Full Press Release Details
Gene therapy vectors that deliver biologic drugs enter late-state clinical trials
Gene therapy can be more than a niche solution to a rare genetic disease. That’s the belief of a set of gene therapy developers repositioning the modality as a vehicle for once-and-done administration of an array of biologic drugs. The clinical data are promising. The next hurdles will be gaining patient, physician and payer buy-in.
All gene therapies marketed in the U.S. align with the initial vision for the modality, which centered on treating genetic diseases by delivering a functional, or even optimized, version of a patient’s mutated gene. That straightforward biological rationale — replace a broken gene with a good one — combined with the high risk tolerances of patients with severely debilitating rare diseases, made genetic disorders a natural fit for the new modality. ...
BCIQ Company Profiles
4d Molecular Therapeutics Inc.
Adverum Biotechnologies Inc.
Meiragtx Holdings plc
Vectory Therapeutics B.V.
Voyager Therapeutics Inc.
BCIQ Target Profiles
Adenosine deaminase RNA-specific B1 (ADARB1) (ADAR2)
Apolipoprotein epsilon 2 (APOE2)
Cytochrome P450 family 46 subfamily A polypeptide 1 (CYP46A1) (CH24H)
Glial cell-derived neurotrophic factor (GDNF)
Glucosylceramidase beta (GBA) (GBA1) (GCase)
Glutamic acid decarboxylase (GAD)
Vascular endothelial growth factor (VEGF)
BCIQ Company Profiles
4d Molecular Therapeutics Inc.
Adverum Biotechnologies Inc.
Meiragtx Holdings plc
Vectory Therapeutics B.V.
Voyager Therapeutics Inc.
BCIQ Target Profiles
Adenosine deaminase RNA-specific B1 (ADARB1) (ADAR2)
Apolipoprotein epsilon 2 (APOE2)
Cytochrome P450 family 46 subfamily A polypeptide 1 (CYP46A1) (CH24H)
Glial cell-derived neurotrophic factor (GDNF)
Glucosylceramidase beta (GBA) (GBA1) (GCase)
Glutamic acid decarboxylase (GAD)
Vascular endothelial growth factor (VEGF)