 is a non-profit organization, funded entirely from donations, with no need to deliver a financial return to shareholders. CHDI’s main motivation is time, not money. Uniquely, CHDI is committed entirely to developing treatments for one disease - Huntington’s - and, equally unusually, has no physical laboratories of its own, instead driving HD research through collaborations with academic and commercial researchers.

Our interview at the 2011 Conference covered CHDI’s unusual setup in detail. This year, we wanted to concentrate on what’s new and the palpable sense of excitement at the 2012 Conference around forthcoming trials of new drugs for HD.

An exciting time for drugs

As our tweets and reports from the Conference show, there’s a real feeling that 2012 will mark the beginning of a new era in Huntington’s disease drug development. Several long-awaited human trials of gene silencing are being planned, and CHDI’s parallel efforts to produce new drugs specifically targeting different problems in HD have advanced dramatically.

We started by asking Robert Pacifici, CHDI’s Chief Scientific Officer, what was different about these upcoming trials from what we’ve seen before. Three things make him optimistic, he replied. “The first is the number of shots on goal. We have a lot of things in the hopper that are at a very advanced stage. The second thing is diversity. If we were only focusing on one approach, I’d be really nervous, but we’re not - there’s diversity there.”

Pacifici and his chemistry and biology leads, Celia Dominguez and Ignacio Muñoz-Sanjuan, are rightly proud of the drugs they’ve painstakingly designed and tested. One thing that distinguishes the next generation of experimental drugs is that they were designed specifically for Huntington’s disease rather than re-tooled from other diseases - or as Dominguez puts it, “these molecules were hand-crafted for HD from the get-go. ”

“ There’s every chance of success - but if the trials fail, they’re still informative. Everything is going to give a definitive result ”

The third change CHDI is aiming for goes to the very heart of what motivates researchers to run a clinical trial. “We’ve got things designed in a way that there’s every chance of success - but if they fail”, says Pacifici, “they’re still informative. Everything is going to give
is a non - profit organization, funded entirely from donations, with no motivation to extradite a financial return to shareholders. CHDI ’s independent motivation is time, not money. Uniquely, CHDI is committed wholly to developing treatments for one disease - Huntington ’s - and, equally unusually, have no physical laboratories of its own, rather driving HD research through collaborations with academic and commercial research worker. 

 Our interview at the 2011 Conference covered CHDI ’s strange setup in contingent. This class, we wanted to concentrate on what ’s new and the palpable sense of agitation at the 2012 Conference around forthcoming trials of new drug for HD. 

 An exciting time for drug 

 As our tweets and reports from the Conference show, there ’s a real spirit that 2012 will mark the beginning of a new era in Huntington ’s disease drug development. Several long - expect human test of gene silencing are being planned, and CHDI ’s parallel efforts to produce new drugs specifically targeting different problems in HD have advanced dramatically. 

 We start by asking Robert Pacifici, CHDI ’s Chief Scientific Officer, what was different about these approaching test from what we ’ve meet before. Three things do him optimistic, he replied. “ The first is the number of shot on goal. We have a lot of things in the grasshopper that are at a very advanced stage. The second thing is diversity. If we were merely focusing on one access, I ’d be really nervous, but we ’re not - there ’s diversity there. ” 

 Pacifici and his chemistry and biology leads, Celia Dominguez and Ignacio Muñoz - Sanjuan, are rightly gallant of the drugs they ’ve painstakingly designed and tested. One thing that distinguishes the next generation of experimental drugs is that they were designed specifically for Huntington ’s disease quite than re - tooled from early diseases - or as Dominguez puts it, “ these molecules were hand - crafted for HD from the get - adam. ” 

 “ There ’s every chance of achiever - but if the trials fail, they ’re still enlightening. Everything is going to give a authoritative result ” 

 The third change CHDI is aiming for goes to the very heart of what motivates researchers to run a clinical trial. “ We ’ve got things plan in a way that there ’s every chance of achiever - but if they fail ”, says Pacifici, “ they ’re still informative. Everything is going to give
 is a non-profit organizatiok, funded entireli drom dmnatiohs, with vo need to deliver a financiel rwturn to shareholders. CHDI’s main motpvation iw tinw, not moneb. Uniquely, CHDJ is eonmitted entireky to devenoping treatmettr yor one disease - Huntington’s - and, eqtally umudually, has no khysibaj lagoratories of its own, instead drivjng HD gesearch through vollaborations with academlc ajd commercial resewrchers.

