GENETHON

Genethon, a non-profit gene therapy R&D organization, achieved significant milestones including positive Phase 1/2 results for GNT0004 in Duchene muscular dystrophy, approval for Phase 3 trials, a Phase 2 trial for imlifidase in partnership with Hansa Biopharma, and groundbreaking AI-designed capsids research published in Nature Communications.

Rocket Pharmaceuticals' AAV9 treatment shows good safety and efficacy in a 3-cohort trial. Updated efficacy data from phase 1/2 announced. Phase 2 to assess Hansa's imlifidase as pretreatment for Genethon's GNT-0003. Sana's SC291 CAR T-cell therapy targets lupus. Cartesian plans AURORA phase 3 trial in H1 next year.

The FDA investigates bluebird bio's Skysona over hematologic malignancies. REGENXBIO starts phase 3 AFFINITY DUCHENNE trial for RGX-202 in DMD. Genethon and Hansa Biopharma initiate phase 2 trial for Crigler–Najjar syndrome. Sana Biotechnology's SC291 gets fast track designation for SLE. Roche to acquire Poseida Therapeutics. Exegenesis Bio's EXG110 receives orphan drug designation for Fabry disease. NorthX Biologics partners with Amarna Therapeutics for Nimvec AM510.

Genethon and Hansa Biopharma have launched a phase 2 trial (GNT-018-IDES) evaluating imlifidase as a pretreatment for GNT-0003, an AAV8 vector-based gene therapy for severe Crigler-Najjar syndrome. The trial aims to treat patients with pre-existing antiAAV8 antibodies, with initial results expected next year. Imlifidase, which cleaves IgG antibodies, is intended to enable AAV8 gene therapy in patients previously ineligible due to preexisting antibodies.

Genethon and Hansa Biopharma launch phase 2 trial for Crigler-Najjar disease patients with anti-AAV antibodies, using gene therapy with imlifidase pre-treatment to overcome immune response challenges.

Hansa Biopharma and Genethon initiate Phase 2 trial GNT-018-IDES to evaluate efficacy and safety of gene therapy GNT-0003 in Crigler-Najjar syndrome patients with pre-existing anti-AAV antibodies, using imlifidase to potentially enable gene therapy in these patients.

Hansa Biopharma and Genethon initiate Phase 2 trial, GNT-018-IDES, to evaluate gene therapy GNT-0003 efficacy and safety in Crigler-Najjar syndrome patients with pre-existing anti-AAV antibodies, using imlifidase pre-treatment to potentially enable gene therapy in these patients.

Hansa Biopharma and Genethon initiate Phase 2 trial GNT-018-IDES, evaluating imlifidase as pre-treatment to gene therapy GNT-0003 in Crigler-Najjar syndrome patients with pre-existing AAV antibodies, aiming to expand gene therapy eligibility. The trial includes three patients aged ≥18 requiring phototherapy, with data expected in 2025.

GNT0004, a gene therapy for Duchenne muscular dystrophy (DMD), shows stable or improved motor function in a clinical trial. Genethon plans to expand the study to Phase 3 in 2025, aiming for regulatory approvals. GNT0004 delivers a microdystrophin gene to muscle cells, with higher dose patients showing stable motor function and reduced muscle damage markers.