Media Releases

U of T‑based Structural Genomics Consortium and CHDI Foundation announce open-access collaboration to discover new drug targets for Huntington’s disease

June 5, 2014

Toron­to, Cana­da & New York City, USA — The Struc­tur­al Genomics Con­sor­tium (SGC) and CHDI Foun­da­tion have entered into a unique open-access research col­lab­o­ra­tion to dis­cov­er and char­ac­ter­ize new drug tar­gets for Huntington’s dis­ease (HD) using struc­tur­al and chem­i­cal biol­o­gy. In this first part­ner­ship of its kind, SGC and CHDI have explic­it­ly agreed not to file for patents on any of the col­lab­o­ra­tive research and to make all reagents and knowl­edge avail­able with­out restric­tion to the wider research com­mu­ni­ty, includ­ing phar­ma­ceu­ti­cal, biotech, and aca­d­e­m­ic research groups.

The SGC is a pub­lic-pri­vate part­ner­ship accel­er­at­ing dis­cov­ery of new drugs through an open-access research approach, with par­tic­u­lar focus on neglect­ed areas of the human genome, cre­at­ing an open col­lab­o­ra­tive net­work of sci­en­tists in hun­dreds of uni­ver­si­ties and in nine major phar­ma­ceu­ti­cal com­pa­nies. The col­lab­o­ra­tion with CHDI—a not-for-prof­it drug devel­op­ment orga­ni­za­tion with a mis­sion to rapid­ly devel­op drugs that will slow the pro­gres­sion of HD—further focus­es this approach with­in a spe­cif­ic dis­ease domain.  As part of the col­lab­o­ra­tion, CHDI will sup­port sci­en­tists at the SGC to gen­er­ate research tools to solve pro­tein struc­tures of poten­tial drug tar­gets for HD.

“This is a pio­neer­ing move by CHDI, and a tem­plate for how patient-ori­en­tat­ed fun­ders can help the research com­mu­ni­ty devel­op new drugs,” said Aled Edwards, CEO of the SGC. “With this col­lab­o­ra­tion the SGC is now push­ing our open-access research to exploit spe­cif­ic chem­i­cal probes and define pro­tein struc­tures in a par­tic­u­lar dis­ease, in order to fur­ther clar­i­fy the under­ly­ing dis­ease biol­o­gy and aid drug dis­cov­er­ers in acad­e­mia, biotech and phar­ma with the high­est qual­i­ty infor­ma­tion about new drug tar­gets.”

The SGC will make any research tools devel­oped freely avail­able to the research com­mu­ni­ty imme­di­ate­ly and with­out restric­tion; work­ing with a dis­ease foun­da­tion will enable a tar­get­ed dis­sem­i­na­tion and rapid uptake of the devel­oped research tools with­in the spe­cif­ic dis­ease field.

“Ours is a knowl­edge-dri­ven indus­try. By pro­vid­ing open access to their dis­cov­er­ies, the SGC and CHDI are doing exact­ly what is need­ed to help us dis­cov­er and devel­op new med­i­cines,” said Tet­suyu­ki Maruya­ma, Head of Research at Take­da Phar­ma­ceu­ti­cals. “This tar­get­ed approach with­in a spe­cif­ic dis­ease domain will invite phar­ma­ceu­ti­cal sec­tor researchers to look more close­ly at Huntington’s dis­ease as a tractable neu­rode­gen­er­a­tive dis­or­der.”

Since no patent pro­tec­tion on the results of this col­lab­o­ra­tion will be sought, any inves­ti­ga­tor will have com­plete free­dom to oper­ate. As part of the col­lab­o­ra­tion CHDI will facil­i­tate the dis­sem­i­na­tion of SGC’s exist­ing nov­el inhibitors of epi­ge­net­ics path­ways into its net­work of sci­en­tists and clin­i­cians to uncov­er poten­tial new ther­a­peu­tic tar­gets for HD.

