Ed­i­tas Med­i­cine has re­ceived the FDA’s bless­ing to en­ter the clin­ic with its first ex vi­vo cell ther­a­py — but it will do so with­out CSO Charles Al­bright, who is leav­ing his post for the same gig at an ear­ly-stage com­pa­ny.

The FDA clear­ance was an­nounced min­utes af­ter Ed­i­tas shared word of Al­bright’s de­par­ture ear­ly Mon­day morn­ing. The bit­ter­sweet news caused the biotech’s stock $ED­IT to sink more than 12%, with shares hov­er­ing around $79 apiece.

While we don’t know ex­act­ly where Al­bright is head­ing next, an Ed­i­tas spokesper­son hint­ed that it’s some­thing ear­ly-stage. The Bris­tol My­ers Squibb vet earned his doc­tor­ate in bi­ol­o­gy from MIT, and worked at the Cam­bridge, MA-based biotech for over four years.

“We have an ex­cep­tion­al team in place, and we are con­fi­dent that im­por­tant mile­stones for our pipeline and pro­grams will be met while we search for a re­place­ment for Char­lie,” the spokesper­son said in an email.

Ed­i­tas is now be­gin­ning the search for a new CSO as it aims to dose pa­tients this year in the safe­ty por­tion of a Phase I/II tri­al for its sick­le cell can­di­date, ED­IT-301.

Sick­le cell dis­ease is an in­her­it­ed blood dis­or­der caused by a mu­ta­tion in the be­ta-glo­bin gene that leads to poly­mer­iza­tion of the sick­le he­mo­glo­bin pro­tein (HbS). The dis­ease is char­ac­ter­ized by mis­shapen red blood cells, which can block blood flow and cause ane­mia, pain, or­gan fail­ure and, in some cas­es, death.

ED­IT-301 us­es sick­le pa­tient CD34+ cells that are ge­net­i­cal­ly mod­i­fied us­ing a CRISPR/Cas12a ri­bonu­cle­o­pro­tein (RNP). Ed­i­tas says that red blood cells de­rived from ED­IT-301 CD34+ cells show a sus­tained in­crease in fe­tal he­mo­glo­bin (HbF) pro­duc­tion, which fights sick­le cell dis­ease by in­hibit­ing HbS poly­mer­iza­tion.

Cindy Collins

“The FDA’s clear­ance for ini­ti­a­tion for our ED­IT-301 clin­i­cal tri­al is an ex­cit­ing mo­ment for us and the pa­tients we hope to serve. We look for­ward to bring­ing this po­ten­tial­ly best-in-class, one-time, durable med­i­cine in­to the clin­ic and to pa­tients,” Ed­i­tas CEO Cindy Collins said in a state­ment.

The Phase I/II tri­al, dubbed RU­BY, is ex­pect­ed to kick off this year, and Ed­i­tas plans on sub­mit­ting an­oth­er IND ap­pli­ca­tion for the can­di­date to treat be­ta-tha­lassemia by the end of the year. But be­fore it can be­gin the ef­fi­ca­cy por­tion of the RU­BY study, the biotech says it has to clear up a par­tial clin­i­cal hold by “de­vel­op­ing an im­proved po­ten­cy as­say.”

Back in Au­gust, Ed­i­tas snagged the rights to its lead can­di­date, ED­IT-101, back from Ab­b­Vie. The phar­ma picked up the gene edit­ing pro­gram when it ac­quired Al­ler­gan, which had paid $90 mil­lion up­front to form a CRISPR al­liance with Ed­i­tas a few years ago. The pro­gram cen­ters on LCA10, a rare, in­her­it­ed reti­nal de­gen­er­a­tive dis­ease that ap­pears in child­hood and leads to blind­ness.

Collins said at the time that she was “pleased” to get the oc­u­lar pro­grams back, and that Ed­i­tas planned to con­tin­ue a Phase I/II tri­al for ED­IT-101 called BRIL­LIANCE. The com­pa­ny ex­pects to dose the first pa­tient in an adult mid-dose co­hort of the tri­al in Q1 2021, and read out ini­tial da­ta by the end of the year.

It al­so ex­pects to de­clare a new can­di­date for re­tini­tis pig­men­tosa type 4 (adRP4) by the end of the year.

“I have great­ly en­joyed work­ing along­side the ex­treme­ly tal­ent­ed team here at Ed­i­tas Med­i­cine. To­geth­er, we ad­vanced the rev­o­lu­tion­ary CRISPR gene edit­ing sys­tem in­to a pipeline of ex­per­i­men­tal med­i­cines,” Al­bright said in a state­ment. “I look for­ward to watch­ing Ed­i­tas Med­i­cine’s suc­cess­es con­tin­ue in the fu­ture, de­vel­op­ing many trans­for­ma­tive med­i­cines for peo­ple in great need.”

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.

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.