Table 5. Companies developing gene therapies and the diseases they treat.
| Company | Platform | Diseases treated |
|---|---|---|
| Spark Therapeutics | AAV-based gene therapy | rare forms of blindness, IRDs, such as RPE65-mediated IRDs (positive Phase III results), and choroideremia (Phase I/II on-going) |
| AveXis | AAV-based gene therapy | SMA (positive Phase I/II results) |
| AGTC | AAV-based gene therapy | rare ophthalmological disorders such as XLRS and XLRP (early clinical stages or IND-ready programs) |
| uniQure | AAV-based gene therapy | familial LPLD (Glybera® approved), hemophilia B (Phase I/II), Sanfilippo B (Phase I), and PD (Phase I) and other rare genetic diseases of the liver/metabolism, CNS, and cardiovascular system |
| Bluebird Bio | Lentivirus-based gene therapy | severe genetic disorders such as CALD (Phase II/II), transfusion-dependent β-thalassemia (also known as β-thalassemia major) (Phase II/II), and severe sickle cell disease (Phase I/II) |
| Regenxbio | AAV-based gene therapy | HoFH (Phase I/II trial); MPS type I & wet AMD (IND-ready) |
| Bamboo therapeutics | AAV-based gene therapy | rare genetic disorders such as GAN (Phase I/II on-going), DMD, and FA |
| Voyager Therapeutics | AAV-based gene therapy | rare CNS diseases, such as PD (Phase I/II on-going), ALS, and HD |
| Dimension Therapeutics | AAV-based gene therapy | rare genetic liver disorders, including hemophilia B (Phase I/II) |
| Ionis Pharmaceuticals | antisense-based therapy | HoFH (KYNAMRO® approved) & pouchitis (Alicaforsen approved) and a wide range of rare genetic diseases, including SMA (positive Phase III results) and HD (Phase II on-going) |
| Alnylam Pharmaceuticals | RNAi-based therapy | a wide range of rare genetic diseases, including hereditary amyloidosis ATTR (Phase III), hemophilia, and rare bleeding disorders (Phase II) |
AAV, adeno-associated virus; ALS, amyotrophic lateral sclerosis; AMD, age-related macular degeneration; ATTR, TTR-related amyloidosis; CALD, cerebral adrenoleukodystrophy; DMD, Duchenne's muscular dystrophy; FA, Friedreich's ataxia; GAN, giant axonal neuropathy; HD, Huntington's disease; HoFH, homozygous familial hypercholesterolemia; IRDs, inherited retinal dystrophies; LPLD, lipoprotein lipase deficiency; MPS, mucopolysaccharidosis; PD, Parkinson disease; SMA, spinal muscular atrophy; XLRP, x-linked retinitis pigmentosa; XLRS, x-linked retinoschisis.