Ionis Partners with Metagenomi to Add Gene Editing to its Broad Technology Platform

This col­lab­o­ra­tion is a strate­gic step to com­bine Metagenomi’s lead­ing gene edit­ing tool­box of diverse, spe­cif­ic and high­ly effi­cient nucle­as­es, with Ion­is’ pio­neer­ing exper­tise of RNA-tar­get­ed ther­a­peu­tics, to cat­alyze the next wave of in vivo gene edit­ing applications.”

Ion­is Phar­ma­ceu­ti­cals, Inc. (Nas­daq: IONS) and Metageno­mi today announced that the com­pa­nies have entered a col­lab­o­ra­tion that will lever­age Ion­is’ exten­sive exper­tise in RNA-tar­get­ed ther­a­peu­tics and Metagenomi’s ver­sa­tile next-gen­er­a­tion gene edit­ing sys­tems to pur­sue a mix of val­i­dat­ed and nov­el genet­ic tar­gets that have poten­tial to expand ther­a­peu­tic options for patients. The com­pa­nies will joint­ly con­duct research aimed ini­tial­ly at deliv­er­ing inves­ti­ga­tion­al med­i­cines for up to four genet­ic tar­gets. Ion­is has the right to add four more tar­gets upon achieve­ment of pre-deter­mined devel­op­ment milestones.

Ion­is was found­ed over 30 years ago to dis­cov­er and devel­op nov­el, high­ly per­son­al­ized med­i­cines using our pow­er­ful RNA-tar­get­ing tech­nol­o­gy plat­form. This part­ner­ship with Metageno­mi sup­ports Ion­is’ strate­gic objec­tive to advance our tech­nol­o­gy and expand our capa­bil­i­ties to deliv­er pre­ci­sion genet­ic med­i­cines. Togeth­er, we can broad­en the appli­ca­tion of gene edit­ing by lever­ag­ing Ion­is’ vast expe­ri­ence in nucle­ic acid ther­a­peu­tics to opti­mize and extend the reach of gene edit­ing for liv­er tar­gets and to new tis­sues,” said Ion­is Chief Exec­u­tive Offi­cer Brett P. Monia, Ph.D. This alliance brings our two com­pa­nies clos­er to deliv­er­ing next-gen­er­a­tion ther­a­pies with the poten­tial to rev­o­lu­tion­ize the treat­ment of diseases.”

Gene edit­ing has the poten­tial to trans­form chron­ic ther­a­pies into poten­tial­ly cura­tive treat­ments for patients who cur­rent­ly have lim­it­ed options,” said Bri­an C. Thomas, Ph.D., chief exec­u­tive offi­cer and founder of Metageno­mi. This col­lab­o­ra­tion is a strate­gic step to com­bine Metagenomi’s lead­ing gene edit­ing tool­box of diverse, spe­cif­ic and high­ly effi­cient nucle­as­es, with Ion­is’ pio­neer­ing exper­tise of RNA-tar­get­ed ther­a­peu­tics, to cat­alyze the next wave of in vivo gene edit­ing appli­ca­tions. We con­tin­ue to fol­low our strat­e­gy of part­ner­ing with the most expe­ri­enced teams in the genet­ic med­i­cines field that will allow us to trans­form the lives of patients.”

Under the terms of the agree­ment, Ion­is will pay $80 mil­lion upfront to Metageno­mi plus the poten­tial for future mile­stone pay­ments and roy­al­ties. Wells Far­go Secu­ri­ties act­ed as finan­cial advi­sor to Ion­is on the transaction.


Ion­is will con­duct a web­cast on Nov. 14 at 8 a.m. East­ern Time to dis­cuss this announce­ment and relat­ed activ­i­ties. Inter­est­ed par­ties may access the web­cast here. A web­cast replay will be avail­able for a lim­it­ed time at the same address.

CRISPR Gene Edit­ing
CRISPR (clus­tered reg­u­lar­ly inter­spaced short palin­dromic repeats) gene edit­ing is a tech­nol­o­gy that mod­i­fies the sequence of DNA in cells uti­liz­ing a spe­cif­ic RNA-guid­ed nucle­ase (Cas enzyme). Gene edit­ing enzymes act as mol­e­c­u­lar word proces­sors to cor­rect the genet­ic code at the pre­cise spot where it is malfunctioning.

