Metagenomi to regain full development rights to its wholly-owned base editing and RIGS systems

Enhances strate­gic flex­i­bil­i­ty and con­trol of key gene edit­ing tech­nolo­gies and programs

Metageno­mi, Inc. (Nas­daq: MGX), a pre­ci­sion genet­ic med­i­cines com­pa­ny com­mit­ted to devel­op­ing cura­tive ther­a­peu­tics for patients using its pro­pri­etary, com­pre­hen­sive metage­nomics-derived gene edit­ing tool­box, today announced it has regained full glob­al rights to research, devel­op, man­u­fac­ture, and com­mer­cial­ize its whol­ly-owned gene edit­ing tech­nolo­gies, includ­ing base edi­tors and RNA-medi­at­ed inte­gra­tion sys­tems (RIGS), which were pre­vi­ous­ly sub­ject to exclu­sive rights grant­ed to Mod­er­na, Inc. Metageno­mi and Mod­er­na have mutu­al­ly agreed to ter­mi­nate their col­lab­o­ra­tion on pri­ma­ry hyper­ox­aluria type 1 (PH1), and rights to devel­op the PH1 pro­gram, as well as all oth­er rights grant­ed under the col­lab­o­ra­tion, will be returned as part of the termination.

We are pleased to regain full con­trol of the devel­op­ment of base edit­ing tech­nol­o­gy and RIGS sys­tems so that we may advance them in areas of sig­nif­i­cant need, such as Alpha‑1 antit­rypsin defi­cien­cy and Wilson’s dis­ease,” said Bri­an C. Thomas, Ph.D., Chief Exec­u­tive Offi­cer & Founder, Metageno­mi, Inc.

Mod­er­na’s involve­ment in the col­lab­o­ra­tion, which includes the PH1 pro­gram, end­ed in con­junc­tion with a strate­gic pri­or­i­ti­za­tion by the com­pa­ny. Mod­er­na remains a Metageno­mi shareholder.

This announce­ment rep­re­sents a renewed oppor­tu­ni­ty to advance cura­tive genet­ic med­i­cine through the trans­la­tion of Metagenomi’s broad tool­box of whol­ly-owned gene edit­ing tech­nolo­gies, as well as a broad­ened abil­i­ty to engage with part­ners in tar­get-spe­cif­ic appli­ca­tion of these technologies. 

Over the past sev­er­al years, we have built inter­nal capa­bil­i­ties and exper­tise required to advance pro­grams to the next phase of devel­op­ment,” said Dr. Thomas. In addi­tion to expo­nen­tial growth in terms of tal­ent and foot­print, we have lever­aged sig­nif­i­cant pri­vate and pub­lic fund­ing to great­ly expand our gene edit­ing tool­box, and estab­lished in-house man­u­fac­tur­ing. With this announce­ment, we con­tin­ue to have the oppor­tu­ni­ty to accel­er­ate our mis­sion to become the pre­mier gene edit­ing company.”

Con­fer­ence Call Information

The Com­pa­ny will dis­cuss this announce­ment on a con­fer­ence call today, Wednes­day, May 1, at 5:00pm ET. To join and reg­is­ter for the call please vis­it the link here. 

About RNA-medi­at­ed inte­gra­tion sys­tems (RIGS

Our RIGS are being devel­oped in order to encode any type of genom­ic mod­i­fi­ca­tion in an RNA tem­plate, and thus cre­ate any type of genome mod­i­fi­ca­tion nec­es­sary to address a dis­ease. Lit­tle RIGS describes sys­tems used for prime edit­ing (e.g., for small genom­ic replace­ments such as trans­ver­sions, tran­si­tions, inser­tions, and dele­tions), while Big RIGS describes sys­tems capa­ble of mak­ing large tar­get­ed genom­ic integrations.

About base edit­ing, includ­ing ultra-small (SMART) systems 

Our base edi­tors are high­ly active and pro­gress­ing towards in vivo ther­a­peu­tic appli­ca­tions. Using PAM inter­act­ing domain engi­neer­ing, we expand­ed the genome tar­getabil­i­ty of our base edi­tors by 5‑fold com­pared to SpCas9 base editors. 

We iden­ti­fied a series of nov­el, high­ly effi­cient ultra-small nucle­as­es and demon­strat­ed our abil­i­ty to engi­neer these sys­tems into ultra-small base edi­tors and prime edi­tors. We believe our SMART base edi­tors which are as small as 623 amino acids, rep­re­sent some of the small­est edit­ing sys­tems in the indus­try. As the size of these sys­tems is well-with­in the pack­ag­ing lim­its of AAV vec­tors, our SMART base edi­tors could great­ly expand both the deliv­ery and tar­getabil­i­ty options of these sys­tems for dis­ease tar­gets out­side the liver.