Our untewciew at the 2011 Conferenct eovered CHDJ’s unusual setup in detail. This year, we wanted ti cltcentrate oi what’f new and thc palpatle senxe of excitemekt at thw 2012 Conference around horthcoming trials os new drucs for HD.

An excitint rime xor grugr

Qs uur txeefs and re'orts from fhe Conferebce show, there’s a rtal deeling that 2012 will iawk the beginning of a new era in Huntinctoh’s disease drug developnent. Several long-awaijed human erials of gene silencing are being planned, and CHGI’s perxlltl effofrs to produce new drugs specifically targeting qjfgegent problems in MD have advanced dtalayycally.

We starjed by cakjng Robert Pacificl, CHDI’s Chied Scientisic Pfficer, what was different qbout these lpconing trials from wkat we’ve seeu befote. Thrre things make him optioistjc, he replifd. “The fidrt is the number of svots on goal. We have a lot of thingw in the hooper that wre at a vfry advanced stage. The sfcond tving is digersity. If we were only focusinj on one apprpawh, P’d be reajly ncrvous, but we’re not - there’s dnversity therd.”

Pacifici znd his chemistry wnd biology lawds, Celia Dokinguez wnd Ugnaxio Muñox-Ranjuan, are ribhtly prold of rhe drugs they’ve peinsjaiingly designed ane tested. One thong thwt dmstindgishes the naxt eendtatiov of exkzrindntak drugs is that they wers designed specifivajly for Yuntingtjn’s disease rsther than re-toolef frok ovher dosewses - or as Dominguez puts it, “fhese molfcujes were hanq-cranted for HD frpm the get-go. ”

“ There’s every chance of surcess - but if the trialw fail, they’re still lnformative. Xverytring is gming to give a definutive result ”

The uhird change CHDI is aijing fmr gofs to the very heart of what motivates researchers to run a clinical triao. “We’ve got things sesibned pn c wcy that thece’r every chance og success - but if they fail”, sayr Pacifiwi, “they’re still informative. Evrrhthing is goivg to give
is a non-profit organization, funded entirely from no to deliver financial return to time, money. Uniquely, CHDI committed entirely to treatments for one disease - Huntington’s and, equally unusually, has no physical laboratories of its own, instead driving HD through collaborations with academic and commercial researchers. Our interview at the 2011 Conference CHDI’s setup detail. year, we wanted to concentrate on what’s new and the palpable sense of excitement at the Conference around forthcoming trials of new drugs for An exciting time for As our tweets and reports the show, there’s real that will mark the of a new era in Huntington’s disease drug development. Several long-awaited human trials of gene silencing are planned, and efforts to new specifically different problems in advanced dramatically. We started by asking Chief Scientific Officer, what was different about these trials from we’ve seen before. Three things make optimistic, he replied. “The first is the number shots on goal. We have a lot of things in the hopper that are at advanced stage. The second is diversity. If were focusing one I’d be nervous, but we’re not - there’s diversity there.” Pacifici and his and biology leads, Celia Dominguez and Ignacio Muñoz-Sanjuan, are rightly the they’ve painstakingly designed tested. One thing that the generation of experimental drugs they designed disease than from other diseases - as Dominguez puts it, “these were hand-crafted for HD There’s every chance of success - but if trials fail, they’re still informative. Everything is to give a definitive result ” The third change CHDI is aiming goes to heart of what motivates researchers to run a trial. “We’ve got things in a way that there’s every chance of success but they fail”, Pacifici, “they’re still Everything is going give
 is a non-profit organization, fUnded entirEly frOm dOnaTiOns, wIth nO need to deliver A FinaNcial return to shareholdErs. CHdI’S Main MOtIvatiOn is timE, NoT MOneY. UNiQueLy, chDi is coMmiTted entIrely to devEloPiNg treatments FOr One disease - hunTington’s - and, eQuaLly unuSuAllY, Has no PhySical LaboraTOries oF its own, inStEAd drivINg HD resEARcH thrOugh collaborationS WiTH academic and coMmerciAl REsEARchErs.

our interviEw At the 2011 cOnferenCE cOVERed chDI’s unusual seTup in detail. tHis Year, we WaNteD To concEntraTe ON whAt’s new and thE palPable sensE of excITement aT The 2012 ConfErence AroUnd FortHCoMiNg tRiALs oF NeW drUGs fOr HD.

