Tag Archive for: gene therapy

Belgian 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).

British 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.

Scientists 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.

Amsterdam-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.

Spanish SpliceBio has dosed the first patient with SB-007, the first dual AAV gene therapy in clinical development for Stargardt disease

Fuse 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.

A 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.

Circio 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.

Purespring Therapeutics, a pioneering gene therapy company focused on transforming kidney disease treatment, has raised £80 (€96m) in an oversubscribed Series B round. Led by Sofinnova Partners, this funding also drew support from Gilde Healthcare, Forbion, British Patient Capital, and Syncona Limited.

AviadoBio Ltd has cashed in €30m upfront and €20m as an equity investment from Astellas Pharma Inc for signing a licence option agreement for its Phase I/II gene therapy programme AVR-01.