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.
Tag Archive for: gene therapy
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.
Image by freepikPurespring 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.
Astellas Pharma IncAviadoBio 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.
Evotec SEEvotec SE is widening its 2022 R&D collaboration SynaptixBio Ltdi to optimise and identify new antisense oligonucleotides to treat children with H-ABC leukodystrophy, a rare genetic myelin synthesis defect that shows similar symptoms such as multiple sclerosis.
AI specialist WhiteLab Genomics, the Vision Institute, and IT expert ADLIN Science, have entered into a strategic partnership aimed at developing new AAV vectors targetting retinal dystrophies.
Basel-headquartered Roche AG has inaugurated a brand new €90m gene therapy development centre in Penzberg near Munich.