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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