Genethon and Hansa Biopharma Launch Phase 2 Trial for New Gene Therapy in Crigler-Najjar Syndrome
In a groundbreaking collaboration, Hansa Biopharma and Genethon have announced the initiation of a Phase 2 clinical trial aimed at evaluating the efficacy and safety of imlifidase in patients suffering from severe Crigler-Najjar syndrome. This trial, named GNT-018-IDES, seeks to address a significant challenge in gene therapy – the presence of pre-existing anti-AAV (adeno-associated virus) antibodies that hinder effective treatment.
Imlifidase is a unique enzyme therapy that targets and cleaves immunoglobulin G (IgG) antibodies, which are often a barrier to successful gene therapy. By administering imlifidase prior to the gene therapy GNT-0003, the trial aims to facilitate treatment in patients with severe forms of the disease who possess these objectionable antibodies.
According to Søren Tulstrup, the President and CEO of Hansa Biopharma, up to one in three individuals with anti-AAV antibodies are unable to benefit from gene therapies utilizing AAV vectors. This highlights the critical importance of their collaboration with Genethon, as it represents a pioneering effort to extend the benefits of gene therapy to a broader patient population.
The trial will involve three patients aged 18 years or older, who must meet specific criteria, including the presence of pre-formed antibodies against AAV serotype 8 (AAV8). Initially, participants will enter a three-month observational phase to monitor their condition before receiving the treatment regimen, which consists of a dose of imlifidase followed by GNT-0003.
Genethon's CEO, Frédéric Revah, emphasized the organization’s commitment to developing innovative strategies that widen access to gene therapies for patients facing rare diseases. Currently, patients with anti-AAV antibodies have limited options, and GNT-018-IDES represents a critical step forward, fueled by years of research dedicated to understanding and manipulating the immune response to AAV.
GNT-0003, the gene therapy under investigation, is already undergoing evaluation in pivotal trials, following promising results from earlier Phase 1-2 dose escalation studies that indicated both safety and efficacy. Additionally, it has received PRIME (PRIority MEdicine) designation from the European Medicines Agency (EMA), signifying its potential to address unmet medical needs.
The implications of this trial are significant. Crigler-Najjar syndrome, a rare genetic disorder characterized by the inability of the liver to process bilirubin due to a deficiency in the UGT1A1 enzyme, can cause severe neurological damage and developmental issues if not treated promptly. Patients currently undergo invasive treatments, including phototherapy for extensive hours each day, to manage their bilirubin levels.
Currently classified as an ultra-rare condition affecting less than one individual per million each year, Crigler-Najjar poses substantial treatment challenges. Success in this trial could pave the way for the first gene therapy specifically targeted towards this debilitating syndrome.
Hansa Biopharma, noted for its innovative approaches to rare immunological conditions, has developed imlifidase to help facilitate kidney transplants in sensitized patients, unlocking a pathway to life-saving treatments where options were previously limited.
As the trial progresses, both Hansa Biopharma and Genethon aim to release data in 2025, which could mark a pivotal moment in both their missions to revolutionize the way rare genetic diseases are treated.
The scientific community eagerly anticipates the outcome of this trial, as it could lead to significant advancements in the realm of gene therapy, redefining treatment options and improving the quality of life for patients facing Crigler-Najjar syndrome and potentially other genetic disorders.
Imlifidase is a unique enzyme therapy that targets and cleaves immunoglobulin G (IgG) antibodies, which are often a barrier to successful gene therapy. By administering imlifidase prior to the gene therapy GNT-0003, the trial aims to facilitate treatment in patients with severe forms of the disease who possess these objectionable antibodies.
According to Søren Tulstrup, the President and CEO of Hansa Biopharma, up to one in three individuals with anti-AAV antibodies are unable to benefit from gene therapies utilizing AAV vectors. This highlights the critical importance of their collaboration with Genethon, as it represents a pioneering effort to extend the benefits of gene therapy to a broader patient population.
The trial will involve three patients aged 18 years or older, who must meet specific criteria, including the presence of pre-formed antibodies against AAV serotype 8 (AAV8). Initially, participants will enter a three-month observational phase to monitor their condition before receiving the treatment regimen, which consists of a dose of imlifidase followed by GNT-0003.
Genethon's CEO, Frédéric Revah, emphasized the organization’s commitment to developing innovative strategies that widen access to gene therapies for patients facing rare diseases. Currently, patients with anti-AAV antibodies have limited options, and GNT-018-IDES represents a critical step forward, fueled by years of research dedicated to understanding and manipulating the immune response to AAV.
GNT-0003, the gene therapy under investigation, is already undergoing evaluation in pivotal trials, following promising results from earlier Phase 1-2 dose escalation studies that indicated both safety and efficacy. Additionally, it has received PRIME (PRIority MEdicine) designation from the European Medicines Agency (EMA), signifying its potential to address unmet medical needs.
The implications of this trial are significant. Crigler-Najjar syndrome, a rare genetic disorder characterized by the inability of the liver to process bilirubin due to a deficiency in the UGT1A1 enzyme, can cause severe neurological damage and developmental issues if not treated promptly. Patients currently undergo invasive treatments, including phototherapy for extensive hours each day, to manage their bilirubin levels.
Currently classified as an ultra-rare condition affecting less than one individual per million each year, Crigler-Najjar poses substantial treatment challenges. Success in this trial could pave the way for the first gene therapy specifically targeted towards this debilitating syndrome.
Hansa Biopharma, noted for its innovative approaches to rare immunological conditions, has developed imlifidase to help facilitate kidney transplants in sensitized patients, unlocking a pathway to life-saving treatments where options were previously limited.
As the trial progresses, both Hansa Biopharma and Genethon aim to release data in 2025, which could mark a pivotal moment in both their missions to revolutionize the way rare genetic diseases are treated.
The scientific community eagerly anticipates the outcome of this trial, as it could lead to significant advancements in the realm of gene therapy, redefining treatment options and improving the quality of life for patients facing Crigler-Najjar syndrome and potentially other genetic disorders.
Topics Health）
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