retrieved on: 
Thursday, February 29, 2024

Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.

Key Points: 
  • Genethon , a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.
  • Globally more than 300 million people, most of them children, are living with 7,000 rare diseases.
  • Because patient populations are small, these diseases don’t readily fit into the biopharma industry’s business models.
  • “Despite challenges in generating interest with biopharma companies and investors, Genethon will never abandon patients suffering from rare and ultra-rare diseases,” said Dr. Revah.

Applied BioMath, LLC Announces Participation at AAPS 2023 PharmSci 360

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Thursday, October 19, 2023

CONCORD, Mass., Oct. 19, 2023 /PRNewswire/ -- Applied BioMath ( www.appliedbiomath.com ), the industry-leader in providing model-informed drug discovery and development (MID3) support to help accelerate and de-risk therapeutic research and development (R&D), today announced their participation at AAPS 2023 PharmSci 360 occurring October 22-25, 2023 in Orlando, FL.

Key Points: 
  • CONCORD, Mass., Oct. 19, 2023 /PRNewswire/ -- Applied BioMath ( www.appliedbiomath.com ), the industry-leader in providing model-informed drug discovery and development (MID3) support to help accelerate and de-risk therapeutic research and development (R&D), today announced their participation at AAPS 2023 PharmSci 360 occurring October 22-25, 2023 in Orlando, FL.
  • Applied BioMath will participate in multiple presentations during the conference.
  • Joshua Apgar, PhD, Co-founder and CSO at Applied BioMath will give a Speaker Spotlight presentation entitled, "Evolving Clinical Pharmacology and Translational PK/PD Approaches for New Modalities" on Tuesday, October 24th from 9:30-10:30am.
  • To learn more about Applied BioMath, visit www.appliedbiomath.com .

Positive Phase 1/2 Clinical Trial Results of Genethon’s Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease, Published in The New England Journal of Medicine

Retrieved on: 
Tuesday, August 22, 2023

Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine.

Key Points: 
  • Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced today positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine.
  • The data demonstrate the possibility of restoring expression of liver enzyme UGT1A1 by using gene therapy in cases of Crigler-Najjar syndrome.
  • The Phase 1/2 trial results published in an article titled, “Gene Therapy in Patients with the Crigler-Najjar Syndrome,” showed GNT-0003 lowered bilirubin levels below the toxic threshold with a single intravenous injection.
  • This is the first proof of the efficacy of a gene therapy in a metabolic disease of the liver.

Hansa Biopharma and Genethon announce collaboration to develop imlifidase as pre-treatment to gene therapy in Crigler-Najjar syndrome patients with anti-AAV antibodies

Retrieved on: 
Thursday, April 27, 2023

The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.

Key Points: 
  • The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
  • This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
  • Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003.
  • Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
    Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy.

Hansa Biopharma and Genethon announce collaboration to develop imlifidase as pre-treatment to gene therapy in Crigler-Najjar syndrome patients with anti-AAV antibodies

Retrieved on: 
Thursday, April 27, 2023

The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.

Key Points: 
  • The presence of circulating NAbs today excludes patients from entering clinical studies with potentially curative gene therapy treatments and from future access to approved gene therapies.
  • This research collaboration further validates Hansa's commitment in gene therapy and underscores the important role that our antibody-cleaving enzyme technology can play in ensuring that even more patients can benefit from life-saving gene therapies".
  • Through the collaboration announced today, patients with Crigler-Najjar and pre-formed antibodies to AAV8 will be enrolled in a study with similar design where imlifidase is evaluated as a pre-treatment to enable gene therapy treatment with GNT-0003.
  • Crigler-Najjar syndrome is an ultra-rare disease affecting less than one case per one million people per year.1
    Frédéric Revah, CEO, Genethon added: "Patients with pre-existing neutralizing antibodies against AAV vectors cannot today benefit from gene therapy.

Genethon Given PRIME Status by EMA for Gene Therapy To Treat Crigler-Najjar Syndrome, a Rare Liver Disease

Retrieved on: 
Tuesday, March 7, 2023

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), today announced that the European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.

Key Points: 
  • Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), today announced that the European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.
  • “We’re excited about the EMA’s PRIME recognition of GNT-0003,” said Genethon CEO Frederic Revah.
  • Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia).
  • The gene therapy, administered intravenously, was designed by Genethon's Immunology and Gene Therapy of Liver Diseases team, led by Dr. Giuseppe Ronzitti.

Genethon Launches Pivotal Clinical Trial of Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease

Retrieved on: 
Tuesday, January 10, 2023

Genethon , a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease.

Key Points: 
  • Genethon , a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), has launched a pivotal clinical trial in Europe for treatment of Crigler-Najjar syndrome, a life threatening liver disease.
  • “This new step demonstrates once again the excellence of the research conducted at Genethon, which is today one of the major players in gene therapy at the international level."
  • Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia).
  • The gene therapy, administered intravenously, was designed by Genethon's Immunology and Gene Therapy of Liver Diseases team, led by Dr. Giuseppe Ronzitti.

Rare Liver Disease Summit Seeks to Improve Patient Health Outcomes

Retrieved on: 
Tuesday, June 28, 2022

FAIRFIELD, N.J., June 28, 2022 /PRNewswire/ -- The American Liver Foundation (ALF) will bring together leading rare liver disease experts and clinicians, patients, caregivers, advocacy organizations and industry representatives for a virtual two-day Summit, June 28-29, to identify solutions which can improve the health outcomes of those affected by rare liver diseases.

Key Points: 
  • American Liver Foundation brings together experts, patients and families in a virtual two-day event
    FAIRFIELD, N.J., June 28, 2022 /PRNewswire/ -- The American Liver Foundation (ALF) will bring together leading rare liver disease experts and clinicians, patients, caregivers, advocacy organizations and industry representatives for a virtual two-day Summit , June 28-29, to identify solutions which can improve the health outcomes of those affected by rare liver diseases.
  • "Patients and families struggling with a rare liver disease need our support and deserve better ways to manage their disease," said Lorraine Stiehl, Chief Executive Officer, American Liver Foundation.
  • "So little is known about so many different rare liver diseases that Summits like this one are essential to shedding light on rare liver diseases and advancing knowledge, research and treatments," said Bruce Dimmig of Surprise, AZ, who suffers from liver diseases including the rare disease Nodular Regenerative Hyperplasia .
  • The American Liver Foundation is the nation's largest non-profit organization focused solely on promoting liver health and disease prevention.