James Wil­son turns to old men­tor for gene ther­a­py star­tup's star-stud­ded $115M launch round

Gene ther­a­pies have long held promise for rare dis­eases — many of which are mono­genic — as an el­e­gant cure that cor­rects the sin­gu­lar un­der­ly­ing ge­net­ic de­fects. The ex­e­cu­tion, though, has been any­thing but el­e­gant, spawn­ing headaches about safe­ty is­sues and de­liv­ery meth­ods.

James Wil­son

Penn pro­fes­sor James Wil­son knows the roller coast­er ride all too well. And as the field gains steam fol­low­ing the his­toric ap­proval of the first gene ther­a­py with sev­er­al more lined up at the FDA, he is team­ing up with a rare dis­ease ex­pert and an in­dus­try vet to de­vel­op new ther­a­peu­tics us­ing a tech­nol­o­gy that he’s helped per­fect.

Emerg­ing out of a re­search, col­lab­o­ra­tion and li­cense agree­ment with Penn’s Gene Ther­a­py Pro­gram and Or­phan Dis­ease Cen­ter, Pas­sage Bio has packed its list of sup­port­ers with mar­quee bio­phar­ma in­vestors: Or­biMed Ad­vi­sors, Fra­zier Health­care Part­ners, Ver­sant Ven­tures, New Leaf Ven­ture Part­ners, Vi­vo Cap­i­tal and Lil­ly Asia Ven­tures.

Stephen Squin­to

They are col­lec­tive­ly de­vot­ing $115.5 mil­lion to Pas­sage Bio’s plan of de­vel­op­ing five AAV-based ther­a­pies for rare mono­genic dis­eases of the cen­tral ner­vous sys­tem, tak­ing over once its aca­d­e­m­ic part­ners fin­ish the IND-en­abling pre­clin­i­cal work. Un­der the pact with Penn, the biotech might al­so nab a few more pre­clin­i­cal pro­grams and li­cense new IP lat­er.

Pas­sage Bio will start with two in­di­ca­tions with an eye for clin­i­cal en­try in ear­ly 2020: GM1 gan­gliosi­do­sis (GM1), a neu­ron-de­stroy­ing dis­or­der most com­mon in in­fants; and fron­totem­po­ral de­men­tia, which over­whelm­ing­ly af­fects the el­der­ly.

Alex­ion co-founder and Or­biMed part­ner Stephen Squin­to is run­ning the shop as in­ter­im CEO, while Tachi Ya­ma­da — a VC at Fra­zier for­mer­ly of Glax­o­SmithK­line and Take­da — takes on the chair­man’s role.

As Xcon­o­my notes, Ya­ma­da was an old men­tor of Wil­son’s who of­fered cru­cial sup­port in the im­me­di­ate af­ter­math of a clin­i­cal tri­al that led to the death of a teenag­er, Jesse Gelsinger, and sent the gene ther­a­py field in­to a harsh win­ter.

Tachi Ya­ma­da

Wil­son has since co-found­ed Re­genxbio and ad­vised Sol­id Bio, a biotech de­vel­op­ing a gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy. A year ago, how­ev­er, he abrupt­ly re­signed from Sol­id Bio’s board and sub­se­quent­ly sound­ed an alarm on high-dose AAV stud­ies based on tox­ic re­ac­tions in mon­keys.

There was no men­tion of that in the state­ment about Pas­sage Bio Thurs­day. In­stead, Wil­son her­ald­ed the “tru­ly unique part­ner­ship.”

“Our team at Penn is ex­treme­ly ex­pe­ri­enced and has been on the cut­ting edge of AAV re­search for over 20 years,” he said. “We are con­fi­dent in this team’s abil­i­ty to move new treat­ments for rare CNS mono­genic dis­eases through clin­i­cal de­vel­op­ment in an ef­fort to one day pro­vide new treat­ment op­tions for pa­tients with chron­ic un­met needs with high mor­tal­i­ty.”

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Vlad Coric, Biohaven CEO

Vlad Coric charts course for 'New Bio­haven' with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the “New Biohaven” post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Ying Huang, Legend CEO

Lentivi­ral vec­tor ramp-up: J&J and Leg­end to in­vest $500M in New Jer­sey man­u­fac­tur­ing to sup­port Carvyk­ti

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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