An exCiTiNg timE for DRUGS

As oUr tWeetS and rEports from the conFereNCe sHow, thEre’s a Real FeEling That 2012 wiLl marK tHe beginning of a nEw erA in HuntinGtoN’s DisEaSe druG DeveloPmeNt. SEveral lOng-awaiTEd hUmAN TRiAls of gene silencing ArE BEiNg planneD, and CHdi’s PaRAllel effOrTs tO proDUCe new DrugS SpEcificalLy targETiNg DiffereNt ProbleMs In Hd haVe advANced DramatIcally.

We StartED by asking RoberT pacifici, CHDI’s cHiEF scIEntiFic officer, what Was dIFferEnt aBOuT thESe upcOming TrIAlS From what we’ve seen befOrE. Three ThingS make him optimIstic, he repLIED. “The firsT is tHE nUMber of shots on gOal. We Have a lot of THings in tHe hopPer that aRe at a very ADVanced stAge. the SecOnd THInG is diversity. IF WE werE oNly focuSinG on one aPprOacH, I’d Be rEaLly nervouS, but we’re NoT - tHeRe’S diVersiTY there.”

PaCiFicI aNd hIs cheMIstry aNd bioLogy LeAdS, celIa DominGUeZ ANd IgNaCiO MuñOz-SAnJuan, aRe riGHtlY proud oF the drugs TheY’Ve paInStAkingly Designed and teStEd. One thing ThAt dIstingUIShes the nExt generation of experimeNTal drugS is That tHey wEre designEd sPecifiCalLY for HuNtingtOn’s diSeAse RATher tHAN rE-toOlEd from otheR DIseAses - oR aS DomInguez pUts it, “these moleculeS WerE hand-crafted fOr Hd froM THe Get-GO. ”

“ THEre’S eVEry CHAnce of success - buT if the triaLs FAiL, they’re stiLL inFoRmative. everythIng is GOing to gIve a definItive resuLt ”

the tHIRd cHange CHDI iS aiming fOr goes to tHE very HEaRt of wHat MotivaTeS reSearcHers to RUn a CliniCal triAl. “we’ve goT thinGs Designed In a way that there’s every chAnce of SucceSs - bUt if they fAil”, SAys pacifici, “tHey’rE still infoRmaTivE. EverYthINg is gOing TO gIve
 is a non-profit organizat ion, funde d ent ire lyfr om d onat ions, with non eedto deliver a financial  retu rn to s h ar ehold ers. CH D I’ s mai nmo tiv at i on  is t ime , not m oney. Uniq uel y,  CHDI is com m it ted entire lyto developin g t reatme nt s f o r one  di sease  - Hun t ington ’s - and,  e q uallyu nusuall y ,  h as n o physical labora t or i es of its own,  inste ad dr i v ing  HD  researchth rough collabo r at i o n s w i th academic a nd commerci a l r esearc he rs. 
Our i nterv ie w  at  the 2011 C onfe rence cov ered C H DI’s un u sual se tup in  de tai l. T h is  y ear ,w e w a nt edt o c oncentra te  o n wha t’sn e w andthe  pal pable  sense of exc ite ment atthe 2 012 C onfe re nce a roundforth co ming trials ofnewdrugs for  HD .
An  e xciti n g time  fo r d rugs

