Neu­rol­o­gist and King’s Col­lege Lon­don pro­fes­sor Christo­pher Shaw has been re­search­ing neu­rode­gen­er­a­tive dis­eases like ALS and col­lab­o­rat­ing with drug­mak­ers for the last 25 years in the hopes of push­ing new ther­a­pies for­ward. But un­for­tu­nate­ly, none of those ef­forts have come any­where close to fruition.

“So, you know, af­ter 20 years in the game, I said, ‘Let’s try and do it our­selves,’” he told End­points News. 

In 2019, he launched Avi­ado­Bio along with mol­e­c­u­lar neu­ro­bi­ol­o­gist Youn Bok Lee and vec­tor bi­ol­o­gist Do Young Lee to pur­sue a new take on gene ther­a­py for neu­ro dis­or­ders. And on Thurs­day, they un­wrapped an $80 mil­lion Se­ries A round led to bring their lead can­di­date for fron­totem­po­ral de­men­tia (FTD) in­to the clin­ic.

Christo­pher Shaw

“I was an in­stant be­liev­er,” said Lisa De­schamps, who left her role as CBO of No­var­tis Gene Ther­a­pies to take the helm at Avi­ado­Bio in Oc­to­ber.

The team is tar­get­ing dis­eases that have an ag­gres­sive ear­ly pro­gres­sion, Shaw said. While most of their com­peti­tors are putting vec­tors in­to the cere­brospinal flu­id — a liq­uid found with­in the tis­sue that sur­rounds the brain and spinal cord —  Avi­ado­Bio is look­ing to de­liv­er the vec­tors right to the brain.

“We get fan­tas­tic ex­pres­sion by do­ing that,” Shaw said. “It al­so al­lows us to mi­cro­dose — we’re giv­ing about a thou­sandth of the dose of our com­peti­tors.”

Their lead can­di­date, AVB-PGRN, and an in­trathal­a­m­i­cal­ly dosed ther­a­py de­signed to de­liv­er a func­tion­al copy of the pro­gran­ulin (GRN) gene. FTD is caused by a de­fi­cien­cy of pro­gran­ulin, which plays a role in lyso­so­mal func­tion, or the pro­tein re­cy­cling part of the cell.

“Neu­rons are in­cred­i­bly de­pen­dent on hav­ing good re­cy­cling be­cause the neu­rons you die with are the neu­rons you’re born with,” Shaw said. “So you know, they can’t be ado­les­cents. They can’t have a messy room. They’ve got to have a re­al­ly, re­al­ly crisp, tidy space … So what we’re try­ing to do is just re­turn back to phys­i­o­log­i­cal lev­els this re­al­ly im­por­tant re­cy­cling pro­tein.”

Avi­ado­Bio will com­pete with a hand­ful of play­ers work­ing on po­ten­tial treat­ments for FTD, in­clud­ing GSK-part­nered Alec­tor, which un­veiled da­ta this sum­mer show­ing its an­ti­body AL001 brought pro­gran­ulin lev­els back to near-nor­mal lev­els af­ter a half-a-year. And Ex­pan­sion Ther­a­peu­tics un­veiled an $80 mil­lion Se­ries B round just a cou­ple months ago to bring its small mol­e­cule RNA plat­form one step clos­er to the clin­ic – in­clud­ing a can­di­date for FTD.

De­schamps said Avi­ado­Bio’s can­di­date should en­ter the clin­ic by the end of next year, while an­oth­er pre­clin­i­cal can­di­date for ALS is a bit fur­ther be­hind. The Se­ries A round was led by New En­ter­prise As­so­ci­ates and Mono­graph Cap­i­tal. A hand­ful of big-name back­ers pitched in­to a $16.5 mil­lion seed round last year, in­clud­ing Ad­vent Life Sci­ences, De­men­tia Dis­cov­ery Fund (DDF), F-Prime Cap­i­tal, John­son & John­son In­no­va­tion (JJDC), and LifeArc.

De­spite Big Phar­ma’s re­treat from neu­ro a few years ago, the in­dus­try seems to be warm­ing back up to the field, De­schamps said — and new com­pa­nies are emerg­ing with gene ther­a­pies to take a shot at dif­fi­cult-to-treat con­di­tions. This past sum­mer, for ex­am­ple, Al­cy­one Ther­a­peu­tics emerged from stealth with $23 mil­lion and 12 gene ther­a­pies in tow, with a rare neu­rode­vel­op­men­tal con­di­tion called Rett syn­drome up first.

“I was lead­ing the glob­al neu­ro­science busi­ness at No­var­tis … one of the things we had talked about years ago was how many Big Phar­ma com­pa­nies were ex­it­ing the space. It is quite the op­po­site these days,” De­schamps said. “We see cer­tain­ly a resur­gence across big and small com­pa­nies, you know, com­ing back in­to the neu­ro­science space.”

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Most of the gene therapy world centers around viral vectors, but a few biotechs have sprung up in recent years to try and avoid the safety issues that can come with adeno-associated viruses.

A new biotech has emerged to straddle somewhere “close to the middle,” in the words of Alex Mayweg, Versant managing director and board member of the incubator’s latest startup, Vector BioPharma.

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.