More three years af­ter Al­ler­gan $AGN dipped its toes in­to the world of CRISPR/Cas9 gene edit­ing by en­list­ing Ed­i­tas $ED­IT as a part­ner — the com­pa­nies are now off to the races with a tri­al in pa­tients with a rare eye dis­ease.

This tri­al marks the first in­stance of us­ing the tech­nol­o­gy to ed­it DNA with­in the body.

Mean­while, part­ners Ver­tex $VRTX and CRISPR Ther­a­peu­tics $CR­SP kick­start­ed a tri­al ear­li­er this year that em­ployed the tech­nol­o­gy to en­gi­neer a gene-edit­ed stem cell ther­a­py to treat pa­tients suf­fer­ing from se­vere he­mo­glo­binopathies, a group of blood dis­or­ders. Sang­amo Ther­a­peu­tics $SG­MO has test­ed its old­er zinc fin­ger tech­nol­o­gy in pa­tients with two lyso­so­mal stor­age dis­eases, but a set­back dif­fused en­thu­si­asm for the pro­gram — but the com­pa­ny bounced back with en­cour­ag­ing ear­ly da­ta on its he­mo­phil­ia pro­gram part­nered with Pfiz­er.

Al­ler­gan and Ed­i­tas had orig­i­nal­ly ex­pect­ed to file an ap­pli­ca­tion to test their ther­a­py in hu­mans by the end of 2017, but man­u­fac­tur­ing trou­ble pushed that time­line to Oc­to­ber 2018. To get the FDA en­dorse­ment, Ed­i­tas fought off a stiff patent chal­lenge — and like its peers — bat­ted back per­sis­tent ques­tions about off-tar­get ef­fects to ad­e­quate­ly con­vince the reg­u­la­tor it was armed and ready to make the jump from an­i­mal stud­ies to hu­mans.

The tri­al, chris­tened Bril­liance, will eval­u­ate their ex­per­i­men­tal ther­a­py AGN-151587 (ED­IT-101) for the treat­ment of Leber con­gen­i­tal amau­ro­sis 10 (LCA10), a rare, in­her­it­ed form of blind­ness caused by mu­ta­tions in the CEP290 gene.

LCA is a group of in­her­it­ed reti­nal de­gen­er­a­tive dis­or­ders caused by mu­ta­tions in at least 18 dif­fer­ent genes, and are con­sid­ered the most com­mon cause of in­her­it­ed child­hood blind­ness, oc­cur­ring in two to three per 100,000 live births glob­al­ly. The most com­mon form of the dis­ease, LCA10, ac­counts for ap­prox­i­mate­ly 20% to 30% of all LCA pa­tients.

Ka­trine Bosley Ed­i­tas

Spark Ther­a­peu­tics’ ap­proved gene ther­a­py Lux­tur­na, which de­liv­ered via a vi­ral vec­tor, is tai­lored to treat an­oth­er rare eye dis­ease due to mu­ta­tions in both copies of the RPE65 gene. The FDA nod sparked Roche’s $4.3 bil­lion takeover of the com­pa­ny, al­though the deal is yet to be con­sum­mat­ed.

The Bril­liance study is set to en­roll 18 pa­tients — and in­clude up to five co­horts and eval­u­ate three dos­es of the drug. Each pa­tient will re­ceive a sin­gle dose of AGN-151587 ad­min­is­tered via an eye in­jec­tion aimed at pho­tore­cep­tor cells in the reti­na.

Ed­i­tas’ se­nior team has seen up­heaval in the past year. In Jan­u­ary, CEO Ka­trine Bosley, who led the com­pa­ny for near­ly five years, an­nounced her ex­it and sev­ered ties with the board, with­out ex­pla­na­tion. Chief fi­nan­cial of­fi­cer An­drew Hack an­nounced his de­ci­sion to leave by March, back in De­cem­ber 2018, and chief med­ical of­fi­cer Ger­ald Cox jumped ship at the end of last year.

Be­set by set­backs, Al­ler­gan’s Brent Saun­ders made the best of a bad sit­u­a­tion by agree­ing to be swal­lowed by Ab­b­Vie $AB­BV in an eye-wa­ter­ing $63 bil­lion deal ear­li­er this year. The US drug­mak­er was swayed by the po­ten­tial of Al­ler­gan’s block­buster Botox fran­chise to sta­bi­lize its rev­enue as its $20 bil­lion cash cow Hu­mi­ra hur­tles to­wards a patent cliff in the Unit­ed States.

So­cial im­age: Shut­ter­stock

GlaxoSmithKline’s Benlysta isn’t alone in the small circle of approved lupus nephritis drugs anymore.

Little Aurinia Pharmaceuticals has gotten the green light from the FDA to start marketing its first and only program, voclosporin, under the brand name Lupkynis — something CEO Peter Greenleaf says it’s been ready to do since December.

Regulators went right down to the wire on the decision, keeping the company and the entire salesforce it’s already assembled on its toes.

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.