Art Levin is liv­ing his “sci­en­tist’s dream come true.”

The Avid­i­ty Bio­sciences CSO set out years ago to build a new class of oligonu­cleotide-based ther­a­pies for se­vere mus­cle dis­eases, like Duchenne mus­cu­lar dy­s­tro­phy (DMD) and my­oton­ic dy­s­tro­phy (DM1) — rare, ge­net­ic dis­or­ders that caus­es pro­gres­sive mus­cle weak­ness.

“As you might imag­ine, in the 15 or 16 years I’ve been work­ing on this, I’ve seen boys go from be­ing tod­dlers, to be­ing wheel­chair-bound, or even ven­ti­la­tor-bound,” he said, re­fer­ring to DMD. “It would be re­al­ly … a sci­en­tif­ic dream of mine to ac­tu­al­ly make a dif­fer­ence for this pa­tient pop­u­la­tion.”

At JP Mor­gan, Avid­i­ty is up­dat­ing in­vestors that their DM1 can­di­date is of­fi­cial­ly off to the races — head­ed for a Phase I/II tri­al in the sec­ond half of this year. The team is al­so div­ing in­to a part­ner­ship with Bris­tol My­ers Squibb sub­sidiary MyoKar­dia to ex­plore their ap­proach in car­diac tis­sue.

“It’s re­al­ly grat­i­fy­ing that this tech­nol­o­gy that we built and en­gi­neered in-house our­selves is com­ing to fruition,” Levin said.

DM1 is caused by hun­dreds of thou­sands of nu­cleotide re­peats at the end of a gene called DMPK. When the DMPK gene is tran­scribed in­to RNA, the RNA al­so car­ries the nu­cleotide re­peats, which form a hair­pin loop struc­ture, mak­ing the RNA tox­ic to the cell.

“Us­ing the tech­nol­o­gy that we’re talk­ing about, you can re­verse that and re­place these mis­placed pro­teins or mis­s­pliced RNA with prop­er­ly spliced RNA,” Levin told End­points back in 2019. “In the ac­tu­al treat­ment of pa­tients, we should ac­tu­al­ly be able to re­verse some of the na­ture of this dis­ease.”

The idea that you can use oligonu­cleotides to mod­i­fy hu­man RNA isn’t new. The prob­lem has al­ways been de­liv­ery, Levin said.

“Cells have evolved over eons to try and keep out RNA and keep out for­eign DNA,” he said. “So you re­al­ly have to trick the cell in­to want­i­ng to take up our oligonu­cleotide ther­a­peu­tic.”

The team is work­ing on An­ti­body Oligonu­cleotide Con­ju­gates (AOCs) — oligonu­cleotides com­bined with mon­o­clon­al an­ti­bod­ies in­to a sin­gle con­ju­gate that can be more eas­i­ly di­rect­ed to pre­cise tis­sues.

Avid­i­ty al­so has AOC pro­grams for fa­cioscapu­lo­humer­al mus­cu­lar dy­s­tro­phy (FSHD), Duchenne mus­cu­lar dy­s­tro­phy (DMD), and mus­cle at­ro­phy (MA). The FSHD pro­gram is head­ed for IND en­abling stud­ies this year, and the biotech ex­pects to file for a clin­i­cal tri­al in 2022. There are three pro­grams with­in DMD, the leader of which tar­gets Ex­on 44. The com­pa­ny plans on sub­mit­ting for per­mis­sion to en­ter the clin­ic in 2022.

In June, the biotech land­ed a near­ly $300 mil­lion IPO to make it all hap­pen. They tagged $65 mil­lion for their DM1 can­di­date, $35 mil­lion for DMD, and $25 mil­lion for MA, ac­cord­ing to an S-1/A. 

“We al­so in­tend to pur­sue the de­vel­op­ment of AOCs in cell types in ad­di­tion to mus­cle. In pre­clin­i­cal mod­els, we ob­served the abil­i­ty of AOCs not on­ly to de­liv­er to the liv­er, skele­tal and car­diac mus­cle, but al­so to oth­er tis­sue and cell types, in­clud­ing im­mune cells,” the doc­u­ment states.

Eli Lil­ly holds 6.6% of Avid­i­ty’s stock, ac­cord­ing to the S-1/A. Back in 2019, Avid­i­ty inked a li­cens­ing and re­search deal with Lil­ly, snag­ging $20 mil­lion up­front and an in­vest­ment of $15 mil­lion. Lil­ly is of­fer­ing up to $405 mil­lion more in mile­stones, plus roy­al­ties.

Levin kept mum about what the com­pa­ny is work­ing on with MyoKar­dia, on­ly re­veal­ing that they’re de­vel­op­ing a “sin­gle ther­a­peu­tic en­ti­ty.” The com­pa­nies are keep­ing the fi­nan­cial terms of the deal un­der wraps for now.

“We’re now re­al­ly now see­ing the fruits of all those labors come true,” Levin said. “We can build on peo­ple’s new ap­pre­ci­a­tion for what is a fun­da­men­tal build­ing block of life: RNA. Peo­ple are fi­nal­ly be­gin­ning to re­al­ize that this is a re­al way to cre­ate ther­a­peu­tic en­ti­ties.”

A cor­rec­tion has been made to clar­i­fy that Levin was re­fer­ring to DMD, not DM1, in his quote in the third para­graph.

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses.

In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%.

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.