About Metageno­mi

Metageno­mi is a pre­ci­sion genet­ic med­i­cines com­pa­ny com­mit­ted to devel­op­ing cura­tive ther­a­peu­tics for patients using its pro­pri­etary, com­pre­hen­sive metage­nomics-derived tool­box. Metageno­mi is har­ness­ing the pow­er of metage­nomics, the study of genet­ic mate­r­i­al recov­ered from the nat­ur­al envi­ron­ment, to unlock four bil­lion years of micro­bial evo­lu­tion to dis­cov­er and devel­op a suite of nov­el edit­ing tools capa­ble of cor­rect­ing any type of genet­ic muta­tion found any­where in the genome. Its com­pre­hen­sive genome edit­ing tool­box includes pro­gram­ma­ble nucle­as­es, base edi­tors, and RNA and DNA-medi­at­ed inte­gra­tion sys­tems (includ­ing prime edit­ing sys­tems and clus­tered reg­u­lar­ly inter­spaced short palin­dromic repeat asso­ci­at­ed trans­posas­es). Metageno­mi believes its diverse and mod­u­lar tool­box posi­tions the com­pa­ny to access the entire genome and select the opti­mal tool to unlock the full poten­tial of genome edit­ing for patients. For more infor­ma­tion, please vis­it https://​metageno​mi​.co.

Cau­tion­ary Note Regard­ing Forward‐​Looking Statements

This press release contains​“forward-looking state­ments” with­in the mean­ing of Sec­tion 27A of the Secu­ri­ties Act of 1933 and Sec­tion 21E of the Secu­ri­ties Exchange Act of 1934, each as amend­ed. Such state­ments, which are often indi­cat­ed by terms such as​“anticipate,”​“believe,”​“could,”​“esti­mate,”​“expect,”​“goal,”​“intend,”​“look for­ward to,”​“may,”​“plan,”​“poten­tial,”​“pre­dict,”​“project,”​“should,”​“will,”​“would” and sim­i­lar expres­sions, include, but are not lim­it­ed to, any state­ments relat­ing to our growth strat­e­gy and prod­uct devel­op­ment pro­grams, includ­ing the tim­ing of and our abil­i­ty to con­duct IND-enabling stud­ies, make reg­u­la­to­ry fil­ings such as INDs and oth­er appli­ca­tions and to obtain reg­u­la­to­ry approvals for our prod­uct can­di­dates, state­ments con­cern­ing the poten­tial of ther­a­pies and prod­uct can­di­dates, and any oth­er state­ments that are not his­tor­i­cal facts. For­ward look­ing state­ments are based on management’s cur­rent expec­ta­tions and are sub­ject to risks and uncer­tain­ties that could neg­a­tive­ly affect our busi­ness, oper­at­ing results, finan­cial con­di­tion, and stock val­ue. Fac­tors that could cause actu­al results to dif­fer mate­ri­al­ly from those cur­rent­ly antic­i­pat­ed include: risks relat­ing to our growth strat­e­gy; our abil­i­ty to obtain, per­form under, and main­tain financ­ing and strate­gic agree­ments and rela­tion­ships; risks relat­ing to the results of research and devel­op­ment activ­i­ties; risks relat­ing to the tim­ing of start­ing and com­plet­ing clin­i­cal tri­als; uncer­tain­ties relat­ing to pre­clin­i­cal and clin­i­cal test­ing; our depen­dence on third par­ty sup­pli­ers; our abil­i­ty to attract, inte­grate and retain key per­son­nel; the ear­ly stage of prod­ucts under devel­op­ment; our need for sub­stan­tial addi­tion­al funds; gov­ern­ment reg­u­la­tion; patent and intel­lec­tu­al prop­er­ty mat­ters; com­pe­ti­tion; as well as oth­er risks described in​“Risk Fac­tors,” in our most recent Form 10‑K on file with the SEC. We express­ly dis­claim any oblig­a­tion or under­tak­ing to release pub­licly any updates or revi­sions to any for­ward-look­ing state­ments con­tained here­in to reflect any change in our expec­ta­tions or any changes in events, con­di­tions or cir­cum­stances on which any such state­ment is based, except as required by law, and we claim the pro­tec­tion of the safe har­bor for for­ward-look­ing state­ments con­tained in the Pri­vate Secu­ri­ties Lit­i­ga­tion Reform Act of 1995.

Investor Con­tact:

Simon Harnest — CIO, SVP Investor Relations


Media Con­tact:

Ash­lye Hodge — Com­mu­ni­ca­tions Manager