No­var­tis co-opts Ho­mol­o­gy's gene-edit­ing tech for R&D pipeline

In the lat­est move to build up No­var­tis’ eye drug pipeline, the phar­ma gi­ant is sink­ing fresh cap­i­tal in­to a gene edit­ing com­pa­ny to work on se­cret tar­gets in oph­thal­mol­o­gy.

Fi­nan­cial de­tails weren’t dis­closed, but we do know that No­var­tis made an up­front pay­ment and an eq­ui­ty in­vest­ment in the start­up Ho­mol­o­gy Med­i­cines. The deal gets No­var­tis ac­cess to Ho­mol­o­gy’s gene edit­ing plat­form, which both com­pa­nies hope will lead to new drug tar­gets.

As part of the deal, No­var­tis will pay for fur­ther de­vel­op­ment of the plat­form, ad­vanc­ing new pro­grams and leav­ing the R&D deal some­what open-end­ed to ex­plore oth­er dis­ease ar­eas with the tech.

Arthur Tzian­a­bos

Ho­mol­o­gy’s gene edit­ing tech dif­fers from CRISPR, which us­es an en­zyme from bac­te­ria that “chops” DNA to dis­rupt a dam­aged gene. Ho­mol­o­gy’s CEO Arthur Tzian­a­bos said the CRISPR ap­proach can be prob­lem­at­ic when adding whole genes.

In­stead, Ho­mol­o­gy is em­ploy­ing vi­ral vec­tors to en­cour­age what’s called “ho­mol­o­gous re­com­bi­na­tion” — the body’s nat­ur­al re­sponse to “cor­rect­ing” genes that have been dam­aged. In short, the com­pa­ny pack­ages a sin­gle strand of syn­thet­ic DNA in­side a non-in­fec­tious virus, which then car­ries the DNA to the body’s cells.

“The DNA is de­signed to be a per­fect mir­ror im­age of the DNA you want to take out,” Tzian­a­bos said. Pair­ing the cor­rect­ed DNA with its dam­aged twin in­side the cell is what pro­motes ho­mol­o­gous re­com­bi­na­tion, or the swap­ping of nu­cleotide se­quences be­tween two sim­i­lar strands of DNA.

In the­o­ry, the healthy strand would swap or in­sert cor­rect se­quences where need­ed in pa­tients with mu­tat­ed or dam­aged genes. Out with the bad, dam­aged chunk of DNA, in with the new, cor­rect one.

The idea isn’t new. Log­icBio, based in Fos­ter City, is do­ing some­thing sim­i­lar but with a dif­fer­ent virus. Tzian­a­bos said it’s Ho­mol­o­gy’s ade­no-as­so­ci­at­ed virus (AAV) that’s unique, and it al­lows them to cor­rect “a lot more cells” than Log­icBio can.

No­var­tis par­tic­i­pat­ed in Ho­mol­o­gy’s $83.5 mil­lion Se­ries B round this Au­gust, and is now deep­en­ing its ties with the com­pa­ny with this new R&D agree­ment.

As part of the new deal with No­var­tis, Ho­mol­o­gy will be ex­plor­ing undis­closed tar­gets in oph­thal­mol­o­gy (a key re­search area for No­var­tis), as well as tar­gets in ge­net­ic blood dis­or­ders. No­var­tis has world­wide rights to Ho­mol­o­gy’s plat­form for se­lect oph­thalmic tar­gets and an undis­closed he­mo­glo­binopa­thy dis­ease. Ho­mol­o­gy gets to re­tain US com­mer­cial rights (and a share of US prof­its) for in vi­vo ap­pli­ca­tions re­lat­ed to the he­mo­glo­binopa­thy pro­gram.

The col­lab­o­ra­tion makes sense for No­var­tis, as the com­pa­ny has a strong in­ter­est in oph­thal­mol­o­gy. Its block­buster Eylea ri­val RTH258 has been tout­ed as a $1-bil­lion-plus drug for wet age-re­lat­ed mac­u­lar de­gen­er­a­tion. No­var­tis plans to seek FDA ap­proval for the drug by the end of next year.

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