About Ion­is Phar­ma­ceu­ti­cals, Inc.
For more than 30 years, Ion­is has been the leader in RNA-tar­get­ed ther­a­py, pio­neer­ing new mar­kets and chang­ing stan­dards of care with its nov­el anti­sense tech­nol­o­gy. Ion­is cur­rent­ly has three mar­ket­ed med­i­cines and a pre­mier late-stage pipeline high­light­ed by indus­try-lead­ing car­dio­vas­cu­lar and neu­ro­log­i­cal fran­chis­es. Our sci­en­tif­ic inno­va­tion began and con­tin­ues with the knowl­edge that sick peo­ple depend on us, which fuels our vision of becom­ing a lead­ing, ful­ly inte­grat­ed biotech­nol­o­gy company.

To learn more about Ion­is, vis­it www​.ion​is​phar​ma​.com and fol­low us on Twit­ter @ionispharma.

About Metageno­mi
Metageno­mi is a gene edit­ing com­pa­ny com­mit­ted to devel­op­ing poten­tial­ly cura­tive ther­a­peu­tics by lever­ag­ing a pro­pri­etary tool­box of next-gen­er­a­tion gene edit­ing sys­tems to accu­rate­ly edit DNA where cur­rent tech­nolo­gies can­not. Our metage­nomics-pow­ered dis­cov­ery plat­form and ana­lyt­i­cal exper­tise reveal nov­el cel­lu­lar machin­ery sourced from oth­er­wise unknown organ­isms. We adapt and forge these nat­u­ral­ly evolved sys­tems into pow­er­ful gene edit­ing sys­tems that are ultra-small, extreme­ly effi­cient, high­ly spe­cif­ic and have a decreased risk of immune response. These sys­tems fuel our pipeline of nov­el med­i­cines and can be lever­aged by part­ners. Our goal is to rev­o­lu­tion­ize gene edit­ing for the ben­e­fit of patients around the world. For more infor­ma­tion, please vis­it https://​metageno​mi​.co/.

Ion­is’ For­ward-look­ing State­ments
This press release includes for­ward-look­ing state­ments regard­ing our col­lab­o­ra­tion and license agree­ment with Metageno­mi, Inc., the ther­a­peu­tic and com­mer­cial poten­tial of genet­ic tar­gets using gene edit­ing tech­nolo­gies, Ion­is’ tech­nolo­gies and Ion­is’ oth­er prod­ucts in devel­op­ment. Any state­ment describ­ing Ion­is’ goals, expec­ta­tions, finan­cial or oth­er pro­jec­tions, inten­tions or beliefs is a for­ward-look­ing state­ment and should be con­sid­ered an at-risk state­ment. Such state­ments are sub­ject to cer­tain risks and uncer­tain­ties, includ­ing those relat­ed to the impact COVID-19 could have on our busi­ness, and includ­ing but not lim­it­ed to those relat­ed to our com­mer­cial prod­ucts and the med­i­cines in our pipeline, and par­tic­u­lar­ly those inher­ent in the process of dis­cov­er­ing, devel­op­ing and com­mer­cial­iz­ing med­i­cines that are safe and effec­tive for use as human ther­a­peu­tics, and in the endeav­or of build­ing a busi­ness around such med­i­cines. Ion­is’ for­ward-look­ing state­ments also involve assump­tions that, if they nev­er mate­ri­al­ize or prove cor­rect, could cause its results to dif­fer mate­ri­al­ly from those expressed or implied by such for­ward-look­ing statements.

Although Ion­is’ for­ward-look­ing state­ments reflect the good faith judg­ment of its man­age­ment, these state­ments are based only on facts and fac­tors cur­rent­ly known by Ion­is. As a result, you are cau­tioned not to rely on these for­ward-look­ing state­ments. These and oth­er risks con­cern­ing Ion­is’ pro­grams are described in addi­tion­al detail in Ion­is’ annu­al report on Form 10‑K for the year end­ed Dec. 31, 2021, and our most recent Form 10‑Q quar­ter­ly fil­ing, which are on file with the Secu­ri­ties and Exchange Com­mis­sion. Copies of these and oth­er doc­u­ments are avail­able at www​.ion​is​phar​ma​.com.

In this press release, unless the con­text requires oth­er­wise, Ion­is,” Com­pa­ny,” we,” our,” and us” refers to Ion­is Phar­ma­ceu­ti­cals and its subsidiaries.

Ion­is Phar­ma­ceu­ti­cals® is a trade­mark of Ion­is Phar­ma­ceu­ti­cals, Inc.

SOURCE: Ion­is Phar­ma­ceu­ti­cals, Inc.