Therapeutics
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| Name | Synonyms | FDA Status | Company | Target Type | Therapy Type | Approved For |
|---|---|---|---|---|---|---|
| AAV-GAD | AAV-GAD2, NLX-P101 |
Parkinson's Disease (Phase 1/2) | MeiraGTx Limited | Other Neurotransmitters | DNA/RNA-based | |
| AAV2-BDNF | MCI due to AD (Phase 1), Alzheimer's Disease (Phase 1) | Other | DNA/RNA-based | |||
| ALN-APP | Alzheimer's Disease (Phase 1) | Alnylam Pharmaceuticals, Inc., Regeneron Pharmaceuticals Inc. | Amyloid-Related | DNA/RNA-based | ||
| AVB-101 | Frontotemporal Dementia (Phase 1/2) | AviadoBio | Other | DNA/RNA-based | ||
| BIIB078 | BIIB-078, IONIS-C9Rx |
Amyotrophic Lateral Sclerosis (Discontinued) | Biogen, IONIS Pharmaceuticals | Other | DNA/RNA-based | |
| BIIB080 | IONIS-MAPTRx, ISIS 814907 |
Alzheimer's Disease (Phase 1) | Biogen, IONIS Pharmaceuticals | Tau | DNA/RNA-based | |
| BIIB094 | ION859, IONIS-BIIB7Rx |
Parkinson's Disease (Phase 1) | Biogen, IONIS Pharmaceuticals | Inflammation, Other | DNA/RNA-based | |
| BIIB105 | ION541 | Amyotrophic Lateral Sclerosis (Discontinued) | Biogen, IONIS Pharmaceuticals | Other | DNA/RNA-based | |
| CERE-110 | Nerve Growth Factor gene therapy | Alzheimer's Disease (Discontinued) | Sangamo BioSciences, Inc. | Other | DNA/RNA-based | |
| CpG 1018® | CpG oligodeoxynucleotides, CpG ODN |
Alzheimer's Disease (Phase 1) | Dynavax Technologies | Amyloid-Related, Inflammation | Immunotherapy (active), DNA/RNA-based | |
| Engensis | VM202, pCK-HGF-X7, NL003, hepatocyte growth factor | Amyotrophic Lateral Sclerosis (Phase 2) | Helixmith | Other | DNA/RNA-based | |
| ION464 | BIIB101, IONIS-BIIB6Rx |
Multiple System Atrophy (Phase 1) | Biogen, IONIS Pharmaceuticals | alpha-synuclein | DNA/RNA-based | |
| LX1001 | AAVrh.10-APOE2, AAVrh.10hAPOE2 |
Alzheimer's Disease (Phase 1) | Lexeo Therapeutics | Amyloid-Related | DNA/RNA-based | |
| LY3884961 | PR001, PR001A |
Parkinson's Disease (Phase 1/2) | Eli Lilly & Co., Prevail Therapeutics | Other | DNA/RNA-based | |
| LY3884963 | PR006 | Frontotemporal Dementia (Phase 1/2) | Eli Lilly & Co., Prevail Therapeutics | Other | DNA/RNA-based | |
| NIO752 | Progressive Supranuclear Palsy (Phase 1), Alzheimer's Disease (Phase 1) | Novartis Pharmaceuticals Corporation | Tau | DNA/RNA-based | ||
| PBFT02 | Frontotemporal Dementia (Phase 1/2) | Passage Bio | Other | DNA/RNA-based | ||
| QALSODY™ | Tofersen, BIIB067, Ionis-SOD1Rx , ASO1 |
Amyotrophic Lateral Sclerosis (Approved) | Biogen, IONIS Pharmaceuticals | Other | DNA/RNA-based | ALS |
| QRL-201 | Amyotrophic Lateral Sclerosis (Phase 1) | QurAlis Corporation | Other | DNA/RNA-based | ||
| TPN-101 | OBP-601, BMS-986001, censavudine, festinavir, 4'-ethynyl stavudine, 4'-ethynyl-d4T |
ALS-FTD (Phase 2), Progressive Supranuclear Palsy (Phase 2) | Transposon Therapeutics | Inflammation | Small Molecule, DNA/RNA-based | |
| Ulefnersen | ION363, Jacifusen |
Amyotrophic Lateral Sclerosis (Phase 3) | IONIS Pharmaceuticals | Other | DNA/RNA-based | |
| VY-AADC | VY-AADC01, VY-AADC02 |
Parkinson's Disease (Discontinued) | Neurocrine Biosciences, Voyager Therapeutics | Other Neurotransmitters | DNA/RNA-based | |
| WVE-004 | Frontotemporal Dementia (Discontinued), Amyotrophic Lateral Sclerosis (Discontinued) | Wave Life Sciences USA, Inc. | Other | DNA/RNA-based |