Key Takeaways
A high-dose of AMT-130 slowed Huntington’s disease progression by 75% over 36 months
Most side effects were related to surgery and resolved, researchers said
If approved, this would be the first genetic therapy for Huntington’s disease
kAmu#xsp*[ $6AE] ae[ a_ad Ww62=E9s2J }6HDX — p ?6H 86?6 E96C2AJ 92D D9@H? AC@>:D6 :? D=@H:?8 E96 AC@8C6DD:@? @7 wF?E:?8E@?’D 5:D62D6[ 244@C5:?8 E@ 62C=J EC:2= C6DF=ED C6=62D65 (65?6D52J]k^Am
kAmx? 2 !92D6 `^a DEF5J[ A2E:6?ED 8:G6? 2 9:89 5@D6 @7 k2 9C67lQ9EEADi^^HHH]F?:BFC6]4@>^Qm&?:"FC6’Dk^2m 6IA6C:>6?E2= E96C2AJ p|%\`b_ 6IA6C:6?465 2 fdT D=@H:?8 @7 5:D62D6 AC@8C6DD:@? 27E6C E9C66 J62CD[ E96 4@>A2?J D2:5] %96 E96C2AJ 2=D@ C65F465 =6G6=D @7 2 <6J >2C<6C @7 3C2:? 52>286 — ?6FC@7:=2>6?E =:89E AC@E6:? :? DA:?2= 7=F:5 — 3J 2? 2G6C286 @7 g]aT]k^Am
kAmQ%96D6 7:?5:?8D C6:?7@C46 @FC 4@?G:4E:@? E92E p|%\`b_ 92D E96 A@E6?E:2= E@ 7F?52>6?E2==J EC2?D7@C> E96 EC62E>6?E =2?5D42A6 7@C wF?E:?8E@?’D 5:D62D6[Q k2 9C67lQ9EEADi^^HHH]F?:BFC6]4@>^A:@?66CD\=6256CD^=6256CD9:A^23:\D223\H2=:5QmsC] (2=:5 p3:\$223k^2m[ 49:67 >65:42= @77:46C 2E &?:"FC6[ E@=5 k6>mr}}k^6>m]k^Am
kAm%96 EC:2=[ CF? :? A2CE?6CD9:A H:E9 &?:G6CD:EJ r@==686 {@?5@?[ :?4=F565 ah A2E:6?ED EC62E65 H:E9 6:E96C 2 9:89 @C =@H 5@D6 @7 p|%\`b_ 2?5 7@==@H65 7@C be >@?E9D]Â k^Am
kAm%96 EC62E>6?E H2D 56=:G6C65 DFC8:42==J 3J :?;64E:?8 E96 E96C2AJ 5:C64E=J :?E@ E96 3C2:?’D DEC:2EF>[ 2? 2C62 >@DE 27764E65 3J wF?E:?8E@?’D]k^Am
kAm|@DE D:56 67764ED H6C6 =:?<65 E@ E96 DFC8:42= AC@465FC6 2?5 C6D@=G65[ E96 4@>A2?J C6A@CE65]Â k^Am
kAm%96 7:?5:?8D 92G6 ?@E J6E 366? A66C\C6G:6H65 @C AF3=:D965 :? 2 >65:42= ;@FC?2= 2?5 D9@F=5 36 C682C565 2D AC6=:>:?2CJ]k^Am
kAmwF?E:?8E@?’D 5:D62D6 :D 2 C2C6 86?6E:4 4@?5:E:@? >@DE 4@>>@? 2>@?8 A6@A=6 @7 tFC@A62? 2?46DECJ]k^Am
