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.”

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

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US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.

Pfiz­er and CD­MOs ramp up Paxlovid man­u­fac­tur­ing with Kala­ma­zoo plant ex­pan­sion lead­ing the way

As the Covid-19 pandemic continues to evolve, pharma companies and manufacturers are exploring how to step up production on antivirals.

Pfizer is planning to expand its Kalamazoo-area facility to increase manufacturing capabilities for the oral Covid-19 antiviral Paxlovid, according to a report from Michigan-based news site MLive. The expansion of the facility, which serves as Pfizer’s largest manufacturing location, is expected to create hundreds of “high-skilled” STEM jobs, MLive reported. No details about the project’s cost and timeline have been released, but according to MLive, Pfizer will announce the details of the expansion at some point in early June.

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FDA spells out the rules and re­stric­tions for states seek­ing to im­port drugs from Cana­da

The FDA is offering more of an explanation of the guardrails around its program that may soon allow states to import prescription drugs in some select circumstances from Canada, but only if such imports will result in significant cost reductions for consumers.

While the agency has yet to sign off on any of the 5 state plans in the works so far, and PhRMA’s suit to block the Trump-era rule allowing such imports is stalled, the new Q&A guidance spells out the various restrictions that states will have to abide by, potentially signaling that a state approval is coming.

Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

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Simba Gill, CEO of Evelo Biosciences

While down 87% YOY, Evelo gets Flag­ship and oth­ers to in­fuse new cap­i­tal for come­back hope

Just four years after Flagship spinout Evelo Biosciences went public in an IPO worth $85 million, the biotech has seen its share price tank from $13 a share this time last year (ultimately reaching a peak of over $17) to now under $1.50. And today, it looks like Flagship still thinks the fledging biotech, in a down market, is still worth something after initial pre-IPO backing from the likes of Google’s GV, Celgene, Mayo Clinic and Alexandria Venture.

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Pfizer CEO Albert Bourla at the World Economic Forum (Gian Ehrenzeller/Keystone via AP Images)

All about ac­cess: Pfiz­er moves to a non-prof­it mod­el for drug sales in 45 low­er-in­come coun­tries

Leading the way to increase access to cheaper drugs worldwide, Pfizer said Wednesday it will provide all current and future patent-protected medicines and vaccines available in the US or EU on a not-for-profit basis to about 1.2 billion people in 45 lower-income countries.

Rwanda, Ghana, Malawi, Senegal and Uganda are the first five countries to sign on to this accord, which will also seek to blaze new paths for quick and efficient regulatory and procurement processes to reduce the usual delays in making new medicines and vaccines available in these countries.

Almirall is tapping artificial intelligence on behalf of its sales force for insights and efficiencies. (via Shutterstock)

Almi­rall rolls out sales rep ar­ti­fi­cial in­tel­li­gence sys­tem, cut­ting pre-call prep and 'wind­shield time'

Dermatology specialty pharma Almirall is making its sales reps smarter. Not with extra training or educational courses, but instead with artificial intelligence tools.

It began a soft launch of a sales rep AI and machine learning platform it calls Polaris last August in one of its 7 US coverage regions. The platform from Aktana gathers information from across Almirall internal sources and external ones – such as claims and prescribing data – to generate insights for reps. Now, instead of spending hours prepping for a sales call, Polaris can generate details about a physician’s preferences, past behaviors and prescription habits for reps in minutes, said Almirall head of commercial operations Vincent Cerio.

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