Tag «APPROVALS 2021»

Lisocabtagene maraleucel

It’s only fair to share… Lisocabtagene maraleucel (liso-cel; JCAR017; Anti-CD19 CAR T-Cells) is an investigational chimeric antigen receptor (CAR) T-cell therapy designed to target CD19, [1][2] which is a surface glycoprotein expressed during normal B-cell development and maintained following malignant transformation of B cells. [3][4][5] Liso-cel CAR T-cells aim to target and CD-19 expressing cells through a CAR construct …

Casimersen

It’s only fair to share…   Casimersen カシメルセン; RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2’a→5′)(C-A-A-m5U-G-C-C-A-m5U-C-C-m5U-G-G-A-G-m5U-m5U-C-C-m5U-G), 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] Formula C268H424N124O95P22 CAS 1422958-19-7 Mol weight 7584.4307 FDA 2021/2/25 , Amondys 45, Antisense oligonucleotide Treatment of Duchenne muscular dystrophy Nucleic Acid Sequence Sequence Length: 224 a 7 c 5 g 6 umodified Exon-45: NG-12-0064 SRP-4045 WHO 10354 Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used …

Fosdenopterin hydrobromide

It’s only fair to share… Fosdenopterin hydrobromide FDA APPR 2021/2/26, NULIBRY BBP-870/ORGN001 a cyclic pyranopterin monophosphate (cPMP) substrate replacement therapy, for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A. ホスデノプテリン臭化水素酸塩水和物; Formula C10H14N5O8P. 2H2O. HBr CAS 2301083-34-9DIHYDRATE Mol weight 480.1631 2301083-34-9 (1R,10R,12S,17R)-5-amino-11,11,14-trihydroxy-14-oxo-13,15,18-trioxa-2,4,6,9-tetraza-14λ5-phosphatetracyclo[8.8.0.03,8.012,17]octadeca-3(8),4-dien-7-one;dihydrate;hydrobromide 1,3,2-DIOXAPHOSPHORINO(4′,5′:5,6)PYRANO(3,2-G)PTERIDIN-10(4H)-ONE, 8-AMINO-4A,5A,6,9,11,11A,12,12A-OCTAHYDRO-2,12,12-TRIHYDROXY-, 2-OXIDE, HYDROBROMIDE, HYDRATE (1:1:2), (4AR,5AR,11AR,12AS)- CYCLIC PYRANOPTERIN MONOPHOSPHATE MONOHYDROBROMIDE DIHYDRATE …

Melphalan flufenamide hydrochloride

It’s only fair to share… .HCl   Melphalan flufenamide hydrochloride メルファランフルフェナミド塩酸塩; L-Phenylalanine, 4-[bis(2-chloroethyl)amino]-L-phenylalanyl-4-fluoro-, ethyl ester, hydrochloride Formula C24H30Cl2FN3O3. HCl CAS 380449-54-7 Mol weight 534.8786 FDA APPROVED PEPAXTO, 2021/2/26 Efficacy Antineoplastic, Alkylating agent   Disease Multiple myeloma Ethyl (2S)-2-[(2S)-2-amino-3-{4-[bis(2-chloroethyl)amino]phenyl}propanamido]-3-(4-fluorophenyl)propanoate J 1 J 1 (prodrug) L-Melphalanyl-L-p-fluorophenylalanine ethyl ester Melflufen Melphalan flufenamide Pepaxto Prodrug J 1 Melflufen Molecular FormulaC24H30Cl2FN3O3 Average …

Evinacumab

It’s only fair to share… (Heavy chain) EVQLVESGGG VIQPGGSLRL SCAASGFTFD DYAMNWVRQG PGKGLEWVSA ISGDGGSTYY ADSVKGRFTI SRDNSKNSLY LQMNSLRAED TAFFYCAKDL RNTIFGVVIP DAFDIWGQGT MVTVSSASTK GPSVFPLAPC SRSTSESTAA LGCLVKDYFP EPVTVSWNSG ALTSGVHTFP AVLQSSGLYS LSSVVTVPSS SLGTKTYTCN VDHKPSNTKV DKRVESKYGP PCPPCPAPEF LGGPSVFLFP PKPKDTLMIS RTPEVTCVVV DVSQEDPEVQ FNWYVDGVEV HNAKTKPREE QFNSTYRVVS VLTVLHQDWL NGKEYKCKVS NKGLPSSIEK TISKAKGQPR EPQVYTLPPS QEEMTKNQVS LTCLVKGFYP SDIAVEWESN GQPENNYKTT PPVLDSDGSF FLYSRLTVDK SRWQEGNVFS CSVMHEALHN HYTQKSLSLS LGK (Light chain) …

Tozinameran, Pfizer–BioNTech COVID‑19 vaccine

It’s only fair to share… SEQUENCE 1gagaauaaac uaguauucuu cuggucccca cagacucaga gagaacccgc 51caccauguuc guguuccugg ugcugcugcc ucuggugucc agccagugug 101ugaaccugac caccagaaca cagcugccuc cagccuacac caacagcuuu 151accagaggcg uguacuaccc cgacaaggug uucagaucca gcgugcugca 201cucuacccag gaccuguucc ugccuuucuu cagcaacgug accugguucc 251acgccaucca cguguccggc accaauggca ccaagagauu cgacaacccc 301gugcugcccu ucaacgacgg gguguacuuu gccagcaccg agaaguccaa 351caucaucaga ggcuggaucu ucggcaccac acuggacagc aagacccaga 401gccugcugau cgugaacaac gccaccaacg uggucaucaa agugugcgag 451uuccaguucu gcaacgaccc cuuccugggc gucuacuacc …