kAmxE 52>286D ?6CG6 46==D :? E96 3C2:?[ =625:?8 E@ >@G6>6?E AC@3=6>D[ 4@8?:E:G6 564=:?6 2?5 492?86D :? >@@5 2?5 3692G:@C] %96C6 :D 4FCC6?E=J ?@ 4FC6[ 3FE EC62E>6?ED 42? >2?286 D@>6 DJ>AE@>D]k^Am
kAmk2 9C67lQ9EEADi^^95D2]@C8^23@FE\95D2^DE277^Qmp>J vC2Jk^2m @7 E96 wF?E:?8E@?’D s:D62D6 $@4:6EJ @7 p>6C:42 Wws$pX C6>2:?65 42FE:@FD 3FE 9@A67F= 27E6C C6G:6H:?8 E96 DEF5J 7:?5:?8D]k^Am
kAm“u@C E96 2AAC@I:>2E6 ca[___ p>6C:42?D 2?5 E96:C 72>:=:6D =:G:?8 H:E9 wF?E:?8E@?’D 5:D62D6[ E9:D :D 2 ECF=J EC2?D7@C>2E:G6 56G6=@A>6?E[” vC2J[ H9@ H2D ?@E :?G@=G65 :? E96 C6D62C49[ D2:5 :? 2? 6>2:= E@ k6>mr}}]k^6>mk^Am
kAm“u@C 564256D[ E96C6 92G6 366? ?@ E96C2A:6D E@ D=@H 5:D62D6 AC@8C6DD:@? — @?=J EC62E>6?ED E@ >2?286 DJ>AE@>D[” D96 25565] “(9:=6 E96C6 :D DE:== 2 A2E9 E@ 2AAC@G2= E9C@F89 E96 usp[ E9:D 52E2 3C:?8D FD 4=@D6C E92? 6G6C E@ 2 7FEFC6 H96C6 H6 42? 492?86 E96 4@FCD6 @7 wF?E:?8E@?’D 5:D62D6]”k^Am
kAm&?:"FC6 D2:5 :E A=2?D E@ DF3>:E :ED 7:?5:?8D E@ E96 &]$] u@@5 2?5 sCF8 p5>:?:DEC2E:@? WuspX :? 62C=J a_ae] x7 2AAC@G65[ p|%\`b_ 4@F=5 364@>6 E96 7:CDE EC62E>6?E 7@C wF?E:?8E@?’D]k^Am
kAm~FED:56 6IA6CED D2:5 >@C6 C6D62C49 :D ?66565[ ?@E:?8 E96 EC:2= H2D D>2== 2?5 C6=:65 A2CE=J @? 6I:DE:?8 A2E:6?E 52E2 C2E96C E92? 2 7F== A=2463@\4@?EC@==65 8C@FA]k^Am
kAm$E:==[ k2 9C67lQ9EEADi^^HHH]4:>C]42>]24]F<^DE277^AC@76DD@C\52G:5\CF3:?DKE6:?\7>65D4:\7CDQms2G:5 #F3:?DKE6:?k^2m @7 E96 r2>3C:586 x?DE:EFE6 7@C |65:42= #6D62C49 42==65 E96 C6DF=ED “G6CJ AC@>:D:?8” 2?5 “6I4:E:?8[Q k6>mr}}k^6>m C6A@CE65]k^Am
kAmkDEC@?8m|@C6 :?7@C>2E:@?k^DEC@?8mk^Am
kAm%96 |2J@ r=:?:4 92D >@C6 @? k2 9C67lQ9EEADi^^HHH]>2J@4=:?:4]@C8^5:D62D6D\4@?5:E:@?D^9F?E:?8E@?D\5:D62D6^DJ>AE@>D\42FD6D^DJ4\a_bde``fQmwF?E:?8E@?VDk^2m]k^Am
kAm$~&#rti k6>mr}}k^6>m[ $6AE] ac[ a_adk^Am
k9amkDEC@?8m(92E %9:D |62?D u@C *@Fk^DEC@?8mk^9am
kAm%96C6 :D DE:== ?@ 4FC6 7@C wF?E:?8E@?’D 5:D62D6[ 3FE 62C=J C6DF=ED DF886DE 86?6 E96C2AJ 4@F=5 @?6 52J 36 2 EC62E>6?E]k^Am