Watch this expert-led session to explore the latest trends and insights in gene therapy, including how to model immunosuppressive regimens, key study-design considerations, and the importance of age categories in nonhuman primate research.
Tag Archive for: gene therapy
uniqure Amsterdam-based UniQure NV’s stocks made a 240% after-hours jump at Nasdaq after the company announced its Huntington gene therapy AMT-130 met the endpoints of a Phase II study and it will apply for marketing authorisation next year. In addition, UniQure entered into a US$175m non-dilutive senior secured term loan facility with Hercules Capital and commenced a US$200m underwritten public offering of its ordinary shares to prepare a BLA in Q1 2026.
Romi - adobe stock.comBelgian lysosomal storage disease specialist Azafaros BV has added a new lead investor to its portfolio. In addition to €25m Series A leader Forbion, Jeito Captial co-led a €132m Series B round aimed at advancing two pivotal trials of Azafaros‘ lead compound nizabagloustat (AZ-3102).
adobe stock - aliaksandrBritish cell and gene therapy CDMO eXmoor Pharma plc, and KU Leuven which has just launched a translational cell and gene therapy hub in Belgium have inked a strategic collaboration supporting the hub’s work in four areas.
Dr. Dong-Jiunn Jeffery TruongScientists at German Helmholtz Centre Munich have presented a new transport system for gene and cell therapy that overcomes the limitations of viral vectors. An in vivo gene therapy for hereditary blindness corrected the defect in the Rpe65 gene ten times more efficiently than was possible with viral vectors.
uniqure NVAmsterdam-based uniQure NV’s gene therapy candidate AMT-130 has been granted FDA Breakthrough Therapy Designation for the treatment of the neurodegenerative Huntington’s disease.
SpliceBioSpanish SpliceBio has dosed the first patient with SB-007, the first dual AAV gene therapy in clinical development for Stargardt disease
Fuse VectorsFuse Vectors has secured US$5.2m in pre-seed financing to revolutionise gene therapy manufacturing with its cell-free viral vector technology. The Danish company is aiming to make treatments more accessible and cost-effective by streamlining production.
XyloCor TherapeuticsA US$67.5m Series B financing in XyloCor Therapeutics led by French Jeito Capital will fund two double-blind Phase 2 trials for XC001, a pioneering gene therapy for severe cardiovascular disease.
CertestCircio Holding ASA has inked a collaboration with Certest Biotec, SL to find and test circVec LNP-formulations to deliver its circular RNA therapeutics to target tissues for gene therapies.