“We often refer to CHDI as a col­lab­o­ra­tive enabler with the aim of stim­u­lat­ing drug dis­cov­ery research in Huntington’s dis­ease,” said Robi Blu­men­stein, Pres­i­dent of CHDI Man­age­ment. “CHDI has long oper­at­ed under the prin­ci­ple that the under­ly­ing dis­ease biol­o­gy should be freely acces­si­ble in a pre-com­pet­i­tive man­ner. This col­lab­o­ra­tion with the SGC will expand that pre-com­pet­i­tive domain to include bio­log­i­cal struc­tures, chem­i­cal probes and asso­ci­at­ed research data. Our aim is to ensure the HD research com­mu­ni­ty has access to the best pos­si­ble reagents and tools and that find­ings are made avail­able rapid­ly and with the fewest restric­tions to attract as many researchers as pos­si­ble to work on HD. The ulti­mate goal is to get effec­tive drugs to patients in the short­est pos­si­ble time”.


For more infor­ma­tion, please con­tact:

Struc­tur­al Genomics Con­sor­tium:

Arij Al Chawaf, PhD
Strate­gic Alliances, Man­ag­er
MaRS Cen­tre, 101 Col­lege St.
7th floor South Tow­er
Toron­to, ON, M5G 1L7
: +1 (416) 854‑2745

CHDI Management/CHDI Foun­da­tion:

Simon Noble, PhD
Direc­tor, Sci­en­tif­ic Com­mu­ni­ca­tions
350 7th Avenue, Suite 601
New York, NY 10001
Tel: +1 (212) 660‑8112
Cell: +1 (718) 564‑4321

About Huntington’s dis­ease

Huntington’s dis­ease is an inher­it­ed neu­rode­gen­er­a­tive dis­or­der caused by a muta­tion in the hunt­ingtin gene. The defect caus­es a DNA sequence called a CAG repeat to occur many more times than nor­mal. Each child of a par­ent with a muta­tion in the hunt­ingtin gene has a 50% chance of inher­it­ing the muta­tion. As a result of car­ry­ing the muta­tion, an indi­vid­u­al’s brain cells degen­er­ate lead­ing to behav­ioral, cog­ni­tive, and motor impair­ments that, over the course of the dis­ease, sig­nif­i­cant­ly reduce the indi­vid­u­al’s qual­i­ty of life and ulti­mate­ly cause death with­in 15 to 25 years of overt symp­tom onset. There are cur­rent­ly no ther­a­peu­tics approved that slow the pro­gres­sion of Hunt­ing­ton’s dis­ease.

About the Struc­tur­al Genomics Con­sor­tium (SGC)

With active research facil­i­ties at the Uni­ver­si­ties of Toron­to and Oxford Uni­ver­si­ty, the not-for-prof­it orga­ni­za­tion sup­ports the dis­cov­ery of new med­i­cines by car­ry­ing out open-access research in struc­tur­al and chem­i­cal biol­o­gy. More than 200 researchers in acad­e­mia and in nine phar­ma­ceu­ti­cal com­pa­nies col­lab­o­rate with­in SGC to accom­plish these goals. The SGC is also fund­ed by the Cana­di­an Foun­da­tion for Inno­va­tion, Genome Cana­da, the Ontario Min­istry of Eco­nom­ic Devel­op­ment and Inno­va­tion, the Well­come Trust and nine phar­ma­ceu­ti­cal com­pa­nies. More infor­ma­tion is avail­able at

About CHDI Foun­da­tion, Inc.

CHDI Foun­da­tion, Inc. is a pri­vate­ly-fund­ed, not-for-prof­it, bio­med­ical research orga­ni­za­tion that is exclu­sive­ly ded­i­cat­ed to rapid­ly dis­cov­er­ing and devel­op­ing ther­a­pies that slow the pro­gres­sion of Huntington’s dis­ease (HD). As a col­lab­o­ra­tive enabler, CHDI seeks to bring the right part­ners togeth­er to iden­ti­fy and address crit­i­cal sci­en­tif­ic issues and move drug can­di­dates to clin­i­cal eval­u­a­tion as quick­ly as pos­si­ble. Our sci­en­tists work close­ly with a net­work of more than 600 researchers in aca­d­e­m­ic and indus­tri­al lab­o­ra­to­ries around the world in the pur­suit of these nov­el ther­a­pies, pro­vid­ing strate­gic sci­en­tif­ic direc­tion to ensure that our com­mon goals remain in focus.  More infor­ma­tion about CHDI can be found at