#Sweden #Lund #Hansa Biopharma #Genethon #imlifidase

Introduction to Gene Therapy Innovations

In an important advancement in gene therapy, Genethon and Hansa Biopharma recently announced the start of a Phase 2 clinical trial targeting Crigler-Najjar syndrome using a groundbreaking approach. This collaboration aims to leverage Hansa's innovative treatment, imlifidase, as a pre-treatment for patients facing barriers to accessing essential gene therapy due to pre-existing antibodies.

Understanding Crigler-Najjar Syndrome

Crigler-Najjar syndrome is a rare genetic liver disease that leads to dangerously high levels of bilirubin in the blood, potentially causing severe neurological damage. The disease stems from a deficiency in the UGT1A1 enzyme, which plays a critical role in processing bilirubin. Patients often endure grueling phototherapy treatments for up to 12 hours daily to keep their bilirubin levels in check, highlighting the dire need for effective therapeutic alternatives.

The Role of Imlifidase

Imlifidase is an antibody-cleaving enzyme derived from Streptococcus pyogenes. It selectively targets and cleaves IgG antibodies, thereby mitigating the immune response that can hinder gene therapy effectiveness. In clinical settings, approximately one in three patients possess antibodies against adeno-associated virus (AAV) vectors, making them ineligible for many gene therapies. The introduction of imlifidase hopes to address this challenge head-on.

The Phase 2 Trial Overview

The clinical trial, known as GNT-018-IDES, will involve a small cohort of three patients aged 18 and older who suffer from severe Crigler-Najjar syndrome and exhibit pre-existing anti-AAV8 antibodies. The trial is designed as a single-arm study, starting with a three-month observational period to assess the patients' conditions. Following this phase, the patients will receive imlifidase prior to being administered GNT-0003, the gene therapy developed by Genethon.

Objectives and Expectations

This collaboration signifies a crucial step toward expanding the accessibility of gene therapies. According to Søren Tulstrup, President and CEO of Hansa Biopharma, the partnership with Genethon marks a pivotal moment in making these life-saving treatments available to a larger group of patients, particularly those previously excluded due to their immune responses. Frédéric Revah, CEO of Genethon, emphasized that this trial reflects their commitment to innovative strategies designed to broaden patient access to groundbreaking gene therapies.

Future Implications

The key focus of the trial will be to evaluate the efficacy and safety of combining imlifidase treatment with GNT-0003, which has already demonstrated positive results in earlier studies and was granted PRIME status by the European Medicines Agency. If successful, GNT-0003 has the potential to become the first-ever gene therapy approved for Crigler-Najjar syndrome, potentially transforming the lives of affected patients, who currently face limited treatment options.

Conclusion

As the trial progresses, stakeholders eagerly await data expected to be released in 2025. These advancements are not just a win for the companies involved, but for the future of gene therapy and the patients whose lives may be radically improved as a result. With continued innovation and collaboration in this field, the horizon looks promising for many individuals battling rare genetic diseases.