Treatment type
Treatments and trials using Gene Therapy, with the same country and access zoom as New Developments.
All New Developments
Kebilidi (eladocagene exuparvovec-tneq) — one-time intraputaminal gene therapy for aromatic L-amino acid decarboxylase deficiency
FDA accelerated approval on 13 November 2024 (press 14 November 2024) for Kebilidi (eladocagene exuparvovec-tneq) suspension — an AAV…
This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic…
Zevaskyn (prademagene zamikeracel) — gene-modified cellular sheets surgically applied to treat wounds in recessive dystrophic epidermolysis bullosa
FDA approval on 28 April 2025 for Zevaskyn (prademagene zamikeracel) gene-modified cellular sheets — an autologous cell sheet-based gene therapy…
This is for people living with recessive dystrophic epidermolysis bullosa — and the caregivers who help them…
Papzimeos (zopapogene imadenovec-drba) — four subcutaneous shots over 12 weeks for adults with recurrent respiratory papillomatosis
FDA traditional/full approval on 14 August 2025 for Papzimeos (zopapogene imadenovec-drba) suspension — a non-replicating adenoviral vector…
This is for adults living with recurrent respiratory papillomatosis who still need repeated airway surgeries…
Waskyra (etuvetidigene autotemcel) — one-time gene-modified stem-cell therapy for Wiskott-Aldrich syndrome when no matched related donor is available
FDA approval on 9 December 2025 for Waskyra (etuvetidigene autotemcel) suspension — an autologous hematopoietic stem-cell gene therapy given as a…
This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a…
Fayuvi (rebisufligene etisparvovec-hopf) — one-time IV gene therapy for children with Sanfilippo syndrome type A (MPS IIIA)
FDA approval on 17 September 2026 for Fayuvi (rebisufligene etisparvovec-hopf) suspension — a one-time AAV9 gene therapy given by intravenous…
This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved…
KRESLADI (marnetegragene autotemcel) — one-time gene therapy for children with severe leukocyte adhesion deficiency type I when no matched sibling donor is available
FDA accelerated approval on 26 March 2026 for KRESLADI (marnetegragene autotemcel) suspension — an autologous hematopoietic stem cell-based gene…
This is for families of children with severe LAD-I from confirmed ITGB2 gene changes who do not have an…
GENGLYCOS (pariglasgene brecaparvovec-opnr) — one-time gene therapy to help lower daily cornstarch needs in glycogen storage disease type Ia (ages 8 and older)
FDA accelerated approval on 19 August 2026 for GENGLYCOS (pariglasgene brecaparvovec-opnr) suspension — a one-time AAV8 gene therapy given by…
This is for people 8 years and older with glycogen storage disease type Ia who still rely on carefully timed…
OTARMENI (lunsotogene parvec-cwha) — gene therapy for severe genetic hearing loss from biallelic OTOF variants
FDA accelerated approval on 23 April 2026 for OTARMENI (lunsotogene parvec-cwha) suspension — a one-time intracochlear gene therapy — for…
This is for children and adults with severe genetic hearing loss from confirmed OTOF gene changes who may be…