Table 2.
Ex vivo gene therapies for inherited neurometabolic disorders.
| Conditions | Clinical trial | Interventions | Sponsor/Collaborators | Phase | Enrollment | Status |
|---|---|---|---|---|---|---|
| X-ALD/CALD | NCT02559830 | Autologous HSCs transduced with lenti-ABCD1 | Shenzhen Second People's Hospital | I/II | 50 | Recruiting |
| NCT03852498 | Lenti-DTM* | Bluebird bio | III | 35 | Active, not recruiting | |
| NCT01896102 | Lenti-DTM | Bluebird bio | II/III | 32 | Completed | |
| MLD | NCT01560182 | OTL-200 | Orchard Therapeutics| | I/II | 20 | Active, not recruiting |
| NCT03725670 | Autologous HSCs transduced with lenti-ARSA | Shenzhen Geno-Immune Medical Institute | Not Applicable | 10 | Unknown status | |
| NCT02559830 | Autologous HSCs transduced with lenti-ARSA | Shenzhen Second People's Hospital | I/II | 50 | Recruiting | |
| NCT04283227 | OTL-200 | Orchard Therapeutics | III | 6 | Recruiting | |
| NCT03392987 | OTL-200 | Orchard Therapeutics | II | 10 | Active, not recruiting | |
| MPSI | NCT03488394 | OTL-203 | Orchard Therapeutics | I/II | 8 | Active, not recruiting |
| MPS IIIA | NCT04201405 | Autologous HSCs transduced lenti-SGSH | University of Manchester| Orchard Therapeutics |
I/II | 5 | Active, not recruiting |
| Fabry disease | NCT03454893 | AVR-RD-01 | AVROBIO | I/II | 11 | Terminated |
| NCT02800070 | Autologous HSCs transduced with lenti-GLA | University Health Network, Toronto|Ozmosis | I | 5 | Active, not recruiting | |
| Gaucher's disease | NCT00001234 | Autologous HSCs transduced with γ-retro-GBA | NINDS|NIH-Clinical Center | I | 120 | Completed |
| NCT04145037 | AVR-RD-02 | AVROBIO | I/II | 16 | Recruiting |
*Breakthrough Therapy Designation granted by FDA in May 2018.
X-ALD: X-linked adrenoleukodystrophy; CALD: cerebral adrenoleukodystrophy; ABCD1: ATP binding cassette subfamily D member 1; Lenti-DTM:: Autologous HSCs transduced with lenti-ABCD1; MLD: metachromatic leukodystrophy; ARSA: arylsulfatase A; OTL-200: Autologous HSCs transduced with lenti-ARSA; MPSI: mucopolysaccharidosis Type I; OTL-203: Autologous HSCs transduced with lenti-IDUA; IDUA: alpha-L-iduronidase; MPS IIIA: mucopolysaccharidosis Type IIIA; SGSH: N-sulfoglucosamine sulfohydrolase; GLA: Alpha-galactosidase A; AVR-RD-01: Autologous HSCs transduced with lenti-GLA; GBA: glucosylceramidase beta; AVR-RD-02: Autologous HSCs transduced with lenti-GBA; NINDS: National Institute of Neurological Disorders and Stroke.