Therapeutic Asset Ratings

464 results matching 1 filter

129,767 assets · 8,707 sponsors · 3,128 indications

Methodology v0.2

Asset	Sponsor	Phase	Rating
lx1001 Alzheimer Disease	Lexeo Therapeutics	P1/2	CCC (49)
g207 Neoplasm	Pediatric Brain Tumor Consortium	P2	CCC (48)
aav9 bbp-812 Canavan Disease	Aspa Therapeutics	P1/2	CCC (48)
aav9-hsyn1-migrik2 Temporal Lobe Epilepsy	UniQure Biopharma B.V.	P1/2	CCC (48)
Aav Bbp-631 Congenital Adrenal Hyperplasia	Adrenas Therapeutics Inc	P1/2	CCC (48)
Alnugranogene Aldeparvovec Frontotemporal Dementia And/Or Amyotrophic Lateral Sclerosis 1	Passage Bio, Inc.	P1/2	CCC (48)
Aproart-Cd34 Severe Combined Immunodeficiency	University of California, San Francisco	P1/2	CCC (48)
Autologous Cd34+ Hscs Transduced Ex Vivo With Cd11B Lv Encoding Human Ids Tagged With Apoeii Mucopolysaccharidosis Type 2	University of Manchester	P1/2	CCC (48)
Avr-Rd-02 Gaucher Disease	John Bernat	P1/2	CCC (48)
Biottt001 Neoplastic Meningitis	Henan Cancer Hospital	P1/2	CCC (48)
Bmn 331 HAE	BioMarin Pharmaceutical	P1/2	CCC (48)
Capsomere, recombinant human adeno-associated virus AAV3B viral capsid protein VP1-VP2-VP3 complex (1:2:7), hexacontamer, complex with DNA (recombinant replication-defective adeno-associated virus serotype 3B vector VTX-801 liver-specific human ?-1 antitrypsin promoter plus human 57-486-copper transporter ATP7B codon-optimized gene plus rabbit ?-globin polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks) (1:1) Wilson Disease	Vivet Therapeutics SAS	P1/2	CCC (48)
db107-rrv Glioblastoma	University of California, San Francisco	P1/2	CCC (48)
DEVAFIDUGENE CIVAPARVOVEC Mucopolysaccharidosis Type 1	Sangamo Therapeutics	P1/2	CCC (48)
DNA (recombinant adeno-associated virus vector AAV-Spark100-GAA human liver-specific expression cassette including human lysosomal ?-glucosidase codon-optimized gene GAA plus adeno-associated virus 2 inverted terminal repeat flanks) Lysosomal Storage Disease	Spark Therapeutics, Inc.	P1/2	CCC (48)
gc101 Spinal Muscular Atrophy, Type II	GeneCradle Inc	P1/2	CCC (48)
gc301 Glycogen Storage Disease Due To Acid Maltase Deficiency, Infantile Onset	GeneCradle Inc	P1/2	CCC (48)
High Dose Lx2006 Extrinsic Cardiomyopathy	Lexeo Therapeutics	P1/2	CCC (48)
Intrathalamic Avb-101 Frontotemporal Dementia	AviadoBio Ltd	P1/2	CCC (48)
Lentivirus Vector Rhiv7-Shi-Tar-Ccr5Rz-Transduced Hematopoietic Stem/Progenitor Cells Hodgkins Lymphoma	AIDS Malignancy Consortium	P1/2	CCC (48)
Lentivirus Vector Rhiv7-Shi-Tar-Ccr5Rz-Transduced Hematopoietic Stem/Progenitor Cells Follicular Lymphoma	AIDS Malignancy Consortium	P1/2	CCC (48)
Lentivirus Vector Rhiv7-Shi-Tar-Ccr5Rz-Transduced Hematopoietic Stem/Progenitor Cells Mantle Cell Lymphoma	AIDS Malignancy Consortium	P1/2	CCC (48)
Low Dose Lx2006 Extrinsic Cardiomyopathy	Lexeo Therapeutics	P1/2	CCC (48)
Oncolytic Measles Virus Encoding Thyroidal Sodium Iodide Symporter Malignant Ovarian Brenner Tumor	Mayo Clinic	P1/2	CCC (48)
Pbgene-Dmd Duchenne Muscular Dystrophy	Precision BioSciences, Inc.	P1/2	CCC (48)

Showing 151 to 175 of 464 assets

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