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.”

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”

CDC’s Robert Redfield, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust of the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

Laura Shawver (Silverback Therapeutics)

Fol­low­ing a hefty Se­ries B, Sil­ver­back Ther­a­peu­tics quick­ly pulls in $85M for 'an im­por­tant growth phase'

Months after reeling in a $78 million Series B round, Silverback Therapeutics has hooked an even larger Series C.

The Seattle-based company announced Wednesday that it netted $85 million from a slate of new and previous investors. The quick boost could be a sign that an IPO is on the way.

In an email, Silverback CEO Laura Shawver told me she was “not able to provide any additional comments about Silverback” beyond what was shared in the company’s news release. In the prepared statement, she said the company is at “an important growth phase.”

Covid-19 roundup: Op­er­a­tion Warp Speed's 7th vac­cine is live at­ten­u­at­ed; Small biotech touts big suc­cess where gi­ants have failed

Operation Warp Speed is stacking its vaccine portfolio with a “TBD” new candidate: a live attenuated vaccine that can be administered in a single dose, potentially as an oral formulation rather than an injection.

Sound familiar?

That could be because the unannounced candidate appears to match the profile of an inoculation being developed by Merck, according to Bloomberg, which first reported the development based on a presentation by Moncef Slaoui.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.

On­ly five months af­ter a Se­ries A launch, Taysha goes pub­lic with $157M IPO

As has been the trend in 2020, Taysha Gene Therapies has become the latest biotech to make a quick ascent from a small, privately-funded company to enjoying its very own Nasdaq ticker.

The Dallas-based biotech raised $157 million for its IPO after pricing shares at $20 apiece Thursday, the high-point of its expected range. Initially pegging $100 million in financing, Taysha offered a little less than 8 million shares and will trade under the $TSHA symbol.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,500+ biopharma pros reading Endpoints daily — and it's free.