A day af­ter Eli Lil­ly inked a $1 bil­lion-plus deal to part­ner with a qui­et RNA edit­ing start­up, a new play­er has emerged to turn up the vol­ume.

ADARx Phar­ma­ceu­ti­cals un­cloaked on Thurs­day with a $75 mil­lion Se­ries B round to ush­er its RNA pro­grams in­to the clin­ic. The San Diego-based biotech was found­ed in 2019 to dis­cov­er ther­a­pies that pre­cise­ly tar­get and ed­it sin­gle-point mu­ta­tions on RNA tran­scripts.

Un­like gene edit­ing, which looks to di­rect­ly — and per­ma­nent­ly – al­ter DNA, the goal of RNA edit­ing is to in­ter­cept the bro­ken mes­sages sent out by the DNA of pa­tients with cer­tain dis­eases and cor­rect it be­fore it gets turned in­to pro­teins. To do so, sci­en­tists are ex­ploit­ing a nat­u­ral­ly oc­cur­ring en­zyme called ADAR, or adeno­sine deam­i­nas­es act­ing on RNA.

ADARx’s first edit­ing pro­gram is be­ing de­vel­oped for Al­pha-1 an­tit­rypsin de­fi­cien­cy (AAT de­fi­cien­cy), an in­her­it­ed con­di­tion in which AAT, a pro­tein that pro­tects the lungs, isn’t the cor­rect shape and gets stuck in the liv­er where it’s pro­duced. Pa­tients with AAT de­fi­cien­cy are at an in­creased risk for both lung and liv­er dis­ease.

The team is helmed by Zhen Li, who led siR­NA de­vel­op­ment at Mer­ck be­fore tak­ing the se­nior VP role at Ar­row­head Phar­ma­ceu­ti­cals in 2014. Rui Zhu, a for­mer Ar­row­head col­league, al­so hopped on board as VP of R&D.

“We are ex­cit­ed that our RNA base edit­ing tech­nol­o­gy could give rise to a sig­nif­i­cant pipeline of high­ly ef­fec­tive ther­a­peu­tics,” Li said in a state­ment, adding that the com­pa­ny is al­so work­ing on tech for in­hi­bi­tion, degra­da­tion and de­liv­ery across a range of ge­net­ic, car­diometa­bol­ic and cen­tral ner­vous sys­tem dis­eases.

The RNA edit­ing space is heat­ing up, with Roche tee­ing up a $3 bil­lion-plus deal for Shape’s RNA edit­ing plat­form back in Au­gust. The deal came on the heels of Shape’s $112 mil­lion Se­ries B round in Ju­ly, with Roche hop­ing to dis­cov­er po­ten­tial “one-time” cures for Alzheimer’s, Parkin­son’s and oth­er rare dis­eases. And just yes­ter­day, Eli Lil­ly put down $50 mil­lion up­front and $1.25 bil­lion in biobucks for ac­cess to five RNA edit­ing can­di­dates from the Dutch biotech Pro­QR Ther­a­peu­tics.

“Or­biMed is pleased to con­tin­ue to back Zhen Li and her team in the cre­ation of an ex­cit­ing oligonu­cleotide-based drug com­pa­ny, which is cre­at­ing new treat­ment modal­i­ties,” said Carl Gor­don, found­ing part­ner at Or­biMed, which co-led ADARx’s Se­ries B round along with SR One Cap­i­tal Man­age­ment. Sirona Cap­i­tal and Lil­ly Asia Ven­tures al­so chipped in.

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.