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.”

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Michael Rome (Foresite)

In search of 'house­hold health­care brands of the fu­ture,' Fore­site Cap­i­tal rais­es $969M to sa­ti­ate a tech-heavy ap­petite

Back in April 2018, just before Foresite Capital unveiled its $668 million Fund IV and a strategy to focus on tech-driven life science bets, one of its portfolio companies quietly made an announcement.

Fount Therapeutics, a drug discovery outfit backed by Foresite and Eshelman Ventures, had raised $22 million in Series A cash to hatch several fledgling spinouts. “The first ‘NewCo,’ Kinnate, will be focused on developing precision oncology treatments,” read a press release.

S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

CEO Fabrice Chouraqui (Cellarity)

Flag­ship up­start Cel­lar­i­ty gath­ers $123M to fi­nance its ex­plo­ration of cell be­hav­ior, blaz­ing a new path to build­ing a broad pipeline

Right from the start, the discovery plan at Flagship-spawned Cellarity was to take their cues from cell biology and follow them to new drugs. Rather than start with a target and develop a drug to hit it, they’d use new technology to digitally map cell behavior and then develop new drugs from what they learned.

“Over the past decades it has always been about finding a target, about reducing a disease to a single molecular target,” says Fabrice Chouraqui, the Novartis vet who was recruited to run the operation about 9 months ago. “And that approach has produced thousands of life-saving medicines. Yet, this approach has limitations. A molecular target approach is fine when you talk about a simple disease, but for very complex diseases like neurodegeneration, like metabolic disease, like cancer, you hope to really harness the complexity of human biology.