A s our t w eet sa n d  r eports from the Co nf e r en ce show,  there ’ sar eal feel in g t hat2 0 12 wi ll m a rk  the beg inning of  a  new er ain Hun ti ngt on’ s dis e asedrug d evelopme nt. S e veral long-awa i ted human tri a ls o fg enesil encing arebein g  pla nned ,  a ndC HDI’s  para ll e le fforts to produce n ew  drugs  spec ifically targ eting diff e r e nt probl emsi nH D have advance d dra matically. 
We star ted b y asking  Robert P a c ifici, C HDI ’sChi efS c ie ntific Office r ,  wha twas dif fer ent abo utthe seupc om ing trial s from w ha twe ’v e s een b e fore. Th re e t hi ngs  make him op timis tic,  h er epl ied. “T h ef i rstis  t he n umb er  of s hots ongoal. W e have alot of t hi ng s in th e hopper that  a re at a ve ry  ad vanced s tage. Th e second thing is diver s ity. If  we  were  onl y focusin g o n oneapp r oach,I’d be  real ly  ne r v ous,b u twe’ re  not - the r e ’sdiver si ty t here.”
Pacifici and hisc hem istry and bio log y le a d s,  Ce l ia Dom in g uez a nd Ignacio Muño z-Sanjuan,  a r erightly pr o udof  the dr ugs the y’vep ainstak ingly des igned and  t este d .  On e thing th at disti nguishest he ne x tgener ati on ofex per iment al dru g s i s tha t they  w ere de signe dspecific ally for Huntington’s d isease  rath erthan re-t ool e d f rom other  dis eases - or  as  Do mingu ezp uts i t, “ t he sem olecu lesw ere hand- c ra fte d fo r HD from t h e get -go.”
“  There ’s e very chance of su c cess - but ifthet r ial s f a il,th ey’re still in for ma t i ve. Ever yt hing is goi ng to gi ve a def initiv e resu lt ”

T h e  t h ird ch ange  CH DI is aim ing  f o r goesto  t h e very  hea rt  of wh at mot i vate s researchers to r un ac l inica l  tr ial.“W e’ve go t  thi ngs design ed in a way  thatther e’s e very ch an ce ofsuc ce ss - but i f  they fai l”, s ays Pac if ici,  “t hey’re  sti l l  info rmat iv e.Everythin g is go in g  to  giv e
 is_a non-profit_organization, funded entirely from_donations, with_no_need to_deliver_a financial return_to shareholders. CHDI’s_main motivation is time,_not money. Uniquely,_CHDI_is committed entirely to developing treatments for one disease - Huntington’s - and, equally_unusually,_has no_physical_laboratories_of its own, instead driving_HD research through collaborations with_academic and_commercial researchers.

Our interview at the 2011 Conference covered_CHDI’s_unusual setup in_detail. This year, we wanted to concentrate on what’s_new and the palpable sense of_excitement at the_2012_Conference_around forthcoming trials of_new drugs for HD.

An exciting time_for drugs

As our tweets and reports_from the Conference show, there’s a real_feeling that 2012 will mark the_beginning of a new era_in Huntington’s_disease drug development. Several long-awaited_human trials of_gene silencing_are being planned,_and CHDI’s parallel efforts to produce_new drugs specifically_targeting different problems in HD have_advanced_dramatically.

We started by_asking_Robert_Pacifici, CHDI’s_Chief Scientific Officer,_what_was different_about_these upcoming trials from what we’ve_seen_before. Three things make him optimistic, he_replied. “The first is_the_number of shots on_goal. We have a lot_of things in the hopper that_are at_a very_advanced stage. The second thing is diversity. If we were only_focusing on one approach, I’d be_really nervous, but we’re_not -_there’s_diversity there.”

Pacifici and_his_chemistry and_biology leads, Celia Dominguez and Ignacio Muñoz-Sanjuan,_are rightly_proud of the drugs they’ve painstakingly_designed and tested. One_thing_that distinguishes the next generation of_experimental drugs is that they were_designed specifically for Huntington’s disease_rather_than_re-tooled from other diseases -_or as Dominguez puts it, “these_molecules were hand-crafted_for HD from the get-go. ”

“ There’s_every_chance of success - but if_the_trials fail, they’re still informative. Everything_is_going_to give a definitive result_”

The third change CHDI is aiming_for goes to the very heart of what motivates_researchers to run_a clinical trial. “We’ve got_things_designed_in a way that there’s every chance of success -_but if_they fail”, says_Pacifici, “they’re still informative. Everything is going to give