View source for 20250205272. Modified Rna Age (ALNYLAM PHARMACEUTICALS, .)

= MODIFIED RNA AGENTS WITH REDUCED OFF-TARGET EFFECT =

'''Abstract''':
one aspect of the present invention relates to double-stranded rna (dsrna) agent capable of inhibiting the expression of a target gene. the antisense strand of the dsrna molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsrna comprises at least four 2′-fluoro modifications, and the sense strand of the dsrna molecule comprises ligand, wherein the ligand is an asgpr ligand. other aspects of the invention relate to pharmaceutical compositions comprising these dsrna molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsrna molecules, e.g., for the treatment of various disease conditions.

'''Inventor(s)''': [[:Category:Mark K. SCHLEGEL of Cambridge MA US|Mark K. SCHLEGEL]], [[:Category:Maja JANAS of Cambridge MA US|Maja JANAS]], [[:Category:Vasant R. JADHAV of Cambridge MA US|Vasant R. JADHAV]], [[:Category:Muthiah MANOHARAN of Cambridge MA US|Muthiah MANOHARAN]], [[:Category:Kallanthottathil G. RAJEEV of Cambridge MA US|Kallanthottathil G. RAJEEV]], [[:Category:Muthusamy JAYARAMAN of Cambridge MA US|Muthusamy JAYARAMAN]], [[:Category:Alexander V. KEL'IN of Cambridge MA US|Alexander V. KEL'IN]], [[:Category:Shigeo MATSUDA of Cambridge MA US|Shigeo MATSUDA]], [[:Category:Klaus CHARISSE of Cambridge MA US|Klaus CHARISSE]], [[:Category:Jayaprakash K. NAIR of Cambridge MA US|Jayaprakash K. NAIR]], [[:Category:Martin A. MAIER of Cambridge MA US|Martin A. MAIER]], [[:Category:Alfica SEHGAL of Cambridge MA US|Alfica SEHGAL]], [[:Category:Christopher BROWN of Cambridge MA US|Christopher BROWN]], [[:Category:Christopher THEILE of Cambridge MA US|Christopher THEILE]], [[:Category:Stuart MILSTEIN of Cambridge MA US|Stuart MILSTEIN]]

'''CPC Classification''': [[:Category:CPC_A61K31/713|A61K31/713]] (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES  (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))


[[Category:Patent Applications]]
[[Category:ALNYLAM PHARMACEUTICALS, INC.]]
[[Category:CPC_A61K31/713]]
[[Category:Mark K. SCHLEGEL of Cambridge MA US]]
[[Category:Maja JANAS of Cambridge MA US]]
[[Category:Vasant R. JADHAV of Cambridge MA US]]
[[Category:Muthiah MANOHARAN of Cambridge MA US]]
[[Category:Kallanthottathil G. RAJEEV of Cambridge MA US]]
[[Category:Muthusamy JAYARAMAN of Cambridge MA US]]
[[Category:Alexander V. KEL'IN of Cambridge MA US]]
[[Category:Shigeo MATSUDA of Cambridge MA US]]
[[Category:Klaus CHARISSE of Cambridge MA US]]
[[Category:Jayaprakash K. NAIR of Cambridge MA US]]
[[Category:Martin A. MAIER of Cambridge MA US]]
[[Category:Alfica SEHGAL of Cambridge MA US]]
[[Category:Christopher BROWN of Cambridge MA US]]
[[Category:Christopher THEILE of Cambridge MA US]]
[[Category:Stuart MILSTEIN of Cambridge MA US]]

[https://wikipatents.org/index.php?search=20250205272+rejection&title=Special%3ASearch&wprov=acrw1_-1 Search for rejections for patent application number 20250205272]


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